Status:

RECRUITING

An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705

Lead Sponsor:

ModernaTX, Inc.

Conditions:

Methylmalonic Acidemia

Eligibility:

All Genders

1+ years

Phase:

PHASE1

PHASE2

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-...

Detailed Description

Participants with isolated MMA due to MUT deficiency who were previously enrolled in other clinical studies of mRNA-3705 will have the option to enroll into this extension study provided all eligibili...

Eligibility Criteria

Inclusion

  • 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

  • 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.

Key Trial Info

Start Date :

March 8 2022

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

April 2 2034

Estimated Enrollment :

56 Patients enrolled

Trial Details

Trial ID

NCT05295433

Start Date

March 8 2022

End Date

April 2 2034

Last Update

December 23 2025

Active Locations (12)

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Page 1 of 3 (12 locations)

1

UCLA Medical Center

Los Angeles, California, United States, 90095

2

Lucile Packard Children's Hospital at Stanford

Palo Alto, California, United States, 94304

3

Altman Clinical and Translational Research Institution

San Diego, California, United States, 92037

4

Stollery Children's Hospital University of Alberta

Edmonton, Alberta, Canada, T6G 2R7