Long-term evaluation of mRNA-3705 for treating methylmalonic acidemia
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
This study is testing if a new treatment called mRNA-3705 is safe and effective for people with methylmalonic acidemia who have already tried it in previous studies.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 56 (estimated) |
| Ages | 1 Year and up |
| Sex | All |
| Sponsor | ModernaTX, Inc. Industry-sponsored |
| Locations | 12 sites (Los Angeles, California and 11 other locations) |
| Trial ID | NCT05295433 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the long-term safety and clinical effectiveness of mRNA-3705 in patients with isolated methylmalonic acidemia (MMA) who have previously participated in other studies involving this treatment. Participants will undergo a Treatment Period followed by a Follow-up Period lasting up to two years after their last dose. The study will monitor safety and clinical outcomes, with the possibility of transitioning to market access if mRNA-3705 receives approval. The goal is to ensure continuous treatment without interruption for eligible participants.
Who should consider this trial
Good fit: Ideal candidates are individuals with isolated methylmalonic acidemia due to methylmalonyl-coenzyme A mutase deficiency who have previously participated in mRNA-3705 studies.
Not a fit: Patients who are not expected to receive clinical benefit from continued mRNA-3705 administration, as determined by the investigator, will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a long-term treatment option for patients with methylmalonic acidemia, potentially improving their health outcomes.
How similar studies have performed: Other studies involving mRNA therapies have shown promise, indicating potential for success with this approach, although this specific extension study is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Los Angeles, California and 11 other locations
- UCLA Medical Center — Los Angeles, California, United States (Recruiting)
- Lucile Packard Children's Hospital at Stanford — Palo Alto, California, United States (Recruiting)
- Altman Clinical and Translational Research Institution — San Diego, California, United States (Recruiting)
- Stollery Children's Hospital University of Alberta — Edmonton, Alberta, Canada (Recruiting)
- Hospital For Sick Children — Toronto, Ontario, Canada (Recruiting)
- Hôpital Necker - Enfants Malades APHP — Paris, France (Recruiting)
- Erasmus MC — Rotterdam, Netherlands (Recruiting)
- Universitair Medisch Centrum Utrecht — Utrecht, Netherlands (Recruiting)
- Hospital Universitario Cruces — Barakaldo, Vizcaya, Spain (Recruiting)
- Hospital Universitario 12 de Octubre — Madrid, Spain (Recruiting)
- Birmingham Children's Hospital NHS Foundation Trust — Birmingham, United Kingdom (Terminated)
- Royal Manchester Childrens Hospital — Manchester, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Moderna WeCare Team
- Email: WeCareClinicalTrials@modernatx.com
- Phone: 1-866-663-3762
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.