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.
Eligibility
Sex
ALL
Min age
1 Year
Max age
—
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 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.
Primary outcome measure(s)
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 — Baseline up to follow-up period (up to 6 years + 6 months)
Trial sites (12)
Facility
City
Region
Status
UCLA Medical Center
Los Angeles
California
Recruiting
Lucile Packard Children's Hospital at Stanford
Palo Alto
California
Recruiting
Altman Clinical and Translational Research Institution
San Diego
California
Recruiting
Stollery Children's Hospital University of Alberta
Edmonton
Alberta
Recruiting
Hospital For Sick Children
Toronto
Ontario
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
Recruiting
Hospital Universitario 12 de Octubre
Madrid
Spain
Recruiting
Birmingham Children's Hospital NHS Foundation Trust
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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