Condition

Propionic Acidemia clinical trials and research

• 7Clinical trials
• 13Countries

Newest Propionic Acidemia clinical trials

All recruiting trials (7)

• AAV-based gene therapy for Propionic Acidemia in young children

  This trial tests whether an AAVrh10-PCCA gene therapy can be given safely to children with Propionic Acidemia to help their bodies manage the metabolic problem.

  Recruiting Phase 1 Mayo Clinic · Rochester, Minnesota · NCT07643844

• Characterizing people with propionic acidemia using past medical records

  This project collects and reviews medical records to see what health events and outcomes people with propionic acidemia experience.

  Recruiting at 15 sites ModernaTX, Inc. · Stanford, California and 28 other locations · NCT05769621

• Long-term safety assessment of mRNA-3927 for propionic acidemia patients

  This study is testing the long-term safety and effectiveness of the mRNA-3927 treatment for people with propionic acidemia who have already participated in a previous study.

  Recruiting at 12 sites Phase 1/2 ModernaTX, Inc. · Los Angeles, California and 21 other locations · NCT05130437

• Observing the effects of Carbaglu® in treating MMA and PA in patients

  This study is testing how safe Carbaglu® is for treating high ammonia levels in people with Methylmalonic Acidemia and Propionic Acidemia, both kids and adults.

  Recruiting at 5 sites Recordati Group · Washington D.C., District of Columbia and 4 other locations · NCT05040178

• Long-term management of organic acidemia with CARBAGLU®

  This study looks at how well carglumic acid helps people with propionic acidemia and methylmalonic acidemia over a long time, while also talking to patients and caregivers about their experiences.

  Recruiting at 31 sites Recordati Group · Bordeaux and 31 other locations · NCT04176523

• Treatment for Propionic Acidemia using mRNA-3927

  This study is testing a new treatment called mRNA-3927 to see if it is safe and effective for people with propionic acidemia, including infants and older children.

  Recruiting at 19 sites Phase 1/2 ModernaTX, Inc. · Los Angeles, California and 34 other locations · NCT04159103

• Understanding Propionic Acidemia and Its Effects

  This study is trying to learn more about propionic acidemia by looking at how it affects people with the condition and their families over time.

  Recruiting at 2 sites National Institutes of Health Clinical Center (CC) · Bethesda, Maryland and 1 other locations · NCT02890342

Related topics

Quick answers

How many Propionic Acidemia clinical trials are recruiting right now?

7, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 13 countries. The most sites are in United States (6 trials), France (4 trials), Spain (4 trials), United Kingdom (4 trials), Canada (3 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1/2: 2, Phase 1: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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