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Study of Growth Hormone Inhibition Using Pegvisomant in Severe Insulin Resistance

Insulin Receptor MutationPartial Lipodystrophy

Background: Lipodystrophy (LD) syndromes are a group of rare disorders that affect how a person s body can store and use fat tissue. Many people with LDs become severely insulin resistant. Some people are insulin resistant because of a variant in the insulin receptor gene. Insulin resistance causes many health problems. Objective: To learn if blocking the effects of growth hormone in the body will help people with severe insulin resistance. Eligibility: Adults aged 18 to 65 years with either a known variant in the insulin receptor gene or with a diagnosis of partial LD. Design: Participants will have 2 hospital stays, about 1 month apart. Each stay will be 3 or 4 nights. During each hospital stay, participants will have many tests. They will have a physical exam with blood tests. They will have all of their urine collected for a 24-hour period. They will have scans to measure their muscle, bone, and fat tissues. They will have tests to measure metabolism and insulin sensitivity. They may have an optional biopsy of fat tissue. During the first hospital visit, participants will learn how to give themselves shots of a drug (pegvisomant) that blocks growth hormone. The drug is injected under the skin. Participants will continue to give themselves these shots once a day at home. After the first hospital visit, participants will talk on the phone with members of the study team once each week. After 2 weeks they will have blood drawn for tests. Participants will stop the shots after the second hospital visit.

Recruiting23 enrollment criteria

A Study to Evaluate the Efficacy, Safety, and Drug Levels of Oral Ozanimod in Pediatric Participants...

Crohn Disease

The purpose of this study is to evaluate the efficacy, safety, drug levels, and drug effects of ozanimod in pediatric participants with moderately to severely active Crohn's Disease.

Recruiting7 enrollment criteria

L-Arginine in Children Having Sickle Cell Disease With Increased Tricuspid Regurgitant Jet Velocity...

Sickle Cell Disease

This study aims to investigate the possible efficacy and safety of L-Arginine in children having Sickle Cell Disease with increased Tricuspid Regurgitant Jet Velocity

Recruiting8 enrollment criteria

Brighter Bites & UT Physicians Produce Rx Program

ObesityChildhood

The purpose of this study is to clinically evaluate the effectiveness of an at-home produce delivery prescription or grocery store vouchers prescription at improving weight status and obesity-related health outcomes of participants across the two clinics as compared to a control group and to examine the impacts of the program on child dietary behavioral outcomes (child fruit and vegetable intake, junk food consumption, and eating at any type of restaurant), and parent feeding practices (preparing foods from scratch, use of nutrition facts labels to make purchasing decisions, and eating meals with their referent child)

Recruiting6 enrollment criteria

Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis

Generalized Myasthenia Gravis

The purpose of this study is to evaluate the safety and efficacy of ALXN1720 for the treatment of generalized MG (gMG) in adults with autoantibodies against acetylcholine receptor (AChR).

Recruiting6 enrollment criteria

STAY-STRONG Study of Exercise Training During Chemotherapy

LymphomaB-Cell10 more

This study evaluates the effectiveness of a supervised progressive resistance training program in patients malignant lymphomas with the primary outcome being lean body mass. The study is designed as a a single center, two-armed, parallel-group, investigator-initiated clinical randomized controlled superiority trail evaluating the effectiveness of a 4-month supervised progressive resistance training intervention compared to usual care.

Recruiting12 enrollment criteria

A Study to Evaluate the Efficacy, Safety, and Tolerability of MYK-224 in Participants With Symptomatic...

CardiomyopathyHypertrophic

The purpose of this study is to characterize the safety, tolerability, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of MYK-224 in participants with obstructive Hypertrophic Cardiomyopathy (oHCM)

Recruiting24 enrollment criteria

A Study of Modakafusp Alfa in Adult Participants With Multiple Myeloma

Multiple Myeloma

The main aims of this study are to test for any side effects from modakafusp alfa in combination therapy and to determine the recommended dose of combination therapy with modakafusp. The dose of modakafusp alfa will be increased a little at a time until the highest dose that does not cause harmful side effects is found. Participants will be given modakafusp alfa through a vein.

Recruiting32 enrollment criteria

Multi-Center Study of Panosyl-Isomaltooligosaccharides Adjunctive to PPI Therapy to Treat GERD

Gastroesophageal Reflux

This study will be conducted as a multi-center, randomized, double-blind, placebo-controlled trial to evaluate the effect of MHS-1031 on heartburn-free days in subjects with GERD-related heartburn symptoms.

Recruiting94 enrollment criteria

Reduced-dose Chemotherapy Followed by Blinatumomab in Induction Therapy of Newly Diagnosed Non-elderly...

B Acute Lymphoblastic Leukemia

Blinatumomab, a CD3/CD19 bisespecific T-cell conjugative antibody, has shown high efficacy in phase I/II studies of relapsed/refractory B-lymphoblastic leukemia (B-ALL), particularly in the context of low tumor burden.Meanwhile, Blinatumomab also plays an important role in rapid and efficient clearance of MRD in patients. Therefore, its use in combination with less intensive chemotherapy for initial induction therapy in newly diagnosed patients may result in favorable response rates, greater depth of remission, and lower treatment-related toxic effects. In this study, newly diagnosed non-elderly patients with Philadelphia chromosomal negative (PH-) B-ALL were enrolled and treated with reduced-intensity chemotherapy followed by Blinatumomab as the basis of induction therapy. The clinical remission rate, MRD negative rate and treaty-related adverse reactions were evaluated in newly diagnosed non-elderly PH-B-ALL patients during induction therapy.

Recruiting17 enrollment criteria
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