Clinical trials: Propionic Acidemia
Methylmalonic Acidemia, Propionic Acidemia
"The MaP Study": Mapping the Patient Journey in MMA and PA
Longitudinal, exploratory, natural history study of patients with MMA due to mut deficiency and PA to characterize the changes in blood disease biomarkers over time and the frequency and severity of clinical events related to…
- Trial name
- NCT03484767
- Where
- 17 sites in United States, France and 2 more
- Volunteers
- 97 people
- Sponsor
- ModernaTX, Inc.
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.
Methylmalonic Acidemia, Propionic Acidemia
Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia
This is an interventional study to assess the safety, PK, and efficacy of HST5040 in 12 subjects - 6 with Methylmalonic Acidemia (MMA) and 6 with Propionic Acidemia (PA).
- Trial name
- HERO (NCT04732429)
- Testing
- HST5040
- Where
- 16 sites in United States, Australia and 1 more
- Volunteers
- 26 people
- Sponsor
- HemoShear Therapeutics
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
Healthy Volunteers, Propionic Acidemia
A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia
The purpose of this study is to assess the safety, tolerability, PK and PD of BBP-671 in healthy volunteers and patients with Propionic Acidemia or Methylmalonic Acidemia.
- Conditions
- Healthy Volunteers, Propionic Acidemia, Methylmalonic Acidemia, Organic Acidemia
- Trial name
- NCT04836494
- Testing
- BBP-671
- Where
- 3 sites in United States
- Volunteers
- 79 people, healthy volunteers welcome
- Sponsor
- CoA Therapeutics, Inc., a BridgeBio company
Methylmalonic Acidemia, Propionic Acidemia
Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia
The JUMP (Journey to Understand MMA and PA) Study is being conducted by HemoShear Therapeutics and AllStripes, a rare disease online research platform.
- Trial name
- JUMP (NCT05438485)
- Testing
- Registry
- Where
- 3 sites in United States, Canada and 1 more
- Volunteers
- 11 people
- Sponsor
- HemoShear Therapeutics
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.
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.