Clinical trials: Amino Acids
Phenylketonuria
Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria
This Phase 2 study in patients with phenylketonuria (PKU) will be an open-label, dual-arm study of either a SYNB1618 or SYNB1934 dose-ramp regimen.
- Trial name
- SynPheny-1 (NCT04534842)
- Testing
- SYNB1618, SYNB1934
- Where
- 11 sites in United States
- Volunteers
- 20 people
- Sponsor
- Synlogic
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
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
Locally Advanced or Metastatic EGFR(+) NSCLC Patients
ARTEMIS DIANE T790M (An Amino Acid Substitution at Position 790 in EGFR, From a Threonine (T) to a Methionine (M)) Mutation at Hospital Laboratories in Comparison With Central Laboratory
The study primary objective is to assess the concordance of T790M resistance mutation testing from hospital-based laboratories with T790M resistance mutation testing from a central laboratory.
- Trial name
- NCT02991274
- Testing
- genomic testing of T790M mutation
- Where
- 31 sites in China
- Volunteers
- 897 people
- Sponsor
- AstraZeneca
Sports Injury
Clinical Trial for Vespa Amino Acid Mixture (VAAM®)
ATP is generated mainly through anaerobic metabolism during strenuous exercise, which is then followed by the generation of lactic acid.
- Trial name
- NCT03121040
- Testing
- Vespa amino acid mixture
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Shanghai Meiji Health Science and Technology Co., Ltd.
Phenylketonuria
Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period
The primary objective of the study is to evaluate the proportion of responders (that is, greater than or equal to [>=] 30 percent reduction from Baseline in blood phenylalanine [Phe] level) to treatment with Kuvan® (sapropterin…
- Trial name
- ENDURE (NCT01082328)
- Testing
- Kuvan®
- Where
- Norway
- Volunteers
- 59 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Hyperammonemia, Methylmalonic Acidemia
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
- Conditions
- Hyperammonemia, Methylmalonic Acidemia, Propionic Acidemia
- Trial name
- NCT05040178
- Testing
- Carglumic Acid
- Where
- 5 sites in United States
- Volunteers
- 20 people
- Sponsor
- RECORDATI GROUP
Healthy
Study to Determine D-amino Acid Oxidase Brain Enzyme Occupancy of TAK-831 After Single-dose Oral Administration
The purpose of this study is to determine the relationship between TAK-831 dose, plasma exposure, extent and duration of brain D-amino acid oxidase (DAO) enzyme occupancy following single oral dosing of TAK-831 in healthy…
- Trial name
- NCT02716987
- Testing
- TAK-831, [18F]PGM299
- Where
- United Kingdom
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Neurocrine Biosciences
Essential Hypertension
Efficacy of Amlodipine-folic Acid Tablets on Reduction of Blood Pressure and Plasma Homocysteine
To evaluate the efficacy of Amlodipine-folic Acid Tablets on reduction of blood pressure and plasma homocystein.
- Trial name
- NCT01848873
- Testing
- Amlodipine, amlodipine-FA tablet, low dose group, amlodipine-FA tablet ,high dose group
- Where
- 16 sites in China
- Volunteers
- 756 people
- Sponsor
- Shenzhen Ausa Pharmed Co.,Ltd
Arginase I Deficiency, Hyperargininemia
Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency
CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D.
- Trial name
- NCT03921541
- Testing
- Pegzilarginase
- Where
- 32 sites in United States, Austria and 5 more
- Volunteers
- 32 people
- Sponsor
- Immedica Pharma AB
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also…
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
- Trial name
- NCT06805695
- Testing
- No Intervention
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 13 people
- Sponsor
- iECURE, Inc.
Cytomegalovirus Retinitis, HIV Infections
A Study of Valganciclovir in the Treatment of Cytomegalovirus (CMV) Retinitis in Patients With AIDS
The purpose of this study is to see if valganciclovir is a safe treatment for CMV retinitis in patients who have been treated for this condition in the past.
- Trial name
- NCT00002222
- Testing
- Valganciclovir
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Hoffmann-La Roche
Bioavailability of Amino Acids
Amino Acids in the Blood After Eating Yoghurt and Milk Proteins
Participants will visit the study site twice in a fasting state. Participants asked to consume one of the two study products in a random order. Participants will consume one serving of the study product (T = 0 minutes).
- Trial name
- NutraFerm2 (NCT07243730)
- Testing
- Full fat yogurt (fermented), Generic full fat pasteurized milk (non-fermented)
- Where
- Netherlands
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Nutricia Research
Homocystinuria Due to CBS Deficiency
Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 1 to 65 years under current clinical management practices
- Trial name
- NCT02998710
- Where
- 11 sites in United States, Ireland and 2 more
- Volunteers
- 110 people
- Sponsor
- Travere Therapeutics, Inc.
Phenylketonuria
Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
This is an open-label, non-comparative, Phase 3 study to evaluate the degree, frequency of response and safety of Kuvan® (sapropterin dihydrochloride) in subjects aged 4 to 18 years who have phenylketonuria and with elevated…
- Trial name
- NCT01732471
- Testing
- Kuvan®
- Where
- Germany
- Volunteers
- 90 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Phenylketonuria
Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
The study is a single-cohort, interventional, open-label trial to evaluate long-term neurocognitive (NC) outcomes in children aged 4 to 5 years with phenylketonuria (PKU) treated with Kuvan® and Phenylalanine-restricted diet over…
- Trial name
- KOGNITO (NCT01965912)
- Testing
- Kuvan®
- Where
- 12 sites in Germany, Italy and 2 more
- Volunteers
- 34 people
- Sponsor
- BioMarin Pharmaceutical
Hereditary Tyrosinemia, Type I
Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)
The purpose of this study is to determine whether Nitisinone 10 mg Tablets (Test Product 1) and Nitisinone 10 mg Tablets 'Baked' for 6 months @ 40°C/75% RH (Test Product 2) are bioequivalent to the reference product Orfadin 10 mg…
- Trial name
- NCT02750345
- Testing
- Nitisinone, Nitisinone Baked Tablet, Orfadin
- Where
- South Africa
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.
Phenylketonuria (PKU)
PKU EASY Microtabs Plus - Acceptability and Tolerance
PEMP-AT is a prospective, single-arm, open-label, 1-week acceptability study to evaluate PKU EASY Microtabs Plus for the dietary management of participants with phenylketonuria (PKU).
- Trial name
- PEMP-AT (NCT06694662)
- Testing
- PKU EASY Microtabs Plus
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- Galen Limited
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
Cytomegalovirus Retinitis, HIV Infections
A Randomized Study Comparing the Safety and Efficacy of Two Regimens of Oral Ganciclovir to Intravenous Ganciclovir Maintenance Therapy for Cytomegalovirus Retinitis in People With AIDS Who Have Received Prior Ganciclovir Therapy
To compare the safety and tolerance of oral ganciclovir at a double dose 3 times/day or a single dose 6 times/day to IV ganciclovir given for 20 weeks of maintenance therapy.
- Trial name
- NCT00002247
- Testing
- Ganciclovir
- Where
- 20 sites in United States, Canada
- Volunteers
- 225 people
- Sponsor
- Hoffmann-La Roche