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

Last updated: December 22, 2025
Sponsor: ModernaTX, Inc.
Overall Status: Active - Recruiting

Phase

1/2

Condition

N/A

Treatment

mRNA-3705

Clinical Study ID

NCT05295433
mRNA-3705-P101-EXT
2022-501997-20-00
2021-000446-17
  • Ages > 1
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Completed the assigned dose regimen treatment time period in other clinical studiesof mRNA-3705 or is eligible for early transition to this study because they missedmore 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 ofunscheduled dosing) in Study mRNA-3705-P101 within 10 days of their first dose ofmRNA-3705 in this extension study.

Exclusion

Exclusion Criteria:

  • Not expected to receive clinical benefit from continued mRNA-3705 administration, inthe opinion of the Investigator.

  • Any clinical or laboratory abnormality or medical condition that, at the discretionof the Investigator, may put the individual at increased risk by participating inthis study.

  • History of liver and/or kidney transplant.

NOTE: Other inclusion and exclusion criteria may apply.

Study Design

Total Participants: 56
Treatment Group(s): 1
Primary Treatment: mRNA-3705
Phase: 1/2
Study Start date:
March 08, 2022
Estimated Completion Date:
April 02, 2034

Study 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 eligibility criteria have been met. The study will include 2 periods:

  1. Treatment Period and 2) Follow-up Period (up to 6 months after the last dose of study drug).

Treatment Period will continue unless one of the following occurs: mRNA-3705 receives marketing approval and reimbursement in the country of origin of the participant (following market approval and access being in place, all participants who wish to continue treatment will be offered mRNA-3705 through market access, with the intention of preventing treatment interruption. Other appropriate access program is offered as allowed by local regulation to continue treatment with mRNA-3705. Safety monitoring will be performed for all participants under treatment per market access requirements), the participant discontinues study drug, the participant is no longer receiving clinical benefit in the opinion of the Investigator, or Sponsor discontinues the development of mRNA-3705.

Connect with a study center

  • Stollery Children's Hospital University of Alberta

    Edmonton, Alberta T6G 2R7
    Canada

    Site Not Available

  • Stollery Children's Hospital University of Alberta

    Edmonton 5946768, Alberta 5883102 T6G 2R7
    Canada

    Active - Recruiting

  • Hospital For Sick Children

    Toronto, Ontario M5G 1X8
    Canada

    Site Not Available

  • Hospital For Sick Children

    Toronto 6167865, Ontario 6093943 M5G 1X8
    Canada

    Active - Recruiting

  • Hôpital Necker - Enfants Malades APHP

    Paris, 75015
    France

    Site Not Available

  • Hôpital Necker - Enfants Malades APHP

    Paris 2988507, 75015
    France

    Active - Recruiting

  • Erasmus MC

    Rotterdam, 3015 AA
    Netherlands

    Site Not Available

  • Erasmus MC

    Rotterdam 2747891, 3015 AA
    Netherlands

    Active - Recruiting

  • Universitair Medisch Centrum Utrecht

    Utrecht, 3584 CX
    Netherlands

    Site Not Available

  • Universitair Medisch Centrum Utrecht

    Utrecht 2745912, 3584 CX
    Netherlands

    Active - Recruiting

  • Hospital Universitario Cruces

    Barakaldo, Vizcaya 48903
    Spain

    Site Not Available

  • Hospital Universitario Cruces

    Barakaldo 3109453, Vizcaya 48903
    Spain

    Active - Recruiting

  • Hospital Universitario 12 de Octubre

    Madrid, 28041
    Spain

    Site Not Available

  • Hospital Universitario 12 de Octubre

    Madrid 3117735, 28041
    Spain

    Active - Recruiting

  • Birmingham Children's Hospital NHS Foundation Trust

    Birmingham, B4 6NH
    United Kingdom

    Site Not Available

  • Birmingham Children's Hospital NHS Foundation Trust

    Birmingham 2655603, B4 6NH
    United Kingdom

    Terminated

  • Royal Manchester Childrens Hospital

    Manchester, M13 9WL
    United Kingdom

    Site Not Available

  • Royal Manchester Childrens Hospital

    Manchester 2643123, M13 9WL
    United Kingdom

    Active - Recruiting

  • UCLA Medical Center

    Los Angeles, California 90095
    United States

    Site Not Available

  • Lucile Packard Children's Hospital at Stanford

    Palo Alto, California 94304
    United States

    Site Not Available

  • Altman Clinical and Translational Research Institution

    San Diego, California 92037
    United States

    Site Not Available

  • UCLA Medical Center

    Los Angeles 5368361, California 5332921 90095
    United States

    Active - Recruiting

  • Lucile Packard Children's Hospital at Stanford

    Palo Alto 5380748, California 5332921 94304
    United States

    Active - Recruiting

  • Altman Clinical and Translational Research Institution

    San Diego 5391811, California 5332921 92037
    United States

    Active - Recruiting

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