cyrilzakka/clinical-trials huggingface dataset:
Evaluating Cell Damage in Patients With Acute Myeloid Leukemia, Myelodysplastic Syndromes, or Fanconi Anemia; in Patients Who Were Exposed to Alkylating Agents; and in Healthy Volunteers

RATIONALE: Studying samples of bone marrow from patients with cancer and from healthy volunteers in the laboratory may help doctors learn more about changes that occur in bone marrow stromal (connective tissue) cells. It may also help doctors understand the effects of alkylating agents on bone marrow stromal cells.

PURPOSE: This laboratory study is evaluating stromal cells in patients with acute myeloid leukemia, myelodysplastic syndromes, or Fanconi anemia; in patients who were exposed to alkylating agents; and in healthy volunteers.

OBJECTIVES:

Primary

* Determine abnormal stromal function in patients with acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or Fanconi anemia; in patients who were exposed to alkylating agents; and in healthy volunteers.

Secondary

* Determine whether clonal progenitors from patients with secondary AML or MDS are resistant to selected extracellular apoptotic cues.
* Determine whether stromal function in patients with secondary AML or MDS is more aberrant than stromal function in patients with primary AML or MDS.
* Determine whether cytotoxic agents known to induce secondary MDS or AML influence the supportive function of the bone marrow stroma.
* Determine whether cytoprotective agents reduce both cytotoxicity and genotoxicity in hematopoietic progenitor cells and stromal cells.

OUTLINE: Patients and healthy volunteers undergo bone marrow sample collection. Progenitor cells are grown in culture. Cell survival is quantified by flow cytometric and cytogenetic analysis, sister chromatid exchange, and FISH for chromosome 11 changes (for etoposide-exposed samples only).

PROJECTED ACCRUAL: A total of 24 patients and healthy volunteers will be accrued for this study.

Eligibility Criteria:
DISEASE CHARACTERISTICS:

* Meets 1 of the following criteria:

  * Diagnosis of acute myeloid leukemia or myelodysplastic syndromes and requires bone marrow aspiration/biopsy for clinical purposes

    * Primary or secondary disease
  * Diagnosis of Fanconi anemia by positive mitomycin C test (age 5 to 55 years)
  * Received prior chemotherapy containing any of the following alkylating agents: mechlorethamine, chlorambucil, cyclophosphamide, melphalan, busulfan, or topoisomerase inhibitors
  * Healthy volunteer (age 18 and over), meeting the following criteria:

    * CBC normal
    * WBC \> 1,000/mm³
    * Hemoglobin \> 10 g/dL
    * Platelet count \> 70,000/mm³
* No bone marrow metastases
* No evidence of non-hematopoietic malignancy

PATIENT CHARACTERISTICS:

* ECOG performance status 0-2
* No clinical signs and symptoms of acute or subacute infection (viral, bacterial, or fungal infection)
* No allergy to lidocaine or xylocaine

PRIOR CONCURRENT THERAPY:

* See Disease Characteristics
* More than 6 months since prior cytotoxic or immunosuppressive agents
* No prior extensive pelvic radiotherapy (\> 20 Gy)
Lexaptepid Pegol (NOX-H94) in ESA-hyporesponsive Anemia in Dialysis Patients

Dialysis patients regularly suffer from anemia which may be caused by various contributing factors, alone or in combination, including blood loss, low erythropoietin and iron sequestration. In most patients, the anemia is responsive to treatment with erythropoietin or other erythropoiesis stimulating agents (ESA) alone or in combination with intravenous (i.v.) iron. In about 10% of patients however, the anaemia does not respond appropriately to this standard treatment and high to very high doses of ESA and i.v. iron are used to maintain acceptable hemoglobin concentrations. In these patients, hepcidin was identified as a causative factor leading to anemia of chronic disease with functional iron deficiency and ESA-hyporesponsiveness.

The Spiegelmer lexaptepid pegol (NOX-H94) offers a hepcidin-specific approach to the treatment of anemia of chronic disease. The safety and the activity of lexaptepid pegol are supported by data from healthy subjects and patients with multiple myeloma or lymphoma. The present study in dialysis patients with functional iron deficiency and ESA-hyporesponsiveness is conducted to demonstrate the safety of lexaptepid pegol in this population, to investigate its pharmacokinetic (PK) and pharmacodynamic (PD) profiles and its efficacy in increasing haemoglobin (Hb) in dialysis patients.


Eligibility Criteria:
Inclusion Criteria:

* End stage renal disease treated with maintenance hemodialysis.
* Anemia : Hb 7 to 11 g/dL.
* Functional iron deficiency: Transferrin saturation \<30%, Ferritin ≥300 ng/mL.
* ESA-hyporesponsiveness with erythropoietin dose ≥12,000 IU/ week.

Exclusion Criteria:

* Treatment with darbepoetin or methoxy-polyethyleneglycol-epoetin.
* Uncontrolled / unstable cardiovascular , peripheral arterial or cerebrovascular disease.
* Congestive heart failure: New York Heart Association Class III or IV.
* Unstable angina, myocardial infarction, percutaneous transluminal coronary angioplasty/stents, or coronary artery bypass grafting \<3 months prior screening.
* Any other medical conditions requiring a change in treatment within 4 weeks prior to screening or making study participation unadvisable.
* History of clinically relevant hemolysis and/or blood loss.
* AST, ALT, or bilirubin ≥2.0 times the upper limit of normal.
* Known bone marrow fibrosis.
* Treatment with i.v. iron \<4 weeks prior to screening or during the screening period or change in erythropoietin dose during last month.
* Any acute or chronic infection, viral or bacterial within 4 weeks prior to screening or during the screening period considered as systemic infection.
The Copenhagen Gender Identity Cohort

The goal of this observational study is to systematically collect data on adults seeking medical and surgical treatments for gender incongruence. This prospective, longitudinal cohort aims to generate insights into the safety, effectiveness, and overall satisfaction with current treatments. Additionally, the study seeks to identify areas for improvement and support healthcare professionals in making informed decisions. Most importantly, it aims to enhance the quality of life for transgender and gender-diverse individuals by ensuring that the care they receive aligns with their needs and goals.

Participants will include patients seeking treatment at the Copenhagen Center for Gender Identity, Denmark. Data collection will be both clinician- and patient-reported. Participants will be asked to complete online surveys at baseline, after the assessment process, and annually if they initiate hormone therapy. The study will systematically gather information on gender identity and transition experiences, sociodemographics, self-medication, health status, self-reported quality of life, and treatment preferences. Additionally, detailed records of treatment courses and specific interventions will be collected from all involved healthcare providers across specialties.

The study will evaluate the effects of different treatment options, both in the short and long term. It will explore how quality of life is associated with gender identity, transition, sociodemographics, lifestyle, and health, as well as assess the medical impact of various treatments, including counseling, hormone therapy, and surgery. Furthermore, the study will investigate the prevalence of side effects and complications related to treatment, as well as factors influencing treatment discontinuation, including cases of detransition.

Finally, the project will focus on quality indicators such as waiting times and patient satisfaction, contributing to the ongoing development of high-quality, patient-centered care for transgender and gender-diverse individuals.


Eligibility Criteria:
Inclusion Criteria:

* All patients referred to The Center for Gender Identity in Copenhagen

Exclusion Criteria:

* Inability to read and understand Danish
A Study to Assess the Efficacy of Raltegravir (Isentress®), Administered in Combination With Other Antiretroviral Drugs as Treatment for Adults and Older Adults Infected With the Human Immunodeficiency Virus 1 (HIV-1)(MK-0518-145) (Wirksamkeit Von Isentress® Unter Praxisbedingungen)

This is an observational, non-comparative, multicenter, open-label study. Participants will be treated with Raltegravir according to standard clinical practice, and monitored over a total period of 96 weeks. In an extension to the study (Amendment 1), a new cohort of aging participants (≥ 50 years) will be recruited and monitored over a total period of 48 weeks. Participants who stop taking Raltegravir before the end of the 96-week period or 48-week period, respectively, will be followed up for 3 months after discontinuing the drug. The primary objective is to determine the proportion of participants with a human immunodeficiency virus (HIV)-1 viral load \< 50 copies/mL after 48 weeks of treatment with Raltegravir.


Eligibility Criteria:
Inclusion Criteria

The prospective participant must meet, at least, all of the criteria below to be eligible for study participation. The participant:

* Is a minimum age of 18 years (adults) or 50 years (aging participants);
* Is male or female;
* Has confirmed infection with HIV-1 (positive HIV test according to appropriate standard practice);
* Has commenced antiretroviral treatment with Raltegravir according to the recommendations made in the Summary of Product Characteristics at the time of enrollment on the study, or a maximum 6 months prior to enrollment on the study;
* Has any cluster of differentiation (CD4) cell (specialized white blood cell) count upon enrollment on the study.

Exclusion criteria

If the prospective participant meets any of the criteria below (among others determined by the study staff) they will NOT be eligible for study participation. The participant:

* For which Raltegravir, or its ingredients, are contraindicated;
* Has intolerance to Raltegravir, or its ingredients;
* If female, is pregnant, breastfeeding, or is planning a pregnancy or egg donation during the study.
Testing Implementation Strategies to Improve Delivery of PrEP for Pregnant and Postpartum Women in Kenya

This study tests strategies for improving PrEP implementation in maternal and child health clinics using an interrupted time series.

This study aims to improve integrated delivery of PrEP to women seeking health services in maternal and child health clinics by piloting and evaluating four strategies or bundles of strategies for optimized PrEP delivery. Strategies to be tested include three packages of strategies identified by stakeholders.

Eligibility Criteria:
Inclusion Criteria:

* Women receiving Maternal and Child Health (MCH) services HCW participating in satisfaction surveys

Exclusion Criteria:

* Unwilling or unable to provide informed consent
Development and Validation of an Noninvasive Model for Predicting High Risk Esophageal Varices in Cirrhosis

The aim of the study is to develop and validate a new noninvasive method based on routine examination during clinical practice for predicting high risk esophageal varices in cirrhosis.

Portal hypertension is the main consequence of cirrhosis. Variceal bleeding is the principal and life-threatening complication of portal hypertension. Endoscopic screening is the golden standard to detect esophageal varices. However, endoscopy is limited by its invasiveness, cost and the discomfort it imposes on patients. With the goal of circumventing these disadvantages, we intend to develop an noninvasive method for predicting high risk esophageal varices.

Eligibility Criteria:
The inclusion criteria:

1. patients who were diagnosed with liver cirrhosis.
2. patients who underwent the abdominal contrast-enhanced CT examination.
3. patients who received endoscopic screening.
4. patients with written informed consent.

The exclusion criteria:

1. patients who previously underwent endoscopic therapy, transjugular intrahepatic portosystemic shunt (TIPS), splenectomy, partial splenic embolization (PSE), hepatectomy, balloon-occluded retrograde transvenous obliteration, or liver transplantation.
2. patients with liver cancer.
3. patients with severe ascites or hepatic encephalopathy.
4. lacks abdominal contrast-enhanced CT within 1 month of endoscopy.
Treatment Period and Long-term Effect of Functional Electric Stimulation (FES) for Bruxism

To investigate the effect of the treatment with Grindcare® on the muscles- and jaw symptoms and at the jaw muscle activity during sleep.


Eligibility Criteria:
Inclusion Criteria:

* Signed and dated ICF
* 18 years or older
* Fulfill the RDC-criteria for myofascial TMD diagnose (group 1a or 1b)
* Average pain more that 3 on a Visual Analog scale (VAS) in the baseline period

Exclusion Criteria:

* Contraindication of concommitant diseases for the study judged by investigator
* Daily use of pain medication, e.g. prophylaxis against migraine or headache.
* Patients who are using occlusal splints in the treatment period.
The Exploration of OX40 (CD134) Expression Levels in Sarcoma Specimens and Its Clinical Application in Prognosis Determination

This study aims to investigate the expression of OX40 (CD134) in sarcomas and its impact on prognosis, with the goal of identifying novel biomarkers for early resistance detection and optimizing treatment strategies for sarcoma patients. Sarcomas are a highly heterogeneous group of malignant tumors, for which current treatment options are often suboptimal in certain patients, and effective biomarkers to guide therapy are lacking. As a T-cell costimulatory receptor, OX40 plays a significant role in immune regulation across various solid tumors and holds promise as a potential therapeutic target.

Our research team has previously identified high OX40 expression in sarcomas through database analysis and validated the therapeutic efficacy of antibody-drug conjugates targeting OX40 in in vivo experiments using sarcoma cell lines in murine models. This study will evaluate the mRNA expression levels of OX40 in tumor tissues from sarcoma patients and assess OX40 protein expression using immunohistochemical staining. By integrating these findings with clinical and pathological data, the study will explore the potential of OX40 expression levels in tumor tissues as biomarkers for predicting treatment response and prognosis in primary bone tumors. The results aim to provide scientific evidence for the clinical application of OX40-targeted therapies and to propose novel therapeutic strategies to improve survival outcomes in sarcoma patients.


Eligibility Criteria:
Inclusion Criteria:

* Aged 8 years or older.
* Diagnosed with high-grade osteosarcoma or Ewing sarcoma, confirmed through clinical evaluation or histopathological examination. For patients with localized tumors or solitary pulmonary lesions, pathological confirmation is required; for patients with multiple pulmonary metastases, pathological examination is not necessary.
* Treatment-naïve patients who have not received standard first-line chemotherapy regimens for osteosarcoma or Ewing sarcoma.(First-line chemotherapy drugs include high-dose methotrexate, anthracyclines, cisplatin, and ifosfamide. For Ewing sarcoma, the standard first-line regimen is VDC followed by IE.)
* All other examination indicators suggest that the patient is eligible for standardized chemotherapy and surgical treatment for bone tumors.

Exclusion Criteria:

* Inability to undergo standardized chemotherapy and surgical treatment for bone tumors at this center.
* Refusal to adhere to follow-up according to standardized diagnostic and treatment protocols.
* Any condition that, in the investigator's opinion, may harm the participant or prevent them from meeting or complying with the study requirements.
Efficacy of Let's Know! First Grade

Oral language skills are vital for reading comprehension. Some children, however, are at increased risk for reading comprehension difficulties due to underlying oral language deficits. School-based interventions that target children's abilities to understand and produce spoken language have shown positive effects for improving language and reading comprehension of children with typical development and those at-risk for language disorders, so it is likely that they will benefit children with low oral language skills.

The purpose of this study is to examine the efficacy of a small-group intervention for improve language skills in first grade students with low oral language skills.

Oral language skills, which consist of both lower-level (vocabulary and grammar) and higher-level skills (inferencing, comprehension monitoring, and text structure knowledge) are crucial for reading comprehension. Children with low oral language skills are at increased risk for negative academic outcomes due to their deficits in understanding and producing language. Multi-component interventions have shown positive effects for improving language comprehension and reading comprehension outcomes of children with typical development and at-risk for language and literacy disorders, and thus may benefit children with low oral language skills.

The purpose of this study is to examine the efficacy of Let's Know!, a small group multi-component intervention, for improving language comprehension skills in first-grade students with low oral language skills. A randomized controlled trial will be employed to compare the language comprehension outcomes for a treatment group compared to a business-as-usual control group.

Eligibility Criteria:
Inclusion Criteria:

* enrolled in first-grade;
* receive a score of below than 5 on two items on the Student Language Scale (Nelson et al., 2018) or be receiving school speech-language services for language impairment;
* be proficient in English, per caregiver report;
* have no other neurological impairments (e.g., intellectual disability, autism) per parent report;
* have corrected vision;
* no hearing impairment.

Exclusion Criteria:

* cannot be enrolled in English-Language Learner services in the school.
Effects of Montelukast in Children With Asthma

Leukotriene receptor antagonists (LTRAs) reduce fractional exhaled nitric oxide (FENO) concentrations in children with asthma, but the effect of LTRA withdrawal on FENO and lung function is unknown. We will aim to study the effect of treatment and withdrawal of montelukast, a LTRA, on airway inflammation as reflected by FENO and lung function in children with asthma.

A double-blind, randomized, placebo controlled, parallel group study will be undertaken in 18 atopic children with mild persistent asthma who will be treated with oral montelukast (5 mg/day for 4 weeks) and 18 atopic children with mild persistent asthma who will receive matching placebo.

A follow-up visit will be performed 2 weeks after montelukast or placebo withdrawal.


Eligibility Criteria:
Inclusion Criteria:

* Children 6 to 14 yrs old, step 1 and step 2 GINA (Global INitiative for Asthma) guidelines
* exhaled nitric oxide (NO) \>20 PPB at visit 2
* symptoms more often than twice a day, FEV1 equal or higher than 80% of predicted value and reversibility equal or higher than 12% to salbutamol, or a positive provocation test with methacholine, or exercise
* no regular medication, inhaled short-acting beta-2 agonists for symptom relief

Exclusion Criteria:

* Patient is hospitalized
* Patient has FEV1 \< 80% predicted on visit 1
* Patient has 2 or more nighttime awakenings for asthma per week or pef variability 30% or more
* upper respiratory infection in the previous 3 weeks
* treatment with glucocorticoids or LTRAs in the previous 4 weeks
* treatment with inhaled glucocorticoids for more than 4 weeks in the previous year
Metabolomics of Liver Regeneration Following Partial Hepatectomy in Healthy Living Liver Donors

The PI will study metabolism during liver regeneration in living liver donors

Samples of blood and urine will be collected from study participants at defined timepoints. The samples will be evaluated to study the metabolism of the regenerating liver after a portion of the liver has been donated from a living donor.

Eligibility Criteria:
Inclusion Criteria:

1. Potential donor must be in excellent medical and psychological health.
2. Potential donor must be between the ages of 18 and 60.
3. Must have a compatible blood type to the recipient.
4. Must have health care coverage for non-donor related health issues that arise post donation
5. Recipient and donor must have compatible body size.
6. Donors Body Mass Index (BMI) must be less than 30.
7. All previous donor abdominal surgeries will be reviewed on a case by case basis by the surgeon.

Exclusion Criteria:

1. Positive blood test for Human Immunodeficiency Virus, Hepatitis C virus, Hepatitis B virus
2. The size of the donor liver is not large enough to safely resection and still retain enough volume to function normally after the surgery.
3. Liver biopsy show liver cirrhosis or early stages of HCC.

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Becaplermin Use and Cancer Risk in a Patient Population of U.S. Veterans With Diabetes

The purpose of this study is to evaluate risk of cancer incidence and mortality associated with the use of REGRANEX (becaplermin) in patients with diabetes who are members of a U.S. Veterans Health Administration.

This is an observational study of patients with diabetes who are eligible for care through the national Department of Veterans Affairs Health Care System (VA), representing the largest sample of older adults in an integrated health care system in the U.S. with comprehensive electronic medical records and linkable health survey data. REGRANEX (becaplermin) is topical medication used to treat lower extremity diabetic neuropathic ulcers. VA patients with diabetic foot ulcers who have no prior history of cancer who have received treatment with becaplermin will be compared to a group of similar patients who have not received treatment with becaplermin. Becaplermin users (from the time of initial dispensing of becaplermin) and becaplermin nonusers will be followed forward in time for up to 8 years to identify new cancers and cancer related deaths, with confirmation of cases through registries and medical chart review. This is an observational study; no investigational drug will be administered.

Eligibility Criteria:
Inclusion Criteria:

* Patients with diabetic foot ulcers who are members of the U.S Department of Veterans Affairs Health Care System (VA)

Exclusion Criteria:

* History of cancer (including nonmelanoma skin cancer) prior to study entry
Study of the Blood Concentrations of Two Formulations of REGN1033 in Healthy Subjects

Primary Objective: Determine blood concentrations of two formulations of REGN1033.

Secondary Objective: Assess safety and tolerability of REGN1033.


Eligibility Criteria:
Key Inclusion Criteria:

1. Healthy males and females (not of childbearing potential) between the ages of 18 and 65 years
2. Body weight between 50.0 kg and 95.0 kg
3. Willing and able to comply with clinic visits and study-related procedures
4. Provide signed informed consent

Key Exclusion Criteria:

1. Donation or loss of approximately 400 mL or more of blood within 8 weeks prior to dosing, or plasma up to 14 days prior to dosing
2. Hemoglobin not within normal limits
3. Positive drug and alcohol screen test results at screening visits 1 and 2
4. Low or elevated blood pressure and/or heart rate after 3 minutes resting in the seated position:
5. Recent use (within 3 months prior to screening) of androgenic steroids
6. Participation in any clinical research study that evaluated another investigational drug or therapy within 30 days or at least 5 half-lives, whichever is longer, of the investigational drug, prior to the screening visit
7. Previous exposure to any biological therapeutic agent, except vaccines
8. Known sensitivity to tamoxifen, doxycycline, or similar compounds (ie, tetracyclines)
9. Pregnant or breastfeeding women, and women of childbearing potential
10. Sexually active men who are unwilling to practice adequate contraception during the study