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Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
NCT04159103 · View on ClinicalTrials.gov ↗
Study Summary
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).
Conditions Studied
Interventions
- BIOLOGICAL mRNA-3927
Study Locations (20)
California
- UCSD Altman Clinical and Transalational Research Institute Building — Los Angeles
- Ronald Reagan UCLA Medical Center — Los Angeles
- Lucile Packard Children's Hospital Stanford — Stanford
Florida
- Nicklaus Children's Hospital — Miami
- University of South Florida - 12901 Bruce B Downs — Tampa
Ohio
- Cincinnati Children's Hospital Medical Center — Cincinnati
- University Hospitals Cleveland Medical Center - 11100 Euclid Ave — Cleveland
Other
- CHU de Marseille - Hôpital de la Timone — Marseille
- Hôpital Necker - Enfants Malades — Paris
Illinois
- Ann and Robert H Lurie Childrens Hospital of Chicago — Chicago
Maryland
- Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit — Baltimore
Massachusetts
- Boston Children's Hospital — Boston
Michigan
- University of Michigan Hospitals — Ann Arbor
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 77 participants |
| Start Date | 2021-04-15 |
| Est. Completion | 2027-08-31 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04159103
The ClinicalTrials.gov registry entry for NCT04159103 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 77 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is ModernaTX, which has 67 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Propionic Acidemia appearing as the primary indexed condition, and to 1 intervention — of which mRNA-3927 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04159103 reports 20 study locations spanning 15 distinct geographic areas — top geographies include California, Florida, Ohio. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04159103 about?
NCT04159103 is a clinical study titled "Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia". 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 a...
What is the current status of trial NCT04159103?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 77 participants. The study started on 2021-04-15. Estimated completion is 2027-08-31.
What conditions does trial NCT04159103 study?
This clinical trial studies the following conditions: Propionic Acidemia. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04159103?
The interventions under investigation include: mRNA-3927 (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04159103?
This trial is sponsored by ModernaTX, which has 67 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04159103 being conducted?
This trial has 20 study locations across California, Florida, Illinois, Maryland, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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