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A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia

NCT04899310 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2026-05-06

No results posted yet for this study

Summary

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.

Conditions

  • Methylmalonic Acidemia

Interventions

BIOLOGICAL

mRNA-3705

Sterile liquid for infusion

BIOLOGICAL

Placebo

Sterile liquid for infusion

Sponsors & Collaborators

  • ModernaTX, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
1 Year
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-08-06
Primary Completion
2026-04-01
Completion
2026-04-01
FDA Drug
Yes

Countries

  • United States
  • Canada
  • France
  • Netherlands
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04899310 on ClinicalTrials.gov