Clinical trials: BCAA
Phenylketonurias
Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
This is a 2 arm, randomised, controlled, cross-over study in 16 children with PKU. Subjects who are currently taking a Phe free/low Phe protein substitute will be recruited for a 31-day trial.
- Trial name
- NCT05487378
- Testing
- PKU GOLIKE
- Where
- United Kingdom
- Volunteers
- 16 people
- Sponsor
- APR Applied Pharma Research s.a.
Type I Diabetes Mellitus
Glutamine and Insulin Sensitivity in Type I Diabetes
Insulin is crucial to help the body metabolize ('burn') sugar (glucose). Even though juvenile (type 1) diabetes (T1D) is primarily due to the lack of insulin, patients with T1D tend to become less sensitive to insulin…
- Trial name
- NCT01467063
- Testing
- Glutamine
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Nemours Children's Clinic
Urea Cycle Disorders, Inborn
Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders
The investigators will study and compare how effectively sodium phenylbutyrate, sodium benzoate, and a combination of the two, help excrete nitrogen in healthy volunteers.
- Trial name
- BPA/Benzoate (NCT02111200)
- Testing
- Sodium Benzoate, Sodium Phenylbutyrate
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Baylor College of Medicine
Albinism
Trial of L-DOPA as a Treatment to Improve Vision in Albinism
This project will evaluate the effect of two doses of levodopa (L-DOPA) in a randomized, placebo-controlled, double-masked clinical trial to see if vision can be improved in individuals with albinism.
- Trial name
- NCT01176435
- Testing
- Levodopa
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- University of Minnesota
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
Hereditary Tyrosinemia, Type I
Taste and Palatability of Orfadin Suspension
The purpose of this study is to verify that pediatric patients, especially those who are not old enough to swallow capsules, accept the taste and palatability of a new suspension.
- Trial name
- NCT01734889
- Testing
- Nitisinone
- Where
- 7 sites in France, Germany and 1 more
- Volunteers
- 18 people
- Sponsor
- Swedish Orphan Biovitrum
Phenylketonuria
Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
This is a Phase 3b, multicenter, open-label, randomized, controlled study to evaluate efficacy, safety and population pharmacokinetics of sapropterin dihydrochloride (Kuvan®) in less than 4 year-old infants and children with…
- Trial name
- SPARK (NCT01376908)
- Testing
- Kuvan®, Phenylalanine (Phe)-restricted diet
- Where
- 22 sites in Austria, Belgium and 7 more
- Volunteers
- 56 people
- Sponsor
- BioMarin Pharmaceutical
Phenylketonuria
Efficacy and Safety of SYNB1934 in Patients With PKU (SYNPHENY-3)
SYNB1934-CP-003 was designed as a 3-part, adaptive study consisting of a dose-escalating, open-label period (DEP; Part 1) of up to 15 weeks, followed by a 4-week, double-blind, placebo-controlled, randomized withdrawal period…
- Trial name
- SYNPHENY-3 (NCT05764239)
- Testing
- SYNB1934v1
- Where
- 21 sites in United States, Canada and 2 more
- Volunteers
- 35 people
- Sponsor
- Synlogic
Cytomegalovirus Infection
CMV Antiviral Prevention Strategies in D+R-Liver Transplants ("CAPSIL")
This is a trial of preemptive therapy vs. prophylaxis for prevention of Cytomegalovirus (CMV) disease in R-D+ liver transplant patients.
- Trial name
- NCT01552369
- Testing
- Valganciclovir
- Where
- 6 sites in United States
- Volunteers
- 205 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Arginase 1 Deficiency
Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency
This is an open-label, multicentre study to evaluate the safety, PK, and activity (PD) of weekly subcutaneous (SC) administration of pegzilarginase in subjects with ARG1-D who are < 24 months of age.
- Trial name
- NCT06582524
- Testing
- Pegzilarginase
- Where
- 3 sites in Austria, Portugal and 1 more
- Volunteers
- 3 people
- Sponsor
- Immedica Pharma AB
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
Phenylketonuria
An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
The BMN 165 clinical development program has been designed to demonstrate the safety and efficacy of BMN 165 in reducing blood Phe concentrations in patients 18 to 70 years old with hyperphenylalaninemia due to PKU.
- Trial name
- Prism301 (NCT01819727)
- Testing
- BMN 165
- Where
- 31 sites in United States
- Volunteers
- 261 people
- Sponsor
- BioMarin Pharmaceutical
Hyperhomocysteinemia, Obesity
Effect of Folic Acid Supplementation on Plasma Homocysteine Level in Obese Children
The purpose of the present study was to determine whether folic acid supplementation could reduce plasma homocysteine in obese children and to determine the association between dietary folate, serum folate and homocysteine level…
- Trial name
- NCT01766310
- Testing
- Folic Acid
- Where
- 2 sites in Thailand
- Volunteers
- 50 people
- Sponsor
- Queen Sirikit National Institute of Child Health
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
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
Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia
Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
The overall objective of this drug trial is to determine whether the treatment of acute hyperammonemia with N-carbamyl-L-glutamate (NCG, Carglumic acid) in propionic acidemia (PA), methylmalonic acidemia (MMA), late-onset CPS1…
- Conditions
- Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia, Carbamoyl-Phosphate Synthase I Deficiency Disease, Ornithine Carbamoyltransferase Deficiency
- Trial name
- STO (NCT01599286)
- Testing
- Carbaglu
- Where
- 9 sites in United States
- Volunteers
- 35 people
- Sponsor
- Mendel Tuchman
Phenylketonuria
Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria
For individuals with Phenylketonuria (PKU), the investigators hypothesize that glycomacropeptide will provide an acceptable form of low-phenylalanine dietary protein that will improve dietary compliance, blood phenylalanine…
- Trial name
- PKU (NCT01428258)
- Testing
- GMP Diet/GMP Medical Foods, AA Diet/AA Medical Foods
- Where
- 2 sites in United States
- Volunteers
- 32 people
- Sponsor
- University of Wisconsin, Madison
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
Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU
The purpose of this study is to evaluate whether weekly injections of phenylalanine ammonia lyase (rAvPAL-PEG) can reduce blood phenylalanine concentrations in PKU subjects and whether repeated administration is safe.
- Trial name
- NCT00925054
- Testing
- rAvPAL-PEG 0.001 mg/kg, rAvPAL-PEG 0.003 mg/kg, rAvPAL-PEG 0.01 mg/kg
- Where
- 11 sites in United States
- Volunteers
- 40 people
- Sponsor
- BioMarin Pharmaceutical
Anatomic Stage IV Breast Cancer AJCC v8, Invasive Breast Lobular Carcinoma
Fluciclovine and PSMA PET/CT for the Classification and Improved Staging of Invasive Lobular Breast Cancer
This phase I trial studies how well fluciclovine positron emission tomography (PET)/computed tomography (CT) and PSMA PET/CT work in helping doctors understand and classify invasive lobular breast cancer in patients with invasive…
- Conditions
- Anatomic Stage IV Breast Cancer AJCC v8, Invasive Breast Lobular Carcinoma, Metastatic Breast Lobular Carcinoma, Prognostic Stage IV Breast Cancer AJCC v8
- Trial name
- NCT04750473
- Testing
- Computed Tomography, Fluciclovine F18, Gallium Ga 68 Gozetotide
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Emory University