Clinical trials: Propionic Acidemia, open to volunteers
- Condition Propionic Acidemia
- Status Open to volunteers
- Funder type Industry (pharma, biotech, devices)
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.
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.
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
Methylmalonic Acidemia, Propionic Acidemia
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
This is a prospective mixed-design study focused on the long-term management of propionic aciduria (PA) and methylmalonic aciduria (MMA) with N-carbamylglutamate (NCG) maintenance therapy.
- Trial name
- PROTECT (NCT04176523)
- Testing
- Carglumic Acid
- Where
- 32 sites in France, Germany and 5 more
- Volunteers
- 95 people
- Sponsor
- RECORDATI GROUP
Propionic Acidemia
Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with…
- Trial name
- NCT04159103
- Testing
- mRNA-3927
- Where
- 35 sites in United States, Canada and 6 more
- Volunteers
- 77 people
- Sponsor
- ModernaTX, Inc.