Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
Condition(s) studied
Investigational drug(s) / intervention(s)
mRNA-3927: mRNA-3927 dispersion for IV infusion
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).
Eligibility
Primary outcome measure(s)
- Part 1: Number of Participants with Treatment-emergent Adverse Event (TEAE), Serious Adverse Events (SAE) and TEAEs Leading to Discontinuation — Day 1 (initial mRNA-3927 dose) up to Week 150 (End of Study)
- Part 2: Change in Annualized Frequency of Clinical Event Committee (CEC)-adjudicated Metabolic Decompensation Events (MDEs) During 12-month Treatment Period With mRNA-3927 Compared to Annualized Frequency of CEC-adjudicated MDE During Pretreatment Period — Pretreatment period (12 months before consent to first mRNA-3927 dose in the study) up to Month 12
- Part 3: Number of Participants with TEAEs, SAEs, Adverse Events (AEs) of Special Interest (AESIs) and TEAEs Leading to Discontinuation — Day 1 up to Week 73
Trial sites (35)
| Facility | City | Region | Status |
|---|---|---|---|
| UCSD Altman Clinical and Transalational Research Institute Building | Los Angeles | California | Not Yet Recruiting |
| Ronald Reagan UCLA Medical Center | Los Angeles | California | Recruiting |
| Lucile Packard Children's Hospital Stanford | Stanford | California | Recruiting |
| Nicklaus Children's Hospital | Miami | Florida | Not Yet Recruiting |
| University of South Florida - 12901 Bruce B Downs | Tampa | Florida | Not Yet Recruiting |
| Ann and Robert H Lurie Childrens Hospital of Chicago | Chicago | Illinois | Recruiting |
| Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit | Baltimore | Maryland | Completed |
| Boston Children's Hospital | Boston | Massachusetts | Completed |
| University of Michigan Hospitals | Ann Arbor | Michigan | Recruiting |
| Icahn School of Medicine at Mount Sinai - Clinical Research Unit | New York | New York | Recruiting |
| Duke University Medical System (Duke Health) | Durham | North Carolina | Recruiting |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | Completed |
| University Hospitals Cleveland Medical Center - 11100 Euclid Ave | Cleveland | Ohio | Active Not Recruiting |
| Children's Hospital of Philadelphia (CHOP) | Philadelphia | Pennsylvania | Recruiting |
| Texas Children's Hospital | Houston | Texas | Recruiting |
| Stollery Children's Hospital University of Alberta | Edmonton | Alberta | Recruiting |
| Hospital For Sick Children | Toronto | Ontario | Recruiting |
| CHU de Marseille - Hôpital de la Timone | Marseille | France | Active Not Recruiting |
| Hôpital Necker - Enfants Malades | Paris | France | Recruiting |
| Fujita Health University Hospital | Toyoake-shi | Aichi-ken | Recruiting |
| Tohoku University Hospital | Sendai | Miyagi | Recruiting |
| National Center for Child Health and Development | Tokyo | Japan | Active Not Recruiting |
| Erasmus MC | Rotterdam | South Holland | Recruiting |
| Universitair Medisch Centrum Utrecht - PPDS | Utrecht | Netherlands | Active Not Recruiting |
| King Faisal Specialist Hospital & Research Center - Riyadh | Riyadh | Ar Riya | Active Not Recruiting |
| King Fahad Medical City | Riyadh | Ar Riya | Not Yet Recruiting |
| King Abdullah Children's Specialist Hospital | Riyadh | Ar Riya | Not Yet Recruiting |
| Hospital Sant Joan de Deu - PIN | Esplugues de Llobregat | Barcelona | Active Not Recruiting |
| Hospital Universitario Cruces | Barakaldo | Biscay | Recruiting |
| Hospital Universitario 12 de Octubre | Madrid | Spain | Recruiting |
| Hospital Universitario Virgen del Rocio - PPDS | Seville | Spain | Active Not Recruiting |
| University Hospital Birmingham NHS Foundation Trust | Birmingham | United Kingdom | Recruiting |
| Birmingham Children's Hospital | Birmingham | United Kingdom | Completed |
| Great Ormond Street Hospital for Children NHS Foundation Trust | London | United Kingdom | Recruiting |
| Willink Biochemical Genetics Unit - PPDS | Manchester | United Kingdom | Recruiting |
More ModernaTX, Inc. trials in Japan
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04159103 on ClinicalTrials.gov ↗ ← All trials in Japan