A Phase 1/2 interventional study of mRNA-3705 in Methylmalonic Acidemia, sponsored by ModernaTX, Inc.. Recruiting at 12 sites in 6 countries. Open to participants aged 1 Year and older. Per ClinicalTrials.gov, last updated 2025-12-23.
Sponsored by ModernaTX, Inc. (part of Moderna, Inc.) · Phase 1/2, Interventional, and Treatment
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.
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.
26 studies on the registry are indexed under Methylmalonic acidemia; 7 are open to participants now.
This study's planned enrollment of 56 is above the median of 23 across 12 interventional studies indexed under Methylmalonic acidemia.
Browse Methylmalonic acidemia studies →ModernaTX, Inc. is the lead sponsor of 119 studies on the registry; 18 are open to participants now.
Of its 64 completed or terminated interventional studies of FDA-regulated products, 35 (55%) have results posted.
Counted across the registry records on this site, refreshed daily.
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Exclusion Criteria:
NOTE: Other inclusion and exclusion criteria may apply.
Participants will receive mRNA-3705 at the same dose levels at the same dosing interval (every 2 weeks \[Q2W\], or every 3 weeks \[Q3W\]) last received in the clinical study of mRNA-3705 in which they initially participated, unless the Sponsor recommends modification.
Drug: mRNA-3705
A sterile liquid for injection
Also known as: modified mRNA encoding human, methylmalonyl-coenzyme A mutase
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)
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)
Pre- and Postdose Human Methylmalonyl-Coenzyme A Mutase (hMUT) mRNA and SM-86 Levels
Time frame: Baseline up to 6 years
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)
Number of Healthcare Resource Utilization Visits
Time frame: Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up)
Number of Annualized MMA-related Hospitalizations
Time frame: Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up)
Number of Annualized MMA-related Healthcare Visits
Time frame: Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up)
Number of Anti-Polyethylene Glycol (PEG) and Anti-hMUT Antibodies
Time frame: Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up)
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)
Change in Investigator Global Assessment of Improvement (IGA-I) Score
Time frame: Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up)
Change in Investigator Global Assessment of Severity (IGA-S) Score
Time frame: Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up)
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)
Not enrolling at this site.
Plan to share: Undecided
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