Translational Pharmacokinetic/Pharmacodynamic Model for mRNA-3927, an Investigational Therapeutic for the Treatment of Propionic Acidemia.
Husain Attarwala, Matthew Lumley, Min Liang...
https://pubmed.ncbi.nlm.nih.gov/36577040/Actively Recruiting
Phase 1
Phase 2
All Genders
ID04159103
Phase 12 Study to Evaluate Safety and Effects of mRNA-3927 in People with Propionic Acidemia Including Infants and Children
Led by ModernaTX, Inc. · Updated on 2026-01-22
77
Participants Needed
35
Research Sites
N/A
Total Duration
AI-Summary
What this Trial Is About
Researchers are studying mRNA-3927, a biological treatment, in people with propionic acidemia PA, a rare genetic condition. This Phase 12 open-label study aims to evaluate the safety, tolerability, and how the drug works in the body by measuring biomarkers. The study is divided into three parts Part 1 focuses on finding the right dose for participants aged 1 year and older, Part 2 assesses the treatments effects in participants aged 1 year and older, and Part 3 studies infants under 1 year old.
CONDITIONS
Brief Title
Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
Research Team
M
Moderna WeCare Team
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