Long-Term Safety of mRNA-3705 for Methylmalonic Acidemia
Trial Summary
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.
What data supports the effectiveness of the drug mRNA-3705 for treating methylmalonic acidemia?
What safety data exists for mRNA-3705 treatment in humans?
How is the drug mRNA-3705 different from other treatments for methylmalonic acidemia?
mRNA-3705 is unique because it uses a synthetic messenger RNA (mRNA) to produce the enzyme methylmalonyl-CoA mutase, which is often missing or defective in people with methylmalonic acidemia. Unlike traditional treatments, it is delivered intravenously (through a vein) using lipid nanoparticles, which helps the body produce the necessary enzyme without the risks associated with gene therapy, such as immune reactions or genetic damage.12457
What is the purpose of this trial?
The primary objective of this study is to evaluate the long-term safety 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
This trial is for people with Methylmalonic Acidemia who were in a previous study (mRNA-3705-P101). They must have finished the earlier trial or moved to this one because they missed doses due to COVID-19 vaccination.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive mRNA-3705 at the same dose levels and intervals as in previous studies, unless modified by the Sponsor
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants may continue treatment long-term if mRNA-3705 receives market approval and reimbursement
Treatment Details
Interventions
- mRNA-3705
mRNA-3705 is already approved in United States for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
ModernaTX, Inc.
Lead Sponsor
Dr. Stephen Hoge
ModernaTX, Inc.
Chief Medical Officer
MD from Harvard Medical School
Stéphane Bancel
ModernaTX, Inc.
Chief Executive Officer since 2011
MBA from Harvard Business School, MSc in Engineering from École Centrale Paris