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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

NCT ID: NCT05295433

Last Updated: 2025-12-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

56 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-03-08

Study Completion Date

2034-04-02

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Methylmalonic Acidemia

Keywords

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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

mRNA-3705

Intervention Type DRUG

A sterile liquid for injection

Interventions

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mRNA-3705

A sterile liquid for injection

Intervention Type DRUG

Other Intervention Names

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modified mRNA encoding human methylmalonyl-coenzyme A mutase

Eligibility Criteria

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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.
Minimum Eligible Age

1 Year

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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ModernaTX, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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UCLA Medical Center

Los Angeles, California, United States

Site Status RECRUITING

Lucile Packard Children's Hospital at Stanford

Palo Alto, California, United States

Site Status RECRUITING

Altman Clinical and Translational Research Institution

San Diego, California, United States

Site Status RECRUITING

Stollery Children's Hospital University of Alberta

Edmonton, Alberta, Canada

Site Status RECRUITING

Hospital For Sick Children

Toronto, Ontario, Canada

Site Status RECRUITING

Hôpital Necker - Enfants Malades APHP

Paris, , France

Site Status RECRUITING

Erasmus MC

Rotterdam, , Netherlands

Site Status RECRUITING

Universitair Medisch Centrum Utrecht

Utrecht, , Netherlands

Site Status RECRUITING

Hospital Universitario Cruces

Barakaldo, Vizcaya, Spain

Site Status RECRUITING

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status RECRUITING

Birmingham Children's Hospital NHS Foundation Trust

Birmingham, , United Kingdom

Site Status TERMINATED

Royal Manchester Childrens Hospital

Manchester, , United Kingdom

Site Status RECRUITING

Countries

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United States Canada France Netherlands Spain United Kingdom

Central Contacts

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Moderna WeCare Team

Role: CONTACT

Phone: 1-866-663-3762

Email: [email protected]

Facility Contacts

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Monserrath Campos

Role: primary

Thu Quan

Role: primary

Nicolle Mendez

Role: primary

Cheri Copithorne

Role: primary

Preet Dhillon

Role: primary

Michaela Semeraro

Role: primary

Role: primary

Role: primary

Ohiane Martin Rodriguez

Role: primary

Ianire Gallego Amaro

Role: primary

Laura Crowther

Role: primary

Other Identifiers

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2021-000446-17

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2022-501997-20-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

mRNA-3705-P101-EXT

Identifier Type: -

Identifier Source: org_study_id