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Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

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Brief Summary

This First-in-Human (FIH) Phase 1/2 study will evaluate the safety, pharmacological activity, and efficacy of mRNA-3927 in participants 1 year of age and older with genetically confirmed propionic acidemia (PA). This study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the optimal dose of mRNA-3927 in participants with PA. After establishing a dose with acceptable safety and pharmacodynamic (PD) response in a Dose Optimization Part (Part 1), additional participants will be enrolled into the study in a Dose Expansion Part (Part 2) to allow for further characterization of the efficacy, safety, and PD of mRNA-3927.

Primary Purpose
Treatment
Study Type
Interventional
Phase
Phase I/II

Eligibility

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

Inclusion Criteria:

  • Participant must be ≥ 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants in Part 1.
  • Participant must be ≥1 year of age at the time of consent/assent if enrolled after the first 2 participants.
  • Confirmed diagnosis of PA based on diagnosis by molecular genetic testing (PCCA and/or PCCB mutations).
  • Part 2 only: At least one documented MDE in the 12-month period before consent.

Exclusion Criteria:

  • Estimated glomerular filtration rate <30 milliliters (mL)/minute/1.73 square meter (m^2) as estimated by Schwartz formula for participants < 18 years of age or the Chronic Kidney Disease Epidemiology Collaboration creatinine based formula for participants ≥ 18 years of age or for participants of all ages receiving chronic dialysis.
  • History of organ transplantation or planned organ transplantation during the period of study participation.
  • Corrected QT interval (QTc) >480 milliseconds (ms) using Bazett's correction.
  • Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification.
  • COVID-19 vaccination (generally 2 doses or a booster) within 6 weeks between their last COVID-19 vaccination dose and first study drug administration.

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Study Stats
Protocol No.
19-002009
Category
Endocrine and Metabolic Disorders
Pediatric and Prenatal Disorders
Location
  • UCLA Westwood
For Providers
NCT No.
NCT04159103
For detailed technical eligibility, visit ClinicalTrials.gov.