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NCT Number: NCT05295433

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

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.

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Key information

Conditions

Methylmalonic Acidemia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

1 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Stollery Children's Hospital University of Alberta, Edmonton, Alberta, Canada

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

mRNA-3705

Drug

A sterile liquid for injection

Other names: modified mRNA encoding human, methylmalonyl-coenzyme A mutase

Primary outcomes

  1. 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)

Secondary outcomes

  1. Percent Change in Plasma Methylmalonic Acid and 2-Methylcitric Acid (2-MC) Levels (Primary Biomarkers) From Baseline up to 18 Months

    Time frame: Baseline, Year 6 (Treatment Period) + Month 6 (Follow up)

  2. Pre- and Postdose Human Methylmalonyl-Coenzyme A Mutase (hMUT) mRNA and SM-86 Levels

    Time frame: Baseline up to 6 years

  3. Change From Baseline in Pretreatment and Post-treatment Annualized Metabolic Decompensation Events (MDEs) Rate

    Time frame: Baseline, Year 6 (Treatment Period) + Month 6 (Follow up)

  4. Number of Healthcare Resource Utilization Visits

    Time frame: Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up)

  5. Number of Annualized MMA-related Hospitalizations

    Time frame: Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up)

  6. Number of Annualized MMA-related Healthcare Visits

    Time frame: Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up)

  7. Number of Anti-Polyethylene Glycol (PEG) and Anti-hMUT Antibodies

    Time frame: Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up)

  8. Change in Health-Related Quality of Life (HRQoL) as Measured Using the Pediatric Quality of Life Inventory (PedsQL™) at Month 3 up to 18 Months

    Time frame: Month 3, Year 6 (Treatment Period) + Month 6 (Follow up)

  9. Change in Investigator Global Assessment of Improvement (IGA-I) Score

    Time frame: Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up)

  10. Change in Investigator Global Assessment of Severity (IGA-S) Score

    Time frame: Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up)

  11. Change in EuroQoL 5-Dimensions 5-level/Youth Questionnaire (EQ-5D-5L/Y) Score

    Time frame: Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up)

Study contacts

Contact information is provided by the study sponsor or research team.

Moderna WeCare Team

CONTACT

[email protected]

1-866-663-3762

Sponsors and collaborators

Lead sponsor

ModernaTX, Inc.

Industry

Registry information

Official study title

A Phase 1/2, Global, Open-Label, 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

Important dates

Study start
2022
Primary completion
2034
Study completion
2034
First posted
Mar 25, 2022
Registry last updated
Dec 23, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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