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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study evaluates the long-term safety and clinical activity of mRNA-3927 in participants with propionic acidemia who have previously completed the mRNA-3927-P101 study. Participants will receive mRNA-3927 at the same dose and dosing interval as in the prior study. The study consists of a treatment period followed by a follow-up period lasting up to two years after the last dose of the study drug. The aim is to ensure the continued safety and efficacy of mRNA-3927 in this patient population.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Participated in Study mRNA-3927-P101.
* Completed the EOT/ET visit in Study mRNA-3927-P101 and enroll in this study such that the first dose in this study is planned to be within 14±3 days of the last dose of mRNA-3927 in the mRNA-3927-P101 study.

Exclusion Criteria:

* Not expected to receive clinical benefit from continued mRNA-3927 administration, in the opinion of the Investigator.
* Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the individual at increased risk by participating in this study.
* History of liver and/or kidney transplant.

Where it's running

• University of Stanford Medical Center
  Palo Alto, California, United States Recruiting
• University of Michigan Hospitals
  Ann Arbor, Michigan, United States Recruiting
• Icahn School of Medicine at Mount Sinai
  New York, New York, United States Recruiting
• Duke University Medical System (Duke Health)
  Durham, North Carolina, United States Recruiting
• The Children's Hospital of Philadelphia (CHOP)
  Philadelphia, Pennsylvania, United States Recruiting
• Texas Children's Hospital
  Houston, Texas, United States Recruiting

Study contacts

• Study coordinator: Moderna WeCare Team
• Email: WeCareClinicalTrials@modernatx.com
• Phone: 1-866-663-3762

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• 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.

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• 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

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.