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A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia

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Brief Summary

This is a study of mRNA-3705 in participants with isolated elevated methylmalonic acid (MMA) due to methylmalonyl-coenzyme A (CoA) mutase (MUT) deficiency. The main goal of the study is to assess safety, pharmacokinetics, and pharmacodynamics of mRNA-3705.

Primary Purpose
Treatment
Study Type
Interventional
Phase
Phase I/II

Eligibility

Gender
All
Healthy Volunteers
No
Minimum Age
1 Year
Maximum Age
N/A

Key Inclusion Criteria:

  • Participant has a body weight of ≥11.0 kilograms (kg) at the Screening Visit.
  • Participant has a diagnosis of isolated MMA due to MUT deficiency confirmed by molecular genetic testing.
  • Participant has a serum/plasma vitamin B12 level equal to or above the lower limit of normal (based on laboratory reference range) confirmed in the Screening Period. For those participants found to have an elevated serum/plasma vitamin B12 level, the participant may enter if, in the opinion of the Investigator, the cause of the elevation is secondary to B12 supplementation.
  • Participant or his/her legally authorized representative is willing and able to provide informed consent and/or assent as mandated by local regulations and willing and able to comply with study-related assessments.
  • Sexually active females of childbearing potential and sexually active males of reproductive potential agree to use a highly-effective method of contraception during the study and for 3 months after the last administration of study drug.

Key Exclusion Criteria:

  • Participant has a diagnosis of isolated MMA cb1A, cb1B, or cb1D enzymatic subtypes or methylmalonyl-CoA epimerase deficiency or combined MMA with homocystinuria.
  • Participant has previously received gene therapy for the treatment of MMA.
  • Participant has a history of organ transplantation.
  • Participant has an active, unstable, or clinically significant medical condition not related to MMA or history of noncompliance that, in the Investigator's opinion, could potentiate the risk while participating in this study, interfere with the interpretation of study results, or limit the participant's participation in the study. This may include, but is not limited to, history of relevant food or drug allergies; history of cardiovascular, central nervous, gastrointestinal, or infectious disease; history of clinically significant pathology; and/or history of cancer.

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Study Stats
Protocol No.
22-000935
Category
Surgery
Contact
Emma Raffman
Location
  • UCLA Westwood
For Providers
NCT No.
NCT04899310
For detailed technical eligibility, visit ClinicalTrials.gov.