mRNA-3927 for Propionic Acidemia Study

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The purpose of this study is to find out more about an investigational drug called mRNA-3927 (the study drug). An "investigational drug" is a drug that is being tested and is not approved by the U.S. Food and Drug Administration (FDA). We want to see how safe the study drug is and if it works for participants with propionic acidemia (PA).

Who Do I Contact?

If you are interested in participating in the study or want to learn more please contact our study team at maddenr [at] chop.edu or 215-590-2238.

ClinicalTrials.gov Identifier

NCT04159103

View full trial information on ClinicalTrials.gov

Eligibility & Criteria

IRB #:
19-016977
Official Title:
A Global, Phase 1/2, Open-label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia
Study Phase:
Phase I
Phase II
Eligible Age Range:
1 - 99 Years
Gender:
All
Study Categories:

Visit Criteria

If you agree to take part, your participation will last about 1.5 years and will involve approximately 44 study visits, some of which may be completed by a Home Health Nurse should you be interested in using this service. There are differences between this study and your usual care. As a participant in this research, you will:

  • Receive a study drug via an intravenous (IV) infusion.
  • Stay inpatient to monitor reaction to study drug.
  • Have research blood draws, EKGs, Echos, and complete questionnaires.
Leader
Can Ficicioglu

Can Ficicioglu, MD, PhD