Trials / Active Not Recruiting

Active Not RecruitingNCT04899310

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

A Global Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency

Status: Active Not Recruiting
Phase: Phase 1 / Phase 2
Study type: Interventional
Enrollment: 74 (estimated)

Sponsor: ModernaTX, Inc. · Industry
Sex: All
Age: 1 Year
Healthy volunteers: Not accepted

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.

Detailed description

This study comprises 3 parts and is designed to evaluate multiple doses and dosing intervals of mRNA-3705. Parts 1 and 3 are designed to characterize the safety, tolerability, and pharmacological activity of mRNA-3705 administered via intravenous infusion to participants with isolated MMA due to MUT deficiency. Part 2 will evaluate the efficacy of mRNA-3705 as assessed by the change in plasma methylmalonic acid levels. Participants who complete the treatment period in any part of the study, including the end of treatment (EOT) visit, will be offered participation in the mRNA-extension study (mRNA-3705-P101-EXT; NCT05295433) or may transition to the follow-up period of the study. All participants, including those randomized to placebo in Part 2, will receive mRNA-3705 in the extension study.

Conditions

Methylmalonic Acidemia

Interventions

Type	Name	Description
BIOLOGICAL	mRNA-3705	Sterile liquid for infusion
BIOLOGICAL	Placebo	Sterile liquid for infusion

Timeline

Start date: 2021-08-06
Primary completion: 2028-07-01
Completion: 2028-07-01
First posted: 2021-05-24
Last updated: 2026-01-20

Locations

11 sites across 6 countries: United States, Canada, France, Netherlands, Spain, United Kingdom

Regulatory

FDA-regulated drug study

Source: ClinicalTrials.gov record NCT04899310. Inclusion in this directory is not an endorsement.