Clinical trials: Metabolic Disorders
Prader-Willi Syndrome, Hyperphagia
Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
The investigators propose a randomized double-blind 8 week treatment trial of intranasal oxytocin (IN-OXT) vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2)…
- Trial name
- OXT-PWS (NCT02629991)
- Testing
- Intranasal Oxytocin (IN-OXT)
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Montefiore Medical Center
Diabetes Mellitus, Type 1
Oral Insulin for Prevention of Diabetes in Relatives at Risk for Type 1 Diabetes Mellitus
Type 1 diabetes (T1D) is an autoimmune disease. This means that the immune system (the part of the body which helps fight infections) mistakenly attacks and destroys the cells that produce insulin (islet cells found in the…
- Trial name
- TN07 (NCT00419562)
- Testing
- Oral Insulin
- Where
- 18 sites in United States, Australia and 4 more
- Volunteers
- 560 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Trial name
- NCT00837824
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Obesity, Overweight
Mobile Attention Retraining in Overweight Female Adolescents
Background: People are constantly exposed to unhealthy foods. Some studies of adults show that training attention away from unhealthy foods might reduce overeating.
- Conditions
- Obesity, Overweight, Binge Eating, Healthy Volunteers
- Trial name
- NCT02977403
- Testing
- Attention Bias Retraining
- Where
- United States
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Urea Cycle Disorder
Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
THRIVE is an observational study that will collect information on patients with UCDs. THRIVE will follow enrolled participants for up to 10 years.
- Trial name
- THRIVE (NCT01948427)
- Where
- United States
- Volunteers
- 203 people
- Sponsor
- Amgen
Congenital Adrenal Hyperplasia
Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1
Children with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency tend to have elevated circulating levels of androgens, which can accelerate skeletal maturation and adversely impact adult height.
- Trial name
- NCT02574910
- Testing
- Abiraterone acetate
- Where
- 4 sites in United States
- Volunteers
- 4 people
- Sponsor
- University of Texas Southwestern Medical Center
Cystic Fibrosis
Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study
Protocol 0000498: Multicenter, open label study to evaluate the effect of sustained RELiZORB (immobilized lipase) cartridge use during enteral feeding on fat absorption, as well as safety and tolerability of sustained RELiZORB…
- Trial name
- ASSURE (NCT02750501)
- Testing
- RELiZORB (immobilized lipase) cartridge, Impact Peptide 1.5
- Where
- 10 sites in United States
- Volunteers
- 49 people
- Sponsor
- Alcresta Therapeutics, Inc.
Type 2 Diabetes Mellitus
Specified Drug-Use Survey of Trelagliptin Tablets "Survey on Long-term Use in Type 2 Diabetes Mellitus Patients With Severe Renal Impairment or End-stage Renal Disease"
The purpose of this study is to evaluate the long-term safety of trelagliptin tablets in patients with type 2 diabetes mellitus complicated by severe renal impairment or end-stage renal failure in the routine clinical setting.
- Trial name
- NCT04285983
- Testing
- Trelagliptin
- Where
- Japan
- Volunteers
- 89 people
- Sponsor
- Takeda
Type 1 Diabetes Mellitus
Rituximab in New Onset Type 1 Diabetes
Type 1 diabetes is an autoimmune disease in which the immune system mistakenly attacks the insulin-producing beta cells in the pancreas.
- Trial name
- TN05 (NCT00279305)
- Testing
- Anti-CD20 (rituximab)
- Where
- 16 sites in United States, Australia and 2 more
- Volunteers
- 87 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Fanconi Anemia
Eltrombopag for Fanconi Anemia
Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly.
- Trial name
- NCT03206086
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Leber Hereditary Optic Neuropathy
Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)
This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation.
- Trial name
- rAAV2-ND4 (NCT01267422)
- Testing
- rAAV2-ND4
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Bin Li
Fanconi Anemia
Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine
The purpose of this research study is to: (1) determine if the combination of low dose total body irradiation, low dose cyclophosphamide and the addition of fludarabine, and a serum to suppress the immune system can allow…
- Trial name
- NCT00595127
- Testing
- Cyclophosphamide, Fludarabine, Total body irradiation (TBI)
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Memorial Sloan Kettering Cancer Center
Overweight, Obesity
Two-year Research Study Investigating How Well Semaglutide Works in People Suffering From Overweight or Obesity
This study will look at the change in body weight from the start to the end of the study. Researchers will compare the weight loss in people taking semaglutide (a new medicine) to people taking "dummy" medicine.
- Trial name
- STEP 5 (NCT03693430)
- Testing
- Semaglutide
- Where
- 41 sites in United States, Canada and 3 more
- Volunteers
- 304 people
- Sponsor
- Novo Nordisk A/S
Muscular Dystrophy, Duchenne
Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients
The purpose of this study is to evaluate the effect of docosahexaenoic fatty acid and eicosapentaenoic fatty acid supplementation for six months on the inflammation state as well as the process of muscular regeneration and the…
- Trial name
- NCT01826422
- Testing
- EPA and DHA
- Where
- Mexico
- Volunteers
- 40 people
- Sponsor
- Coordinación de Investigación en Salud, Mexico
Type 1 Diabetes
Healthy And Positive Pathways for Young People With Type 1 Diabetes (HAPPY T1D)
In this 2-year randomized clinical trial, we will implement and assess the impact of a behavioral/psychoeducational intervention to reduce diabetes distress and improve glycemic outcomes in adolescents and young adults, aged…
- Trial name
- NCT05413239
- Testing
- Psychoeducation to reduce diabetes distress and improve glycemic outcomes
- Where
- 2 sites in United States
- Volunteers
- 217 people
- Sponsor
- Joslin Diabetes Center
Type1 Diabetes
Yale-Harvard Hotel-based Closed-Loop Studies in Children (HY-GRAID)
The aim of this clinical study is to determine the feasibility, safety, and preliminary effectiveness of the Zone-Model Predictive Control Artificial Pancreas (ZMPC_AP) system in pediatric subjects with type 1 diabetes in an…
- Trial name
- HY-GRAID (NCT04255381)
- Testing
- Zone-Model Predictive Control Artificial Pancreas (ZMPC_AP)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Yale University
Type 1 Diabetes
Glucose Variability in Type 1 Diabetes (T1D) and Glycaemic Responses to Food Composition.
The study proposed to recruit approximately 435 children and young people who have T1D and who regularly use Dexcom continuous glucose monitoring (CGM). Recruitment was be via their local dietitian.
- Trial name
- NCT06546657
- Testing
- Breakfast
- Where
- United Kingdom
- Volunteers
- 96 people
- Sponsor
- University of Stirling
Pediatric Cancer, Nutrition Related Cancer
Pediatric Oncology Nutrition Intervention Trial
Nearly 60% of pediatric patients diagnosed with cancer develop malnutrition caused by a combination of disease burden, side effects of chemotherapy, and the intensity of cancer treatment.
- Conditions
- Pediatric Cancer, Nutrition Related Cancer, Nutrition Aspect of Cancer, Muscle Loss
- Trial name
- POINT (NCT06175273)
- Testing
- Oral Nutrition Supplement, Tube Feed, Cyproheptadine
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Corey Hawes
Fabry Disease
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
The primary objective of this study was to compare the effect of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
- Trial name
- NCT00925301
- Testing
- migalastat hydrochloride
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 67 people
- Sponsor
- Amicus Therapeutics
Cerebral Adrenoleukodystrophy (CALD)
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the…
- Trial name
- NCT01896102
- Testing
- Lenti-D Drug Product (eli-cel)
- Where
- 8 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Genetix Biotherapeutics Inc.
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