Clinical trials: Hyperammonemia
Hyperammonemia
A Healthy Volunteer Study to Compare Fezagepras (PBI-4050) With Sodium Phenylbutyrate
The purpose of this study is to compare the safety, tolerability and pharmacokinetic profile of fezagepras (PBI-4050) to that of sodium phenylbutyrate (PBA) when both products are given as single ascending doses to healthy adult…
- Trial name
- NCT05349435
- Testing
- Fezagepras, Sodium phenylbutyrate
- Where
- United Kingdom
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Liminal BioSciences Ltd.
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Ornithine Transcarbamylase Deficiency
Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI
The purpose of this study is to use various types of MRI and cognitive testing to evaluate changes in the brain and cognitive function that occur in subjects with ornithine transcarbamylase deficiency (OTCD) relative to healthy…
- Trial name
- NCT01569568
- Testing
- MRI scanning, Cognitive testing
- Volunteers
- 49 people, healthy volunteers welcome
- Sponsor
- Andrea Gropman
Hyperammonemia
A Phase 1 Study to Evaluate PK Profile and Food Effects on PK Parameters of TNP-2092 Capsules
The aim of the study was to evaluate the tolerability and pharmacokinetic characteristics of TNP-2092 Capsules after single-dose administration in healthy subjects, and the food effect on pharmacokinetics.
- Trial name
- NCT06178718
- Testing
- TNP-2092 capsules
- Where
- China
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- TenNor Therapeutics (Suzhou) Limited