Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
Recruiting · Phase 1, Phase 2 · NCT04159103
Conditions: Propionic Acidemia
At a glance
- Research payments reported, 2025
- $1.2M
- U.S. investigators named
- 13
- Sites paid
- 14
- Planned or actual enrollment
- 77
About the trial
- Sponsor
- ModernaTX, Inc.
- Tested
- mRNA-3927 (biological)
- Dates
- Started 2021-04-15; completion 2027-08-31
- Results posted
- Not yet
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 genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination of the efficacy, safety, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (\<1 year of age).
From ClinicalTrials.gov, last updated 2026-01-22. Full record, eligibility and contacts (opens in a new tab)
Industry funding
ModernaTX, Inc. reported $1.2M in research payments for this study in 2025 (71 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Gregory M. Enns, M.D. Medical Genetics · Palo Alto, CA
- Gerald S. Lipshutz, MD Transplant Surgery · Los Angeles, CA
- Parul B. Jayakar, MD Medical Genetics · Miami, FL
- Carlos E. Prada, M.D. Medical Genetics · Chicago, IL
- Hilary J. Vernon, M.D., PH.D. Pediatrics · Baltimore, MD
- Ayesha Ahmad, MBBS Medical Genetics · Ann Arbor, MI
- Dwight D. Koeberl Pediatrics · Durham, NC
- Margo S. Breilyn, MD Medical Genetics · Bronx, NY
- Laura L. Konczal, M.D. Medical Genetics · Cleveland, OH
- Loren D. Pena, MD, PHD Medical Genetics · Cincinnati, OH
- Can H. Ficicioglu, M.D. Medical Genetics · Philadelphia, PA
- Kevin E. Glinton, M.D. Medical Genetics · Houston, TX
- Emily M. Shelkowitz, MD Pediatrics · Seattle, WA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Icahn School of Medicine at Mount Sinai $571,158
- Children's Hospital of Philadelphia, PA $462,344
- Ann & Robert H Lurie Childrens Hospital of Chicago, IL $61,155
- Uh Cleveland Medical Center, OH $23,750
- Nicklaus Children's Hospital, FL $23,639
- Johns Hopkins University $20,502
- University of Michigan Health System, MI $19,415
- Duke University $13,368
- Board of Trustees of the Leland Stanford Junior University $10,927
- Baylor College of Medicine $5,066
- Cincinnati Children's Hospital Medical Center, OH $2,800
- Duke University Medical Center $2,480
- Ronald Reagan UCLA Medical Center, CA $2,409
- Seattle Children's Hospital, WA $541
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.