Clinical trials: BCAA
Urea Cycle Disorder
Chronic Liver Disease in Urea Cycle Disorders
This is a pilot, cross-sectional study to assess liver stiffness and markers of hepatic injury, function, and fibrosis in patients with urea cycle disorders.
- Trial name
- NCT03721367
- Testing
- Diagnostic Ultrasound
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Baylor College of Medicine
Phenylketonuria
Phenylketonuria, Oxidative Stress, and BH4
The purpose of this study is to see how tetrahydrobiopterin therapy (BH4; also known as sapropterin dihydrochloride or Kuvan) affects measures of oxidative stress and endothelial function in patients with Phenylketonuria (PKU).
- Trial name
- NCT01395394
- Testing
- Kuvan, Meal Challenge
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Emory University
Unresectable or Metastatic Malignant Gastrointestinal Stromal Tumor (GIST)
A Study of the Efficacy and Safety of Imatinib Mesylate in Patients With Unresectable or Metastatic Gastrointestinal Stromal Tumors Expressing C-kit Gene
In the core study, participants with unresectable or metastatic gastrointestinal stromal tumors expressing c-kit were treated with either 400 mg or 600 mg imatinib mesylate for 3 years.
- Trial name
- NCT00237185
- Testing
- Imatinib mesylate
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 148 people
- Sponsor
- Novartis Pharmaceuticals
Phenylketonuria
Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response
The investigators will investigate the effect of blood phenylalanine on Kuvan responsiveness in the same patients with PKU when their blood phenylalanine concentrations are altered by diet.
- Trial name
- NCT00841100
- Testing
- Kuvan, Diet
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- University of Miami
Schizo-affective Disorder, Bipolar Disorder
Neurobiology of a Mutation in Glycine Metabolism in Psychotic Disorders
The purpose of this study is to assess the efficacy of oral glycine as an augmentation strategy in two psychotic patients with a triplication (4 copies) of the gene glycine decarboxylase (GLDC).
- Trial name
- NCT01720316
- Testing
- Glycine
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Mclean Hospital
Ornithine Transcarbamylase (OTC) Deficiency
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
This is a Phase 1/2, open-label, single arm, multicenter, safety and dose finding study of DTX301 in adults with late-onset OTC deficiency.
- Trial name
- CAPtivate (NCT02991144)
- Testing
- scAAV8OTC, Reactive Corticosteroid Taper Regimen, Prophylactic Corticosteroid Taper Regimen
- Where
- 9 sites in United States, Canada and 2 more
- Volunteers
- 16 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Growth Hormone Treatment, Aging
Effects of an Amino Acid-Based hGH Secretagogue on Triiodythyronine
In a recent randomized, double-blind, cross-over clinical trial, serum growth hormone (hGH) increased 682% above baseline 120 minutes after oral administration of an amino acid-based dietary supplement (SeroVital), p=0.01 vs…
- Trial name
- NCT02987868
- Testing
- Amino acid supplement first day, Amino acid supplement second day
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Pennington Biomedical Research Center
Phenylketonuria (PKU)
Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
The BMN 165 clinical development program has been designed to demonstrate the safety and efficacy of BMN 165 in reducing blood Phe concentrations in adults with PKU.
- Trial name
- Prism-2 (NCT01889862)
- Testing
- BMN165 20mg/day, BMN165 40mg/day
- Where
- 29 sites in United States
- Volunteers
- 215 people
- Sponsor
- BioMarin Pharmaceutical
Phenylketonuria (PKU)
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.
- Trial name
- PEGASUS (NCT05270837)
- Testing
- Pegvaliase, Diet Only
- Where
- 16 sites in United States, Germany
- Volunteers
- 55 people
- Sponsor
- BioMarin Pharmaceutical
Urea Cycle Disorder
Manipulating the Gut Microbiome Study
The objective is to determine if acetohydroxamic acid (AHA) can prevent hydrolysis of urea by inhibiting the bacterial urease of gut flora of both healthy control adults as well as adults with urea cycle disorders
- Trial name
- NCT03181828
- Testing
- Acetohydroxamic Acid Oral Tablet, No treatment
- Where
- United States
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Nicholas Ah Mew
Endothelial Dysfunction, Hyperhomocysteinemia
Effect of L-Arginine and Pycnogenol on Light to Moderate Hypertension and Endothelial Function
The effect of a combination product (Verum ) with L-arginine, Pycnogenol, vitamin K2, R-(+)-alpha-lipoic acid and vitamins B6, B12 and folic acid is investigated in a double blind placebo-controlled cross-over study.
- Conditions
- Endothelial Dysfunction, Hyperhomocysteinemia, Hypertension Grade I, Subgroup "Borderline" (WHO)
- Trial name
- NCT02392767
- Testing
- Verum
- Where
- Germany
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Dr. Loges & Co. GmbH
Depressive Disorder, Major, Depressive Disorder, Treatment-Resistant
Safety, Tolerability, PK Profile, and Symptom Response of a 7-Day Dosing With 25 mg or 50 mg Daily of REL-1017 in MDD
This a Phase 2a, multicenter, randomized, double-blind, placebo controlled 3 arm study to assess the safety and tolerability of multiple oral doses of REL-1017 25 mg and 50 mg as adjunctive therapy in the treatment of patients…
- Trial name
- NCT03051256
- Testing
- REL-1017
- Where
- 10 sites in United States
- Volunteers
- 62 people
- Sponsor
- Relmada Therapeutics, Inc.
Phenylketonuria
A Study of PTC923 in Participants With Phenylketonuria
The main purpose of this trial is to evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in participants with phenylketonuria as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6…
- Trial name
- NCT05099640
- Testing
- PTC923
- Where
- 43 sites in United States, Australia and 13 more
- Volunteers
- 157 people
- Sponsor
- PTC Therapeutics
Gastroenteropancreatic Neuroendocrine Tumors
Post-Authorization Safety Study (PASS) of LysaKare® in Adult Gastroenteropancreatic Neuroendocrine Tumor (GEP-NET) Patients
The purpose of the study was to evaluate the effect of arginine/lysine solution administration on serum potassium levels. A systematic assessment of serum potassium levels was performed during infusion and up to 24 hours post…
- Trial name
- NCT04524442
- Testing
- arginine/lysine
- Where
- 7 sites in Italy, Netherlands and 2 more
- Volunteers
- 42 people
- Sponsor
- Advanced Accelerator Applications
NAFLD
Study of the Safety and Tolerability of AXA1957 in Adolescent Subjects With Non-Alcoholic Fatty Liver Disease (NAFLD)
This is a randomized, single blind study to determine whether AXA1957, a novel composition of amino acids, is safe and well tolerated.
- Trial name
- NCT04073407
- Testing
- AXA1957
- Where
- 4 sites in United Kingdom
- Volunteers
- 9 people
- Sponsor
- Axcella Health, Inc
Disaster, Vulnerable Populations
Nurses Taking On Readiness Measures - Mercer County, New Jersey
Nurses Taking On Readiness Measures (N-TORM) is an innovative nurse-driven household emergency preparedness (HEP) intervention modeled after an existing community intervention provided by emergency management personnel.
- Trial name
- NTORM (NCT05455580)
- Testing
- Nurses Taking On Readiness Measures (N-TORM)
- Where
- United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Hunter College of City University of New York