Recruiting
Phase 1
Phase 2

mRNA-3705

Sponsor:

Moderna

Code:

NCT05295433

Conditions

Methylmalonic Acidemia

Eligibility Criteria

Sex: All

Age: 1+

Healthy Volunteers: Not accepted

Interventions

mRNA-3705

Study Details

Brief summary:

The primary objective of this study is to evaluate the long-term safety and clinical activity of mRNA-3705 administered to participants with isolated methylmalonic acidemia (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency who have previously participated in other clinical studies of mRNA-3705.

Conditions

Methylmalonic Acidemia

Study ID

NCT05295433

Start date

Mar 8, 2022

Status verified date

Nov, 2025

Completion date

Apr 2, 2034

Anticipated

Primary completion date

Apr 2, 2034

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 1+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Completed the assigned dose regimen treatment time period in other clinical studies of mRNA-3705 or is eligible for early transition to this study because they missed more than 3 consecutive doses of study drug due to coronavirus disease 2019 (COVID-19) vaccination during Study mRNA-3705-P101 Part 1.
  • Completed the End of treatment (EOT) Visit (or End of Study Visit in the case of unscheduled dosing) in Study mRNA-3705-P101 within 10 days of their first dose of mRNA-3705 in this extension study.

Exclusion Criteria:

  • Not expected to receive clinical benefit from continued mRNA-3705 administration, in the opinion of the Investigator.
  • Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the individual at increased risk by participating in this study.
  • History of liver and/or kidney transplant.

NOTE: Other inclusion and exclusion criteria may apply.

Study Design

Enrollment

56 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: mRNA-3705

Participants will receive mRNA-3705 at the same dose levels at the same dosing interval (every 2 weeks \[Q2W\], or every 3 weeks \[Q3W\]) last received in the clinical study of mRNA-3705 in which they initially participated, unless the Sponsor recommends modification.

Interventions

mRNA-3705

A sterile liquid for injection

Primary outcome measure

  • Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Drug-Related TEAEs, Unrelated TEAEs, Adverse Events of Special Interests (AESIs), Serious Adverse Events (SAEs) and TEAEs Leading to Discontinuation [ Time Frame: Baseline up to follow-up period (up to 6 years + 6 months) ]

Central Contacts and Locations

Central contacts

Moderna WeCare Team

1-866-663-3762WeCareClinicalTrials@modernatx.com

Locations

UCLA Medical Center

Recruiting

Los Angeles, California, United States, 90095

Contacts

Monserrath Campos

monserrathcampos@mednet.ucla.edu

Lucile Packard Children's Hospital at Stanford

Recruiting

Palo Alto, California, United States, 94304

Contacts

Thu Quan

tquan@stanford.edu

Altman Clinical and Translational Research Institution

Recruiting

San Diego, California, United States, 92037

Contacts

Nicolle Mendez

n3mendez@health.ucsd.edu

Stollery Children's Hospital University of Alberta

Recruiting

Edmonton, Alberta, Canada, T6G 2R7

Contacts

Cheri Copithorne

cheri.robert@ahs.ca

Hospital For Sick Children

Recruiting

Toronto, Ontario, Canada, M5G 1X8

Contacts

Preet Dhillon

preet.dhillon@sickkids.ca

More Information

Sponsor

ModernaTX, Inc.

Last update posted

Dec 23, 2025

Last verified

Nov, 2025

Keywords

  • Isolated Methylmalonic acidemia
  • Isolated methylmalonic aciduria
  • Elevated methylmalonic acid (MMA)
  • Metabolism, Inborn Errors
  • Genetic Diseases
  • Moderna
  • mRNA
  • mRNA-3705

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-09. This information was provided to ClinicalTrials.gov by ModernaTX, Inc. on 2025-12-23. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.