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
ModernaTX, Inc.
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.
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…
Eligibility
- Age range
- 1+ years
- Sex
- All
- Healthy volunteers
- No
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 conti…
Interventions
- DrugmRNA-3705
A sterile liquid for injection
Locations (12)
- UCLA Medical CenterLos Angeles, California
- Lucile Packard Children's Hospital at StanfordPalo Alto, California
- Altman Clinical and Translational Research InstitutionSan Diego, California
- Stollery Children's Hospital University of AlbertaEdmonton, Alberta
- Hospital For Sick ChildrenToronto, Ontario
- Hôpital Necker - Enfants Malades APHPParis