cyrilzakka/clinical-trials huggingface dataset:
A Pilot Study of Pyridostigmine in Pompe Disease

Pyridostigmine is an acetylcholinesterase inhibitor, which degrades acetylcholine at the neuromuscular junction. Based on recent studies, pyridostigmine may be an effective adjuvant treatment for people with Pompe disease, as it increases the functional impact of this neurotransmitter.

Hypothesis: the use of pyridostigmine in Pompe disease will improve transmission of acetylcholine across the neuromuscular junction, skeletal muscle function, respiratory function, and quality of life.

Pompe is a rare disease, which occurs in approximately 1 per 40,000 births. It is a progressive and often fatal neuromuscular disorder resulting from mutation in the gene for acid alpha-glucosidase (GAA), an enzyme necessary to degrade glycogen. Accumulation of glycogen in multiple tissues results in cardiac, respiratory and skeletal muscle dysfunction. Enzyme replacement therapy (ERT) is currently the only treatment available, and although it prolongs survival, adjuvant therapies are needed to help alleviate the dire symptoms of Pompe disease.

Recent data has revealed that degradation of the neuromuscular junction (NMJ) occurs in Pompe disease. Acetylcholinesterase inhibitors (AChEI) are substances that inhibit the AChE enzyme from degrading acetylcholine at the NMJ, and thus increase the functional impact of this neurotransmitter. AChEI are established as a beneficial therapy for individuals with primary diseases of the NMJ, such as myasthenia gravis. Recently, administration of an AChEI was demonstrated to improve NMJ pathology in both mice and individuals affected by other congenital myopathies, including autosomal centronuclear myopathies (CNM), X-linked myotubular myopathy (XLMTM) and mutation of tropomyosin 3 (TPM3). Specifically, both NMJ transmission and motor function were improved. These studies demonstrate that AChEI can be beneficial in myopathy associated with NMJ pathology.

In this study, we will study the acute effects of pyridostigmine on neuromuscular transmission, as well as the prolonged effects on respiratory function, skeletal muscle function and quality of life over a 90 day treatment period.

This project focuses on developing an adjuvant treatment to ERT that targets dysfunction at the NMJ. Our ultimate goal is to reduce the deleterious consequences of Pompe disease and improve the overall quality and duration of life in affected individuals.

Eligibility Criteria:
Inclusion Criteria:

1. Males or females between 8 and 60 years of age;
2. Diagnosis of Pompe disease (protein assay, genotyping, and positive clinical signs)
3. No contraindication to pyridostigmine

Exclusion Criteria:

1. Already receive pyridostigmine as part of their normal clinical care at screening
2. Are pregnant - participants will receive a urine pregnancy test at screening
3. Have received acute administration of antibiotic, corticosteroid, or neuromuscular blockade medications within 30 days prior to screening
4. Any other concurrent medical condition which, in the opinion of the study team, would make the subject inappropriate to participate in the assessments
SOX as Salvage Treatment in Nasopharyngeal Carcinoma

The aim of the study is to evaluate the efficacy and safety of SOX regimen (S-1 plus oxaliplatin) as salvage treatment in patients with relapsed or metastatic nasopharyngeal carcinoma.

The treatment option in patients with platinum-resistant relapsed or metastatic nasopharyngeal carcinoma is limited. Previous studies showed that both S-1 and oxaliplatin were effective in head and neck cancer. Moreover, SOX (S-1 plus oxaliplatin) was proved to be tolerable in advanced stage gastric cancer. Therefore, we aim to evaluate this regimen in salvage setting of nasopharyngeal carcinoma.

Eligibility Criteria:
Inclusion Criteria:

* Age range: 18-75 years old
* Histological confirmed incurable relapsed or metastatic nasopharyngeal carcinoma
* Prior exposure of at least one line of platinum-containing regimen
* ECOG performance status 0-1
* Life expectancy of more than 3 months
* Bone marrow function: ANC≧1.5×109/L, PLT≧100×109/L, Hb≧80g/L
* Liver function: total bilirubin, ALT and AST \<1.5×UNL
* Renal function: Cr\<1.5×UNL, CCR≧50ml/min
* Without \> 1 grade of neuropathy

Exclusion Criteria:

* With curable treatment option
* With CNS involvement
* Prior platinum exposure only in neo-adjuvant/adjuvant setting or concurrently used with radiotherapy
* Treated with \> 2 lines of palliative chemotherapy
* With prior exposure of S-1 or oxaliplatin
* History of other malignancies except cured basal cell carcinoma of skin and carcinoma in-situ of uterine cervix
* Significant active infection
* Pregnant or lactating women
Technology and Human Help at Home After Hospitalisation

Elderly people are still at risk for falling at home. The objective of the study is to evaluate at home the impact of an intervention for 3 months combining technologies for autonomy and tailored physical activity program. The study is performed among patients aged 75 years and over leaving Charles Foix Geriatrics hospital or already supported by carer of professional home-care service Ages \& Vie and with a loss of autonomy requiring physiotherapy and home support workers. It is a prospective, randomized, single-center, in cluster study which compares a Patient group (interventional group) with the intervention program (technologies + physical activities) with a control group benefiting from usual care home. Our hypothesis is that in the interventional group fewer falls were observed and improved different scores (Timed Up and Go Test,Barthel index, Quality of Life questionnaire of Duke) were reported compared to the control group.


Eligibility Criteria:
Inclusion Criteria:

* patients hospitalized in Charles Foix Hospital (France) and returning home or already supported by carer of professional home-care service Ages \& Vie
* \> 75 years old with loss of autonomy
* at risk for falling
* able to read and understand the information letter and consent letter
* patient requiring physiotherapy and caregiver at home.
Radio-frequency Ablation in Knee Osteoarthritis by Three Needles Technique

The study is designed to evaluate and compare the efficacy of three needles approach as a new technique in radiofrequency neurotomy of genicular nerve versus the traditional single needle approach as a treatment of advanced knee osteoarthritic pain. Primary outcome:

1. Pain intensity via the visual analogue score (VAS).
2. Function and pain via the Oxford Knee Score. Evaluated after 6 months of the procedure

Secondary outcome:

1. Failure rate.
2. Incidence of any complication.
3. Total analgesic requirement during the period of follow up.


Eligibility Criteria:
Inclusion Criteria:

* 1- Age 35-85 years old 2- Patients with stage 3 or higher OA-related radiological changes based on Kellgren-Lawrence (K/L) rating scale 3- Knee pain for at least 3 months 4-Patients are clinically unresponsive to conservative treatment modalities (physical therapy and rehabilitation practices, orally administered analgesics and anti-inflammatory drugs).

Exclusion Criteria:

* 1- Patients with other causes of pain as radiculopathy, neurological disorders or intermittent claudication. 2- Previous knee trauma. 3- Previous knee surgery. 4- Patients received intra-articular injection during the previous three months.

  5- Acute knee pain. 6- Crystal arthropathy. 7- Joint effusion. 8- Serious neurological or psychiatric disorders. 9- On anticoagulant medication. 10- Any other connective tissue disorder that affects the knee joint. 11- skin infection at the site of injection. 12-patient refusal.
CLARITY Study - PMCF Study

In the context of the Post-Market Clinical Follow-up (PMCF), this study aimed at evaluating the safety and performance of the RADSAFE® inks in the marking of the radiation field.


Eligibility Criteria:
Inclusion Criteria:

Male or female ≥18 years old, Cancer patient requiring radiotherapy, Patient requiring medical tattoo for treatment alignment, Intact skin at site of standard radiotherapy tattoos, ECOG performance status ≤2 (Karnofsky ≥60%), Able to provide written informed consent, Affiliated patient or beneficiary of a social security scheme.

Exclusion Criteria:

Previous medical tattoo of the targeted zone of treatment Known allergy to pigment ingredients, Infected or diseased skin, past or present, Affected organs close to the skin (eyes), Hypertrophic scars at site of radiotherapy tattoos, Carrying cardiac implant, Patient with blood clotting disorders, No consent to study participation, Under guardianship, curatorship or other legal protection, deprived of liberty by judicial or administrative decision, An existing medical condition that, in the opinion of the Investigator, may put the subject at risk or compromise their participation in the study, Psychiatric illness/social situations that would limit compliance with study requirements, Females who are pregnant, lactating, or unwilling to use adequate birth control for the duration of the study, Personal objection to medical tattooing, Participation to another drug or medical device clinical interventional study that may interfere with the clinical investigation objectives.
The Effect of Spironolactone on Blood Pressure in Type-2 Diabetics With Resistant Hypertension

The purpose of this study is to estimate the effect of spironolactone on blood pressure resistant to therapy in type-2 diabetics.

The primary object of the study is to estimate the effect of addition of low dose spironolactone to antihypertensive treatment with at least three antihypertensive drugs in patients with type-2 diabetes and blood pressure over 130/80 mmHg.

Secondary aims are to estimate how many of these patients have their blood pressure controlled by the addition of spironolactone, to investigate whether the addition of spironolactone affects insulin sensitivity and urinary protein secretion and to estimate the incidence of adverse effects of the aldosterone antagonist.

Eligibility Criteria:
Inclusion Criteria:

* Age \< 75 years
* Type-2 diabetes
* Therapy resistant hypertension (by ABPM)
* Treatment with at least 3 antihypertensives

Exclusion Criteria:

* HbA1c \> 10.0
* BP \> 180/110 mmHg
* Secondary hypertension
* Intolerance to spironolactone
* Permanent treatment with nonsteroidal antiinflammatory drugs or systemic glucocorticoids
* Total cholesterol 10 mmol/l
* New York Heart Association class III and IV
* Pregnancy or planned pregnancy
* Psychiatric disease
* Malignant disease
* Insufficient adherence
Incidence and Factors Associated With the Development and Severity of Oral Mucositis

Our study intends to evaluate prospectively the incidence and severity of oral mucositis in patients undergoing high-dose chemotherapy and hematopoietic stem cell transplantation, as well as to evaluate the potential influence of nutritional-, physical-, and transplant-related factors associated with the development of this complication and its grade of involvement.

Objective: Our study intends to evaluate prospectively the incidence and severity of oral mucositis in patients undergoing high-dose chemotherapy and hematopoietic stem cell transplantation, as well as to evaluate the potential influence of nutritional-, physical-, and transplant-related factors associated with the development of this complication and its grade of involvement.

Research plan: This is a prospective cohort study with an observational basis that involves all of the patients undergoing high-dose chemotherapy and peripheral blood stem cell transplantation at the Audie L. Murphy VA Hospital Bone Marrow Transplant Unit and at the University Hospital Bone Marrow Transplant Unit. We expect to enroll 400 patients during a 5-year period.

Methods: In order to answer the study questions, subjects will be assessed several times during the study period. The study involves a multidisciplinary group that will be evaluating and following the subjects. A baseline will be established by a physician and a nutritionist before the subjects start the high-dose chemotherapy regimen. The subjects will be followed with oral evaluations 3 times per week, beginning on the day of the transplant and ending when mucositis resolves, or the patient is discharged home, or 30 days post-transplant, whichever occurs first. At day 15 post-transplant, patients will be evaluated for the second time by the nutritionist. Further information such as blood values, use of medications and treatment complications, will be obtained by daily chart review.

Clinical relevance: Mucositis is, without a doubt, one of the most common and debilitating complications resulting from the intense cytotoxic therapies preceding hematopoietic stem cell transplantation. Mucositis is not only a problem for the patient who needs to deal with the pain and the rapidly decreasing quality of life, but it is also a problem for the health system. Mucositis frequently results in increased hospital length of stay, resulting in a significant increase in the cost of medical care. Multiple prophylactic and therapeutic attempts have been made in order to control the symptoms and the severity of mucositis. No prevention or cure is available for the disease. Despite the renewed interest in this complication, important gaps in knowledge remain in this area of the supportive care of cancer patients. These gaps in knowledge are wider in the area of mucositis in patients who undergo hematopoietic stem cell transplantation.

Eligibility Criteria:
Inclusion Criteria:

* Patients undergoing hematopoietic stem cell transplantation
* Age 18 years and older
* Patients must understand and sign the study informed consent

Exclusion Criteria:

* None
Peripheral Arteriopathy Rate In Stroke

Purpose:

To assess the prevalence of peripheral arterial disease (PAD) in acute stroke patients.

Interventions:

Assessment of the ankle brachial index using the "boso-ABI-system 100" (www.boso.de).

Design:

Prospective, open, cohort study.

Study size planned:

1500.

Follow-up duration:

3 months.


Eligibility Criteria:
Inclusion Criteria:

* All consecutive adult patients with acute ischemic or hemorrhagic stroke.

Exclusion Criteria:

* Congestive heart failure.
Mobile CARE-App to Promote Coping for Caregivers of Patients Receiving Stem Cell Transplant

The goal of this research study is to determine whether a self-administered, psychosocial mobile application (CARE app) is effective at improving the quality of life and experience of caregivers of patients receiving hematopoietic stem cell transplantation (HCT).

This is a randomized clinical trial to determine whether a psychosocial mobile application (CARE app) is effective at improving the quality of life and experience of caregivers of patients receiving hematopoietic stem cell transplantation (HCT). The CARE app was developed with the goal of addressing the needs of caregivers of HCT recipients.

Participants will be randomized into one of two study groups: CARE app plus Usual Care versus Usual Care.

Participation in this study is expected to last up to 100 days after HCT.

It is expected that about 120 people will take part in this research study.

The Leukemia and Lymphoma Society is supporting this research by providing funding.

Eligibility Criteria:
Caregiver Inclusion Criteria:

* Adult caregivers (\>18 years) who is a relative or friend who live with the patient or is a designated caregiver as indicated during the transplant process.
* Caregiver of a patient receiving allogeneic or autologous HCT for the treatment of cancer
* Ability to comprehend and speak English as the CARE app is only available in English

Patient Exclusion Criteria:

* Caregivers of patients undergoing HCT for benign hematologic conditions
* Caregivers with acute or unstable psychiatric or cognitive conditions which the treating clinicians believes prohibits informed consent or compliance with study procedures
Barriers to Adherence to Recommended Follow-up in Women With a History of Gestational Diabetes

The purpose of this study is to find out whether women who have had gestational diabetes mellitus (GDM) will make healthier lifestyle choices, achieve weight goals, and complete postpartum care assessments after receiving two online classes on healthy nutrition and exercise classes at 6 weeks - 3 months and at 9 months postpartum.

For this pilot study, we propose to conduct a 3-grouped randomized controlled trial to evaluate whether a 1 ½ to 2 hour on-line nutrition and physical activity intervention +/- block testing of blood glucoses fasting and 2 hours after each meal for 4 days/month from starting at 3 months postpartum and concluding at 9 months postpartum will: 1) improve adherence to the receipt of diabetes screening at 12 months postpartum through the recommended 75 gram 2 hour oral glucose tolerance test (OGTT) or fasting blood glucose test, 2) lower glucose values on the 75 gram 2 hour oral glucose tolerance test or fasting blood glucose test and 3) improve attainment of weight loss targets among women with a recent history of GDM.

Patients will participate in an online nutrition and physical activity class from 6 weeks to 3 months and 9 months for women assigned to both groups 2 and 3. For those women assigned to group 3, they will also do block testing using an assigned blood glucose meter and test strips 4 times a day for 4 consecutive days each month, starting at 3 months and ending at 9 months. Women will have the option of submitting meter blood glucose data via e-mailed logs or downloading directly to the clinic using Diasend software. Once downloaded, the Diasend software will create a PDF document with all recorded blood glucose values.

For purposes of this study, we will obtain a Diasend account specific to this study, which will allow women to download blood glucose information with an anonymous unique identifier that can be accessed by the study's Principal Investigator and the Collaborative Institutional Training Initiative (CITI)-certified, International Review Board (IRB)-approved research assistant. Upon accessing the PDF document with the blood glucose meter information, both the PI and the Research Assistant can link blood glucose data with questionnaire and other health information collected in this study through the unique identifiers.

Eligibility Criteria:
Inclusion Criteria:

* Gestational Diabetes
* Age between 21 and 45 years
* Preconception BMI 19-40
* Seen for at least 2 visits in the Diabetes in Pregnancy Program during their pregnancy
* Singleton pregnancy

Exclusion Criteria:

* Preexisting diabetes or diabetes diagnosed at the 6 week 75 gram 2 hour OGTT
* BMI \>40
* Multiple gestation (i.e., twins, triplets, etc.)
CD22 Targeting CAR-T Therapy Against B Cell Hematological Malignancies

CD19 expression on B cell frequently lost after CD19-targeting CAR-T therapy. In present study, we construct a CD22-targeting chimeric antigen receptor to overcome this issue.

CD19 is an ideal target with great potential for treating B-cell-derived hematological malignancies. Although the complete remission rate is as high as 93% by using CD19-targeting CAR-T technology, approximately 60% patients will have recurrent disease. Among all the recurrent patients, two thirds is revealed to loss their CD19 expression on B cell surface. For overcoming this issue, we establish a new chimeric antigen receptor containing humanized single chain antibody sequence to target CD22 molecule on B cells.

Eligibility Criteria:
Inclusion Criteria:

* Greater than four years of age
* Survival time\>12 weeks
* B cell hematological malignancies by pathological examination
* Chemotherapy failure or recurrent B cell malignancy
* Creatinine\< 2.5mg/dl
* Glutamic-pyruvic transaminase, glutamic oxalacetic transaminase\< 3 fold of normal level
* Bilirubin\<2.0mg/dl
* Karnofsky Performance Status\>50% at the time of screening
* Adequate pulmonary, renal, hepatic, and cardiac function
* Fail in autologous or allogenic haemopoietic stem cell transplantation
* Free of leukocytes removal contraindications
* Voluntarily join CAR-T clinical trial
* Understand and sign written informed consent

Exclusion Criteria:

* Pregnant or nursing women
* Active hepatitis B, active hepatitis C, or any human immunodeficiency virus (HIV) infection at the time of screening
* Feasibility assessment proves that the efficiency of transduction of lymphocyte is below 10% or the lymphocyte cannot be propagated.
* Abnormal vital signs
* Highly allergic constitution or history of severe allergies, especially allergy to interleukin-2
* General infection or local severe infection, or other infection that is not controlled
* Dysfunction in lung, heart, kidney and brain
* Severe autoimmune diseases
* Other symptoms that are not applicable for CAR-T
Plasmafit® Revision Structan® Hip Endoprosthesis Cup

The aim of this observational study is to collect clinical and radiological results of the new Plasmafit® Revision Structan® Hip Endoprosthesis Cup in a standard patient population and when used in routine clinical practice.


Eligibility Criteria:
Inclusion Criteria

* Indication for the cementless Plasmafit® Revision Structan® Hip Endoprosthesis Cup (primary and revision cases, with bone conditions typically demanding a press-fit revision cup)
* Written informed consent
* Cases where a press-fit cup fixation is possible

Exclusion Criteria

* Patients \< 18 years
* Pregnant patients
* Patients not able to comply with the study demands, i.e. follow-up visits (mentally or physically)
* Patient has an infection in the hip joint region
Role Of Multi-detector Computed Tomography In Differentiation Between Different Types Of Ascites

Role Of Multi-detector Computed Tomography In Differentiation Between Different Types Of Ascites

1. To detect accuracy of MDCT in differentiation between different types of ascites and its underlying cause .
2. To evaluate the diagnostic accuracy of measuring the CT density of the ascitic fluid (in Housefield units) to differentiate between different types of ascites in comparison to aspiration cytology results.

Eligibility Criteria:
Inclusion Criteria:

* All patients who are diagnosed with ascites by clinical examination or by any other investigations such as ultrasound.

Exclusion Criteria:

* Patient with bleeding tendancy and on anticoagulant drugs.-
* Patients who are pregnant .
TTX-030 Single Agent and in Combination With Immunotherapy or Chemotherapy for Patients With Advanced Cancers

This is a phase 1/1b study of TTX-030, an antibody that inhibits CD39 enzymatic activity, leading to accumulation of pro-inflammatory adenosine triphosphate (ATP) and reduction of immunosuppressive adenosine, which may change the tumor microenvironment and promote anti-tumor immune response.

This trial will study the safety, tolerability, pharmacokinetics, and anti-tumor activity of TTX-030 as a single agent and in combination with an approved anti-PD-1 immunotherapy and standard chemotherapies.


Eligibility Criteria:
Abreviated Inclusion Criteria

1. Advanced solid tumor malignancy or relapsed/refractory lymphoma, or

   * eligible to receive single-agent pembrolizumab as standard of care, or
   * eligible to receive single-agent docetaxel as standard of care, or
   * advanced pancreatic adenocarcinoma and eligible to receive gemcitabine plus nab-paclitaxel as standard of care.
2. Age 18 years or older, is willing and able to provide informed consent
3. Evidence of measurable disease
4. Life expectancy \> 12 weeks and Eastern Cooperative Oncology Group (ECOG) performance status of 0-1

Abbreviated Exclusion Criteria

1. History of allergy or hypersensitivity to study treatment components. Patients with a history of severe hypersensitivity reaction to any monoclonal antibody.
2. Use of investigational agent within 28 days prior to the first dose of study treatment and throughout the study
3. Receiving high-dose systemic steroid therapy or any other form of immunosuppressive therapy
4. History of severe autoimmune disease
5. Uncontrolled intercurrent illness or other active malignancy requiring ongoing treatment