mteb/BIRCO-ClinicalTrial-Test huggingface dataset:
Systemic Lupus Erythematosus (SLE) is a systemic autoimmune disorder. It mainly involves the skin, the joints, the nervous system and the kidney and may be life threatening. SLE is associated with production of autoantibodies and perturbations in regulatory T cells and T helper lymphocytes producing interleukin (IL)-17 (Th17 cells). Treatments include corticosteroids, hydroxychloroquine and immunosuppressive agents. Immunomodulatory effects of vitamin D supplementation in VITRO was recently described, notably the expansion of Treg able to suppress inflammatory responses mediated by CD4+ and CD8+ T cells and the decrease of Th17 cells. Systemic Lupus Erythematosus (SLE) is a systemic autoimmune disorder. It mainly involves the skin, the joints, the nervous system and the kidney and may be life threatening. SLE is associated with production of autoantibodies and perturbations in regulatory T cells and T helper lymphocytes producing interleukin (IL)-17 (Th17 cells). Treatments include corticosteroids, hydroxychloroquine and immunosuppressive agents. Immunomodulatory effects of vitamin D supplementation in VITRO was recently described, notably the expansion of Treg able to suppress inflammatory responses mediated by CD4+ and CD8+ T cells and the decrease of Th17 cells. Objective : To evaluate the cellular immune response after vitamin D supplementation in patients with SLE. Methods : This is an open prospective trial. SLE patients with hypovitaminosis D (< 30 ng/mL) receive vitamin D supplementation.
Up to 10% of patients with cancer will develop symptomatic brain metastases. Given this limited survival it is important to consider quality of life (QOL) when treating these patients. Whole brain radiotherapy (WBRT) can increase survival to 6 month. However, WBRT itself has been shown to reduce QOL by increasing drowsiness, leg weakness and hair loss in patients with brain metastases. Both fatigue and hair loss were reported to have the largest decline in QOL scores when WBRT is used in the prophylactic setting in small cell lung cancer. Recent technological improvements in patient positioning and treatment planning will allow us to treat the whole brain with reduced margins, allowing better sparing of the scalp. In view of the large impact of hair loss on quality of life, the investigators hypothesize to see an improved quality of life with scalp sparing techniques. Study hypothesis: Volumetric arc therapy results in a reduced hair loss and a subsequent clinically important improvement in QOL.
Systemic lupus erythematosis (SLE) is an autoimmune disease, meaning that the body's immune system attacks its own organs and tissues. Within the immune system, B-cells and plasma cells make proteins called antibodies, which in autoimmune disease can bind to one's own tissues and are thus referred to as autoantibodies. Atacicept blocks 2 factors in the body, called BLyS and APRIL, which are important for the maintenance of B-cells and plasma cells, and thus the production of antibodies. This study will assess whether treatment with atacicept can reduce SLE disease activity. Atacicept is still an experimental drug, meaning that it is not available outside of a clinical trial, and that its potential benefits and risks have not been fully determined. A total of 175 subjects are planned to be randomized (35 subjects per treatment arm) in a 1:1:1:1:1 ratio to receive either atacicept 5 mg, atacicept 25 mg, atacicept 75 mg, atacicept 115 mg or matching placebo, given subcutaneously once weekly for 24 weeks. The primary objective of the trial is to evaluate the efficacy of atacicept compared to placebo in reducing SLE disease activity in subjects treated with standard of care (SoC) therapy and to investigate the dose-response relationship. The secondary objectives of the trial are: - To evaluate the effect of atacicept in reducing corticosteroid usage - To evaluate the safety and tolerability profile of atacicept in subjects with SLE - To confirm the PK and PD profiles of atacicept in SLE subjects - To evaluate the changes in the Medical Outcomes Study Short Form General Health Survey [SF-36].
The key of this prospective study is to identify a potentially increased cardiovascular risk in patients with systemic Lupus erythematodes, with and without renal affection. Three groups of patients will be compared. Key of this prospective study is to identify a potentially increased cardiovascular risk in patients with systemic Lupus erythematodes, with and without renal affection. Three group of patients will be compared as followed. First group are patients with known systemic Lupus erythematodes without renal affection, second group are patients with known systemic Lupus erythematodes with renal affection and the third group are patients with a non-autoimmun chronic kidney disease
Retrospective study of the safety and efficacy of hydroxychloroquine among patients with immune Thrombopenia (ITP). Hydroxychloroquine (HCQ) is usually used in Patients with Systemic Lupus Erythematosus (SLE) in order to treat mainly arthralgia or skin manifestations of this disease. A few studies have previously shown that HCQ could also be useful for treating autoimmune cytopenia and particularly autoimmune thrombopenia associated with SLE. In this retrospective study, the investigators have selected patients followed in our center and who have received HCQ either for an authentic SLE or because they had positive antinuclear antibodies (> 1/160e on HEP2 cells) without a definite SLE according to the American College of Rheumatology. The main goal of this study is to compare the efficacy of HCQ in these two subgroups of patients.
Primary Objective: - To investigate the efficacy on skin manifestation of 16 weeks treatment of once daily regimen of hydroxychloroquine sulphate (HCQ) in patients with cutaneous lupus erythematosus (CLE) and systemic lupus erythematosus (SLE) with active skin manifestation (CLASI [Cutaneous Lupus Erythematosus Disease Area and Severity Index] activity score is ≥4) concomitant treatment with or without corticosteroid. Secondary Objectives: - To evaluate the efficacy on skin manifestation and the safety of 16 weeks treatment of once daily regiment of HCQ versus placebo as the reference group in patients with CLE and SLE with active skin manifestation (CLASI activity score is ≥4) concomitant treatment with or without corticosteroid. - To investigate the safety of 16 weeks treatment of once daily regiment of HCQ in patients with CLE and SLE with active skin manifestation concomitant treatment with or without corticosteroid. - To investigate the safety and efficacy of 52 weeks long-term treatment of once daily regimen of HCQ in patients with CLE and SLE - To investigate the influence of the dose reduction of corticosteroid on CLE and SLE patients treated with HCQ concomitant with corticosteroid - To investigate efficacy of once daily regimen of HCQ on systemic symptoms, musculoskeletal symptoms and immunological parameters in SLE patients. Total 59 weeks (screening;4 weeks, treatment;52 weeks, Follow-up; 3 weeks)
This study provides subjects who complete the BEL113750 study the option of continuing treatment with belimumab (10 mg/kg intravenously every 4 weeks) for those randomized to belimumab, or the option to begin treatment with belimumab for those randomized to placebo, as an add-on to their standard of care SLE therapy. This is a multicentre, continuation study of belimumab plus standard of care (SOC) in SLE subjects who completed the Phase III BEL113750 protocol in Northeast Asia. This study provides subjects who complete the BEL113750 study the option of continuing treatment with belimumab (10 mg/kg intravenously every 4 weeks) for those randomized to belimumab, or the option to begin treatment with belimumab for those randomized to placebo, as an add-on to their SOC SLE therapy. Subjects participating in this continuation protocol will continue to be monitored for safety and efficacy, as measured by the SLE responder index. Subjects who complete 48 weeks of treatment on the BEL113750 study and who meet inclusion/exclusion criteria, and provide informed consent, will be given the option to enter the continuation study. All subjects will receive belimumab 10 mg/kg IV infused over 1 hour every 4 weeks. Subjects recruited into this study will continue to receive treatment with belimumab until such time as belimumab becomes commercially available in a subject's country of participation, or the subject elects to participate in another belimumab continuation study for SLE, or until either the subject's physician withdraws the subject from the study, or upon the decision by the sponsor to discontinue further development of belimumab for SLE.
The purpose of this study is to evaluate the efficacy, safety, and tolerability of belimumab in adult patients of black race with systemic lupus erythematosus (SLE; lupus). Study participants receive stable standard therapy for lupus in addition to receiving either placebo (no active medicine) or belimumab. The controlled period of the study is 52 weeks. The random assignment in this study is "2 to 1" which means that for every 3 participants, 2 will receive belimumab and 1 will receive placebo. Participants who successfully complete the 52-week study may enter into a 6-month open-label extension. All participants in the open-label extension receive belimumab plus standard therapy.
This is a multi-center study to evaluate the safety, pharmacokinetics, and efficacy of belimumab intravenous (IV) in pediatric patients 5 to 17 years of age with active systemic lupus erythematosus This is a multi-center study to evaluate the safety, pharmacokinetics, and efficacy of belimumab intravenous (IV) in pediatric patients 5 to 17 years of age with active systemic lupus erythematosus (SELENA SLEDAI score ≥ 6). The study will consist of three phases: a 52-week randomized, placebo-controlled, double-blind phase; a long term open label continuation phase; and a long term safety follow up phase. The long term open label continuation and safety follow up periods will continue for at least 5 years and possibly up to 10 years from a subject's initial treatment with belimumab. Enrolment will be staggered by age cohorts to allow safety and PK interim analyses. Subjects will be randomized to belimumab 10mg/kg or placebo IV monthly dosing while continuing to receive background standard therapy throughout the study. An independent data monitoring committee (IDMC) will monitor the study as it progresses.
This trial is conducted in Europe and the United States of America (USA). The aim of the trial is to investigate the safety and tolerability of NNC0114-0006 in subjects with systemic lupus erythematosus (SLE) concomitantly treated with stable background therapies.
The purpose of this study is to compare the genotype-based personal prescription of cyclophosphamide with the traditional prescription. Cyclophosphamide (CPA) has been one of the most successful therapies for severe Systemic lupus erythematosus (SLE). However, cyclophosphamide can cause severe side effects, including bone marrow suppression, infection, gastrointestinal reaction, hemorrhagic cystitis, and the etc. Significant variation in efficacy and toxicity of CPA has been observed. Since the development of applicable therapeutic drug monitoring (TDM) of cyclophosphamide has been reported, it will help to improve the efficacy and reduce toxicities in SLE treatment. However, the TDM is a passive strategy which usually lags behind the appearance of toxicities. Therefore，it is especially crucial to give individuals genotype-based personal prescription of cyclophosphamide in order to gain the most effective therapies. Thus, the purpose of this study is to compare the genotype-based personal prescription of cyclophosphamide with the traditional prescription, in order to verify the efficacy of the genotype-based personal prescription.
The purpose of this study is to further enhance the existing knowledge regarding the side effects of belimumab when given with other lupus medicines to adults with active systemic lupus erythematosus (SLE). This study mainly focuses on collecting information on serious events that are not that common or may only be seen with long-term treatment. These events include death, serious infections and other infections of interest, cancers, serious mental health problems, including depression and suicide, and serious infusion and hypersensitivity reactions. This study is being done to help understand if treatment with belimumab increases the risk for these types of events. This study will also see if patients receiving belimumab with other lupus medicines can reduce their use of steroids, such as prednisone, over 1 year. Study participants receive standard therapy for SLE in addition to receiving the study drug, either placebo (no active medicine) or belimumab. The controlled period of the study is 52 weeks. The random assignment in this study is "1 to 1" which means that participants have an equal chance of receiving belimumab or placebo. After completion of the 52-week study period, participants will be contacted by phone annually for 4 more years to assess health status. Following the 52-week controlled period, participants who wish to continue treatment with belimumab may be able to do so by being prescribed commercially available belimumab.
The purpose of this registry is to collect additional information regarding the side effects and effectiveness of BENLYSTA (belimumab) when given with other lupus medicines to adults with active systemic lupus erythematosus (SLE). Information will be collected on serious events that are not that common or may only be seen with long-term treatment. These events include death, cancers, serious infections and other infections of interest, and serious mental health problems. Information on the effectiveness of BENLYSTA will also be collected. The registry will enroll 2 groups of patients. One group will include patients who are currently taking lupus medicines along with intravenous BENLYSTA (With BENLYSTA). The other group will include patients who are taking lupus medicines but do not take BENLYSTA (Without BENLYSTA). For every 3 participants enrolled in the registry, 2 will be taking BENLYSTA and 1 will not. After enrollment, changes in lupus medications, including starting or stopping BENLYSTA, are at the discretion of the physician, and all patients will continue to be followed regardless of changes in their lupus medicines until study completion. Physicians will manage the patient in accordance with their medical judgment and standard of care. Data will be collected at enrollment and at 6 month intervals for 5 years.
In this multi-center international study, our aim is to determine the effectiveness of HCQ for primary thrombosis prophylaxis in persistently aPL-positive but thrombosis-free patients without systemic autoimmune diseases. Randomized to receive HCQ or no treatment in addition to their standard regimen. 11 study visits and 10 phone visits over 5 years.
The purpose of this study is to characterize the clinical and molecular profiles of patients with cutaneous lupus. This is a Phase 0, multiple-center observational study of biomarkers and clinical parameters in patients with lupus, including discoid lupus erythematosus (DLE), subacute cutaneous LE (SCLE) and active systemic lupus erythematosus (SLE). There is no study-related therapeutic intervention and this protocol will not restrict or introduce any medical interventions including medications. Study participants will undergo procedures that include collection of urine, blood samples, and skin biopsy. At least 30 participants (≥10 DLE, ≥10 SCLE, and ≥10 SLE) will be enrolled in this study. All participants will continue to be managed by their personal physicians per their standard of care. The data obtained in this study will help in the evaluation of new therapeutics for lupus and may facilitate lupus drug discovery. A blood sample for genomic analysis will be collected for research purposes from patients who provide consent and where local regulations permit. There will be a single sample collection time point for each patient. The safety assessments will include laboratory measurements (serum chemistry and hematology), monitoring of adverse events (AEs) related to study procedures, and physical examination.
The purpose of the study is to compare efficacy and safety of minoxidil 1% versus placebo in enhancement of eyebrows.
The purpose of this study is to see if the investigators can prevent or reduce the severity of the Cetuximab-related acne rash. Two different topical agents will be applied to the skin. One topical agent is the dapsone gel and the other is a skin moisturizer. Dapsone gel is an FDA approved medication that you apply to the face. It is commonly used to treat acne. Skin moisturizers are recommended to patients who receive Cetuximab treatment. In addition to these topical agents they will be given a pill to take once a day. This pill has already been shown to help fight rashes from Cetuximab.
This trial seeks to describe the effect of withdrawal from mycophenolate mofetil (MMF) on risk of clinically significant disease reactivation in quiescent SLE patients who have been on long-term MMF therapy. Participants who have had inactive disease for at least 24 weeks will be enrolled. Half the subjects will continue on MMF and half the subjects will be tapered off their MMF within 12 weeks. All subjects will continue hydroxychloroquine and small doses of prednisone as needed. Subject visits to assess endpoints will occur every 4 weeks from Day 0 through Week 24 and then at Weeks 32, 40, 48, and 60.
Alopecia areata (AA) is a common disease of the immune system, known as an "autoimmune" disease. In the disease, the immune system mistakenly destroys the hair follicle, causing hair to fall out. Despite many people having this disease, research into its cause and into new, better ways to treat AA has lagged far behind other similar diseases of the immune system. Currently, there are no Federal Drug Administration approved drugs for AA. Ruxolitinib (made by Incyte) is an intervention known to effectively treat a disease of the bone marrow, known as myelofibrosis. It is also being studied in the treatment of rheumatoid arthritis, another "autoimmune" disease, by fighting inflammation. There are some genetic and chemical similarities between those with myelofibrosis, active rheumatoid arthritis and AA, suggesting that treatment with ruxolitinib may be effective in AA. In mice specially designed for testing drugs for the treatment of human alopecia areata, this medication worked to prevent the disease AA from starting in mice that would have otherwise developed the disease. To test Ruxolitinib, we are going to treat 12 patients with moderate to severe AA for a minimum of 3 months up to 6 months. This is an "open-label" study, meaning that there will not be a placebo group; all patients enrolled in the study will receive the active medication.
Systemic Lupus Erythematosus (SLE) is a chronic autoimmune disease for which the aetiology includes genet-ic and environmental factors. It is rare in children as compared to adults. The severity may be related to greater involvement of genetic factors in children. The impact of genetics in the development of SLE is important, and the risk of recurrence in siblings evaluated by lambda S ratio is 30 in SLE, while it is 15 for type-1 diabetes and 8 rheumatoid arthritis, thereby indicating high impact of genetics in SLE. Recently, the group of Professor Yanick Crow in Manchester and other teams has identified new forms of lupus Mendelian genetics. The TREX1 and genes involved in the SAMHD1 frostbite lupus. Nearly 2 % of all adult subjects with SLE have a heterozygous mutation in the TREX1 gene, which therefore represents the first genetic cause of SLE. The team of Professor Crow also identified the ACP5 gene that is responsible for SLE associated with Spondylo-epiphyseal enchondro-epiphyseal dysplasia (syndromic lupus). Other groups have identified mutations in two genes encoding a DNAse (DNAse1 and DNAse1L3) responsible for familial monogenic forms of SLE. These new genes SLE were identified through research of germ-line mutations in cases of lupus syndromic or family. In collaboration with Professor Crow, we are currently undergoing characterization of a novel gene of SLE in a family and we have identified a second locus identified in another family.
This is an open label, pilot, observational, prospective study of the safety of inactivated influenza vaccine (IIV) in children with systemic lupus erythematosus (SLE) to be conducted during the 2013-2014 influenza season. The study will test conventional and novel biomarkers to assess disease flare and vaccine response and will also collect self-reported signs/symptoms in reactogenicity diaries during the 14 days after vaccination. This is an open label, pilot, observational, prospective study of the safety of inactivated influenza vaccine (IIV) in children with systemic lupus erythematosus (SLE) to be conducted during the 2013-2014 influenza season. Annual receipt of IIV is recommended for persons with SLE and is considered a standard of care medical practice. The study will test conventional and novel biomarkers to assess disease flare and vaccine response and will also collect self-reported signs/symptoms in reactogenicity diaries during the 14 days after vaccination. Little is known about the immune responses to influenza and other vaccines in children and adolescents with autoimmune conditions, both in terms of immunogenicity as well as the potential for triggering or worsening of immune/autoimmune pathways. Patients with SLE often must take two or more immunosuppressive medications to control their illness. Thus, it is critically important to study vaccine safety and immunogenicity within the pediatric SLE population rather than extrapolate the limited data available from adult clinical studies. Newly diagnosed, untreated patients will be too sick to include in this study.
The purpose of this study is to obtain skin, blood, and urine samples from patients with active cutaneous lupus lesions and from healthy participants. This is a Phase 0 multi-center and longitudinal study of biomarkers and clinical parameters in patients with lupus erythematosus (LE). Approximately 80 participants (20 healthy participants and 60 patients with LE) will be enrolled. This will include 30 with Discoid Lupus Erythematosus/Subacute Cutaneous Lupus Erythematosus (DLE/SCLE) without Systemic Lupus Erythematosus (SLE), and 30 with DLE/SCLE with SLE. All patients with LE will continue to be managed by their personal physicians per their standard-of-care. Study participants will undergo interventional procedures that include collection of urine, blood samples and skin biopsy. There will be a single sample collection timepoint for healthy participants and two sample collection timepoints for LE patients, separated by approximately 12 weeks. A blood sample for genomic analysis will be collected for research purposes from patients who provide consent and where local regulations permit. The data obtained in this study will be used in the evaluation of new therapies for lupus and may help in developing new treatments. Safety evaluations will include assessment of adverse events, clinical laboratory tests, vital signs, and physical examinations. The total duration of study participation will be approximately 11 days for healthy participants and 95 days for patients with LE.
The purpose of this study is to evaluate the clinical efficacy of blisibimod as measured by a composite responder index in subjects who, despite corticosteroid use, continue to have autoantibody positive, clinically-active Systemic Lupus Erythematosus (SLE) as defined by SELENA SLEDAI score ≥10.
The main goal of the study is to determine if bone marrow transplant (BMT) from a less specific pool of donors in combination with high dose cyclophosphamide can induce remission of refractory systemic lupus erythematosus. Systemic lupus erythematosus (SLE) is a devastating systemic autoimmune disease that predominantly affects young women, is more common in African-Americans than in whites, and results in poor quality of life. Lupus has no cure, and up to 90% of patients require corticosteroids for disease control. More than half of patients with lupus have permanent organ damage, much of which is either directly due to or increased by corticosteroids. To satisfactorily manage moderate-to-severe SLE, we need effective treatments that will allow corticosteroid-sparing. High-dose chemotherapy followed by autologous BMT or peripheral blood progenitor transplantation (PBSCT) has been proposed as a novel approach to treat severe autoimmune diseases. Allogeneic BMT is not currently utilized for the routine treatment of SLE because of the significant morbidity (GVHD) and mortality associated with the procedure. We have recently developed an approach to BMT using post-transplant cyclophosphamide that allows us to safely perform allogeneic BMT from matched, mismatched, unrelated or haploidentical donors. Transplant-related mortality, graft-failure and risk of GVHD have been very low with this approach. Furthermore, this approach allows us to greatly expand the donor pool since any patient shares exactly one HLA haplotype with each biological parent or child and half of siblings, an eligible haploidentical donor can be identified rapidly in nearly all cases.
This study will assess the effect of a 24-week withdrawal followed by a 28-week reintroduction of belimumab 10 mg/kg plus standard of care medications in subjects with stable low systemic lupus erythematosus (SLE) disease activity. Rebound phenomenon will be assessed for subjects who have permanently withdrawn from further belimumab treatment. This study will assess the effect of a 24-week withdrawal of belimumab followed by a 28-week reintroduction of belimumab 10 mg/kg plus standard of care medications on immunogenicity, markers of biological activity, efficacy, and safety in subjects with stable low systemic lupus erythematosus (SLE) disease activity. Additionally, this study will assess rebound phenomenon in subjects with any disease level of SLE who have permanently withdrawn from further belimumab treatment
Cutaneous lupus is a chronic, relapsing, auto-immune skin disease that can have many presentations. Its effect on physical appearance greatly affects patients' quality of life. In addition, 10% of patients with cutaneous lupus will develop systemic lupus. Topical therapies are the mainstay of cutaneous lupus treatment; however patients often find these treatments to be messy, inconvenient, or ineffective. In addition, for more severe disease patients are often placed on concurrent systemic therapies. The primary hypothesis of our study is that poor adherence contributes to poor treatment outcomes in patients with cutaneous lupus. Cutaneous lupus is a chronic, relapsing auto-immune disease that negatively impacts patients' quality of life. As there are many safe topical therapies that require daily use to be effective, and adherence to medication remains a common problem in dermatology, it is worthwhile to reliably measure adherence to medication in patients with cutaneous lupus. By elucidating barriers to effective control of cutaneous lupus with topical medications, we can improve patient care and guide interventions that will avoid the use of systemic therapy which often has more side effects. The primary aim is to determine adherence to topical and systemic lupus treatment. Subjects will receive standard of care therapy (low potency and high potency topical corticosteroids) and systemic therapy when clinically appropriate. Adherence to medications will be monitored electronically in all subjects. The following hypothesis is to be tested: Non-adherence to topical and systemic medication contributes to treatment failure in patients with cutaneous lupus.
The purpose of this study is to evaluate the safety, tolerability and efficacy of FDX104 Antibiotic Foam in the prevention of EGFRI skin toxicity in cancer patients receiving Cetuximab or Panitumumab. This is a phase II multicenter, randomized, double blind, vehicle controlled clinical study to evaluate the safety, tolerability and efficacy of Doxycycline Foam 4% for the prevention of Epidermal Growth Factor Receptor Inhibition Skin Toxicity, to Subjects with Cancer Receiving Cetuximab or Panitumumab on a weekly or every 2 weeks basis. The study consists of a screening visit, a treatment period where patients will be treated topically on the face twice daily for 5 weeks. A post-treatment follow up visit (4 weeks after end of treatment), will be performed only for subjects who have experienced unresolved possibly-related or related adverse events at the end of the treatment. Seven days after randomization and study drug initiation, subjects will start their EGFRI treatment.
Lupus nephritis is a severe form of systemic lupus erythematosus (SLE) with active disease in the kidneys. SLE is a complex disease in which the body's own immune system attacks some of the body parts: the skin, the joints, the kidneys, the nervous system, the heart, the lungs and the blood. The cause of SLE is not known. Treatment for SLE usually involves drugs that are designed to block the immune system attacks. When SLE affects the kidneys (nephritis), stronger immune suppressing treatment is usually needed. The drugs used in treatment of lupus nephritis often do not cure the disease and can cause serious side effects, including lowering the immune system too much. When the immune system is too low, a person is at a higher risk of getting infections. Therefore, research into new treatments with fewer serious side effects is needed for lupus nephritis. In this experimental study, researchers will try to find out if treatment of lupus nephritis with a combination of rituximab and cyclophosphamide (CTX), or a combination of rituximab and CTX followed by treatment with belimumab is safe and if this drug combination can block the immune system attacks.
The present study investigates the potential of a new therapeutic approach in lupus nephritis combining rituximab (anti-CD20) and belimumab (anti-BAFF). The main goal of the study is to assess the reduction (and seroconversion) of pathogenic autoantibodies, to evaluate clinical improvement and assess the safety and feasibility of long-term B-cell depletion. Introduction Systemic lupus erythematosus (SLE) affects predominantly young women with childbearing potential (20-40 years) and inflammation can occur in virtually every organ, including kidneys, lungs, heart or brain. The disease course in SLE patients is typically characterized by frequent flares, requiring immunosuppressive treatment. Current, evidence-based treatment modalities for SLE consist of immunosuppressive treatment with high dose corticosteroids, cyclophosphamide or mycophenolic mycophenolate acid, that non-specifically target the immune system to reduce inflammation. Side-effects of these treatment strategies are (opportunistic) infections in the short term and risk for malignancy and cardiovascular disease in the long-term. Treating SLE patients with biologicals is an attractive alternative because biologicals specifically target the immune system by blocking cytokines or deplete one specific cell population, thereby reducing the risk for infections or malignancies as compared to conventional immunosuppressants. Furthermore, the scarce treatment options underscore the need to exploit new therapeutic possibilities for SLE patients who frequently experience a flare of the disease. These considerations led to the present study involving a proof-of-concept study in refractory SLE patients to assess the immunological consequences of a combination treatment with rituximab (anti-CD20) and belimumab (anti-BAFF).
Treating severe thrombocytopenia is a challenge in the management of systemic lupus erythematosus. Although rheumatologists have followed some rules in real practice,there is very few evidence to support the current treatment algorithm. The purpose of this study is to compare the complete remission rate and partial remission rate of cyclophosphamide and hydroxychloroquine for treating severe thrombocytopenia in Chinese SLE patients. This is a prospective,randomized,open-label,multi-center clinical trial. The aim of this study is to test the efficacy of GC(gluco-corticosteroid)+HCQ(hydroxychloroquine) and GC(glucocorticosteroid)+CTX(cyclophosphamide) with sequential AZA(azathioprine) in the induction and maintenance therapy of severe thrombocytopenia in SLE patients.
Given that GADD34 has been described as a potential key regulator of pro-inflammatory cytokine production in human and elevated blood marker in SLE patients, this study aim to prove that the GADD34 RNA level in mononuclear blood cells can be used as a prognostic marker to assess the risk of SLE flare. Systemic lupus erythematosus (SLE) has a prevalence estimated between 1 and 6 per 10000 individuals and concerns over 30 000 patients in France for example. As of today, there isn't any reliable specific blood analysis that can be used to assess the prognosis of SLE, that means predict a SLE flare. The discovery of such a blood test could allow a better patients' monitoring in order to help doctors to better adapt treatments (lowering background treatments when they are not needed and increase them ahead of a potential flare when the risk assessment raises). In SLE patients, a recent study shows elevated levels of GADD34 RNA in mononuclear blood cells : higher than twice the control levels for 36 of the 60 SLE patients and ten times higher than the control levels for 13 of the 60 SLE patients. Given that GADD34 has been described as a potential key regulator of pro-inflammatory cytokine production in human and elevated blood marker in SLE patients, this study aim to prove that the GADD34 RNA level in mononuclear blood cells can be used as a prognostic marker to assess the risk of SLE flare.
To study the 5-year immunogenicity against a quadrivalent HPV vaccine in patients with SLE and healthy controls. Most cases of human papilloma virus (HPV) infection are asymptomatic, transient and resolve without treatment. However, in some individuals, especially those patients who are immunocompromised because of various underlying diseases and those who are receiving long-term immunosuppressive agents, HPV infection is persistent and may result in genital warts, cervical smear abnormalities, cervical intraepithelial neoplasia (CIN) and rarely cervical cancer. High-risk HPV serotypes are those which are oncogenic and associated with invasive cancers of the cervix and vulva. Examples are the HPV 16 and HPV 18. HPV 16 accounts for almost half of all cervical cancers whereas HPV 18 infection is present in 10-12% of all cervical cancers. Systemic lupus erythematosus (SLE) patients are at risk of persistent HPV infection. This is because of the immune aberration related to the disease itself and the immunosuppressive state induced by various treatments. One study of 85 SLE patients revealed a 16.5% prevalence of abnormal Pap smears and 12% prevalence of cervical squamous intraepithelial neoplasia (CIN) on routine screening, which was significantly higher than those figures reported in age-matched healthy women (corresponding figures for abnormal Pap smear and CIN were 5.7% and 2%, respectively). Thus, prevention of HPV infection is important in patients with SLE in order to reduce the incidence of CIN lesions and hence invasive cervical cancers in the long run.
The goal of this new lupus research study is two-fold: first, to understand the genetic associations found between people's DNA and this disease, and second, to apply this understanding to drug development efforts with the investigator's partners at Pfizer. Lupus is a difficult disease to diagnose and one that's difficult to manage, given the lack of specific medicines to treat lupus and the side-effects of current medications. The investigators believe genetics may play a critical role in developing better lupus diagnoses and treatments. If individuals are eligible to participate, the investigators will send a 23andMe DNA saliva kit at no cost. Participants will be asked to take a 15 minute online survey with questions about their lupus experience, symptoms and response to treatments, in addition to five additional bi-monthly short surveys and one final study survey during the 12-month participation. Learn more and join the lupus study today: https://www.23andme.com/lupus/
The purpose of this study is to evaluate the safety and efficacy of repeated intravenous infusions of the study drug BT063 in patients with Systemic Lupus Erythematosus (SLE) compared with people who receive a placebo. Study 990 is a Phase IIa, proof-of-concept study of BT063 in subjects with SLE. This study is divided into 2 parts. After Part I an interim analysis will be performed. Each Part will enrol 18 subjects. Subjects will be randomly assigned to receive BT063 or Placebo 8 times over 12 weeks and will be followed for 4 months after their last dose.
Systemic Lupus (SLE) is a chronic disease for which long term treatments are warranted. The aim of this study was to study the possibility of corticosteroids interruption in patients with quiescent SLE treated since at least one year with 5 milligrams of predonisone per day.
Platelet rich plasma (PRP) therapy is a novel therapeutic modality that has seen broad applications for a number of medical indications including those in orthopedics, dentistry, and dermatology. In dermatology, its uses have included treatment of chronic wounds and facial rejuvenation. More recently, anecdotal reports have suggested some efficacy in the treatment of hair loss, but to the best of our knowledge, there has been only one published case series documenting its use for this indication. This study was designed to better assess the efficacy of PRP in hair loss, we therefore propose to study interval intralesional PRP injections for patients with androgenetic alopecia (AGA). Androgenetic alopecia is the most common form of hair loss world-wide. Although there are currently numerous treatment options for this indication including minoxidil, 5-alpha reductase inhibitors and follicular unit transplant, many of the medical treatment options have undesirable side effects, particularly in women of child bearing age. These well-documented adverse reactions include impotency, hypertrichosis, and birth defects.
The purpose of this study is to evaluate the efficacy and safety of mesenchymal stem cells (MSCs) obtained from umbilical cords for the treatment of adults with systemic lupus erythematosus (SLE). The goal of this study is to determine if patients receiving an MSC infusion plus standard of care respond better than patients receiving placebo infusion plus standard of care. Systemic lupus erythematosus (SLE) is a devastating illness striking primarily young women in their reproductive years. Treatments for active SLE remain inadequate with significant adverse effects and response rates for even the most aggressive therapies are capped at 50%. Thus there is a significant need for more effective and safer therapeutic options. Mesenchymal stem cells (MSCs) are stromal cells known to possess significant immunosuppressive properties. Evidence from animal models and uncontrolled trials in patients with SLE supports the effectiveness of allogeneic MSC infusions in reducing SLE disease activity, with remarkably few adverse effects. MSCs are relatively immune-privileged in that they do not express Class II antigens or surface activating markers such as CD80/86, and therefore can be given without cross-matching or any need for bone marrow ablation/conditioning of the recipient. To date there are no reported or ongoing controlled trials of MSC therapy compared to standard of care in SLE. This Phase II multicenter placebo controlled trial will evaluate the efficacy and safety of allogeneic MSCs for the treatment of adults with moderate to severely active SLE.
The purpose of this study is to determine whether genetic factors contribute to an individuals risk of developing obstructive lung disease from smoking cigarettes. Chronic obstructive pulmonary disease (COPD) is among the most important chronic diseases of adults. It is the fourth leading cause of death in the U.S. and affects more than 10% of the U.S. population over age 55. COPD accounts for more than 25,000 discharges annually from VA medical centers. Cigarette smoking is the most important known modifiable cause of COPD, yet only 15-20% of cigarette smokers develop clinically significant COPD. We therefore hypothesize that genetic mechanisms determine susceptibility to the development of COPD. The only genetic abnormality known to cause COPD is deficiency of the serine protease inhibitor 1-protease inhibitor, which causes premature development of emphysema, although it is implicated in fewer than 2% of COPD cases. Several other genetic variants have been proposed as candidate causes of COPD; however, these have been identified on the basis of association studies in unrelated subjects, which have considerable risk of ascertainment bias. In the present study, a sub-pair linkage approach will be utilized in COPD patients and their smoking siblings to identify genes which determine the risk of developing COPD. The following are the short-term objectives of this study: 1. Recruit a cohort of 400 probands with smoking-related COPD and their smoking siblings, whether or not affected by COPD.
To determine if linezolid is superior to vancomycin in the treatment of nosocomial (acquired in the hospital) pneumonia due to Methicillin Resistant Staphylococcus Aureus (MRSA) in adult subjects. Subjects entered in to the study will have proven healthcare-associated methicillin-resistant Staphylococcus aureus pneumonia which will be treated with either linezolid or vancomycin.
The purpose of this trial is to study the effects of roflumilast on lung function parameters indicative of hyperinflation in patients with COPD.
The purpose of this study is to assess whether children with moderate to severe bronchiolitis treated with standard racemic epinephrine therapy via 70:30 helium-oxygen (heliox) driven nebulization will have improvements in measurements of airway more rapidly than those treated with conventional air-oxygen driven nebulization.
The role of antibiotic therapy in patients with COPD remains controversial. While the outcome of several clinical trials is in favour of antibiotics, the quality of these studies in insufficient. In this study the efficacy of doxycycline is compared to placebo. All concommitant treatment (steroids, bronchodilator therapy, physiotherapy) is standardized. The investigators hypothesize that patients with an acute exacerbations will have a better outcome when treated with antibiotics.
The purpose of this study is to determine whether macrolide therapy is effective in treating patients hospitalized with asthma exacerbations or chronic obstructive pulmonary disease (COPD)exacerbations. We hypothesize that compared to placebo, maintenance therapy with macrolides, when added to usual care, a) improves respiratory symptoms, b) improves quality of life, c) reduces airway inflammation, d) reduces airflow obstruction, and e) decreases the rate of re-exacerbations. Asthma and chronic obstructive pulmonary disease (COPD) are the two most common obstructive lung diseases. Chronic airway inflammation and episodic worsening of respiratory symptoms and airflow obstruction (exacerbations) occur in asthma and COPD. Despite major advances in the development of therapies for these respiratory disorders, asthma and COPD exacerbations are common and result in substantial morbidity and mortality. Moreover, patients hospitalized for asthma or COPD exacerbations are at high risk for near fatal and fatal re-exacerbations after discharge home. These observations highlight the need for novel therapies to prevent asthma and COPD exacerbations. The role of macrolide antibiotics (e.g., azithromycin, clarithromycin, erythromycin) in treating bacterial infections is well established. Recent pre-clinical evidence also suggests that macrolides may posses distinct anti-inflammatory properties and even anti-viral properties. These exciting observations have led to research evaluating the use of maintenance therapy with macrolides in patients with asthma and COPD. Small studies in clinically stable asthma or COPD suggest that maintenance macrolide therapy (e.g., use for 6 weeks), when added to usual care, may attenuate airway inflammation, reduce respiratory symptoms, and improve lung function.
The purpose of this study is to determine whether inpatient use of a high frequency chest wall oscillator is an effective therapy for patients hospitalized with asthma exacerbations or chronic obstructive pulmonary disease (COPD) exacerbations. The investigators hypothesize that inpatient use of HFCWO will promote clearance of airway secretions, leading to more rapid resolution of severe asthma and COPD exacerbations and lower rates of re-exacerbations after hospital discharge. Asthma and Chronic Obstructive Pulmonary Disease (COPD) are common chronic pulmonary disorders with periodic exacerbations characterized by increased airway obstruction, mucous hypersecretion, and respiratory symptoms such as dyspnea, cough, and wheeze. Despite major advances in the treatment of these disorders, severe asthma and COPD exacerbations are common and each result in about 500,000 hospitalizations per year in the United States. Moreover, re-exacerbations are common in this population, occurring in about 25% within 1 month and 50% within 12 months of hospital discharge. Inhaled medications, which rely on deposition into distal airspaces to suppress airway inflammation and promote bronchodilation, are used for both the treatment and prevention of asthma and COPD exacerbations. However, excessive mucous production and impaired airway mucociliary clearance lead to airway plugging, reducing the deposition of and response to inhaled medications. These considerations highlight the need for therapies that clear airways of mucus in patients with asthma and COPD. High frequency chest wall oscillation (HFCWO) may be a novel strategy for mechanical airway clearance to improve outcomes in patients with asthma/COPD exacerbations.
Intensive Care Unit (ICU) admission for acute exacerbation of chronic obstructive lung disease (COLD) is a major cause of morbidity and mortality in such patients. Although bacterial of mortality in such patients. Although bacterial and or viral infections are considered as the major precipitating factor, the antibiotic strategy in this setting is unclear. The absence of overt infection remains controversial, and has not been adequately studied in patients admitted to the ICU. To assess the benefit ( or lack thereof ) of routine early systemic antibiotic therapy in patients with COLD admitted to the ICU. The primary objective of the essay is to evaluate the effectiveness of the precocious antibiotic therapy on the length of the respiratory symptoms with the admitted patients in polyvalent medical intensive care of chronic obstructive lung disease ( COLD ) This is a multicenter, randomised, double-blind controlled trial, comparing amoxicillin-clavulanic acid administered for 7 days to a placebo.
Research on the synergistic effect of corticosteroids and beta-agonists on the cytokine production in COPD patients. Blood will be obtained from healthy volunteers and COPD patients and blood monocytes will be stimulated to produce cytokines with or without the prementioned drugs.
This study was designed to evaluate the hypothesis that nebulized budesonide) might be an alternative to systemic corticosteroids (SC) in the treatment of patients with acute exacerbations of COPD (AECOPD). Patients, hospitalized with AECOPD, were randomized into three groups. Group I received only bronchodilator treatment (BDT), Group II received SC (40 mg prednisolon) plus BDT, and Group III received nebulized budesonide (NB)(1500 mcg qid) plus BDT. Improvement rates in multiple parameters during 10-day hospitalization, and acute exacerbation and re-hospitalization rates within one-month after discharge were compared between the groups.