Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
Recruiting
Phase 1
Phase 2
Interventional Study
Propionic Acidemia
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 77 (estimated)
- Sponsor
- ModernaTX, Inc. · Industry
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About This Trial
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, par…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
Participants ≥1 year of age are eligible to be included in the study only if all of the following criteria apply:
* ≥ 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants in Part 1.
* ≥1 year of age at the time of consent/assent if enrolled …
Contacts