Clinical trials: Propionic Acidemia
Children
Branched Chain Amino Acids Ratio in Medical Foods
This study will evaluate the branched-chain amino acids (BCAA) ratio in the medical foods formulated for Methylmalonic and Propionic Acidemias (MMA/PROP) patients.
- Trial name
- NCT03539809
- Testing
- L-Amino Acids
- Where
- Canada
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- University of British Columbia
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
Propionic Acidemia
A Retrospective Study to Characterize Participants With Propionic Acidemia
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
- Trial name
- NCT05769621
- Where
- 29 sites in United States, Australia and 7 more
- Volunteers
- 60 people
- Sponsor
- ModernaTX, Inc.
Urea Cycle Disorders, Inborn, Inborn Errors of Metabolism
Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
Hyperammonemia, which can cause brain damage, occurs in many different kinds of inborn errors of metabolism. The investigators propose to determine if short-term (3 day) treatment with N-carbamylglutamate can diminish…
- Conditions
- Urea Cycle Disorders, Inborn, Inborn Errors of Metabolism, Propionic Acidemia, Methylmalonic Acidemia
- Trial name
- NCT01341379
- Testing
- N-carbamylglutamate
- Volunteers
- healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)
Long-term Efficacy of Carglumic Acid in Organic Acidemia.
Evaluate the Long Term Effectiveness \& Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).
- Trial name
- NCT04284917
- Testing
- Carglumic Acid (Carbaglu®)
- Where
- Taiwan
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Propionic Acidemia
A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103).
- Trial name
- NCT05130437
- Testing
- mRNA-3927
- Where
- 22 sites in United States, Canada and 6 more
- Volunteers
- 50 people
- Sponsor
- ModernaTX, Inc.