A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia
Terminated · Phase 1, Phase 2 · NCT04899310
Conditions: Methylmalonic Acidemia
At a glance
- Research payments reported, 2025
- $167,012
- U.S. investigators named
- 3
- Sites paid
- 3
- Planned or actual enrollment
- 18
About the trial
- Sponsor
- ModernaTX, Inc.
- Tested
- mRNA-3705 (biological), Placebo (biological)
- Dates
- Started 2021-08-06; completion 2026-04-01
- Results posted
- Not yet
This is a study of mRNA-3705 in participants with isolated elevated methylmalonic acid (MMA) due to methylmalonyl-coenzyme A (CoA) mutase (MUT) deficiency. The main goal of the study is to assess safety, efficacy, pharmacokinetics, and pharmacodynamics of intravenously (IV)-infused mRNA-3705.
From ClinicalTrials.gov, last updated 2026-05-06. Full record, eligibility and contacts (opens in a new tab)
Industry funding
ModernaTX, Inc. reported $167,012 in research payments for this study in 2025 (21 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Gregory M. Enns, M.D. Medical Genetics · Palo Alto, CA
- Rebecca Mardach, MD Medical Genetics · San Diego, CA
- Can H. Ficicioglu, M.D. Medical Genetics · Philadelphia, PA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Children's Hospital of Philadelphia, PA $80,833
- University of California Davis Medical Center, CA $65,922
- Stanford University $20,258
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.