Condition: Propionic Acidemia · Sponsor: ModernaTX, Inc.
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).
This description comes directly from the study's public registry record.
Moderna WeCare Team · 1-866-663-3762 · WeCareClinicalTrials@modernatx.com
Always discuss trial participation with your own doctor first.
| UCSD Altman Clinical and Transalational Research Institute Building | Los Angeles, California, United States | Not Yet Recruiting |
| Ronald Reagan UCLA Medical Center | Los Angeles, California, United States | Recruiting |
| Lucile Packard Children's Hospital Stanford | Stanford, California, United States | Recruiting |
| Nicklaus Children's Hospital | Miami, Florida, United States | Not Yet Recruiting |
| University of South Florida - 12901 Bruce B Downs | Tampa, Florida, United States | Not Yet Recruiting |
| Ann and Robert H Lurie Childrens Hospital of Chicago | Chicago, Illinois, United States | Recruiting |
| Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit | Baltimore, Maryland, United States | Completed |
| Boston Children's Hospital | Boston, Massachusetts, United States | Completed |
| University of Michigan Hospitals | Ann Arbor, Michigan, United States | Recruiting |
| Icahn School of Medicine at Mount Sinai - Clinical Research Unit | New York, New York, United States | Recruiting |
| Duke University Medical System (Duke Health) | Durham, North Carolina, United States | Recruiting |
| Cincinnati Children's Hospital Medical Center | Cincinnati, Ohio, United States | Completed |
+ 23 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT04159103