Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
ConditionPropionic Acidemia
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorModernaTX, Inc.
About this trial
This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination of the efficacy, safety, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (\<1 year of age).
Eligibility criteria
Qualifiers
≥ 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants in Part 1.
≥1 year of age at the time of consent/assent if enrolled after the first 2 participants in Part 1.
Confirmed diagnosis of PA based on diagnosis by molecular genetic testing via central laboratory (PCCA and/or PCCB mutations).
Part 2 only: At least one documented MDE in the 12-month period before consent.
Disqualifiers
Any individual with laboratory abnormalities considered to be clinically significant (for example, markedly out of range, associated with clinical symptoms) in the Investigator or Sponsor's opinion that could interfere with or limit the participation in the study.
Estimated glomerular filtration rate (eGFR) <30 milliliters (mL)/minute/1.73 square meter (m²) 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.
Trial design
Treatments tested in this trial
- mRNA-3927
Treatment groups
77 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov