The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103).
The study will assess long-term safety of mRNA-3927. Participants with PA who were previously enrolled and completed the end-of-treatment (EOT)/early termination (ET) visit of the mRNA-3927-P101 study 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 (90 days after the EOT visit). All participants will enter the study receiving mRNA-3927 at the same dose and at the same dosing interval last received in the mRNA-3927-P101 study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
50
mRNA-3927 dispersion for IV infusion
Ronald Reagan UCLA Medical Center
Los Angeles, California, United States
RECRUITINGUniversity of Michigan Hospitals
Ann Arbor, Michigan, United States
Number of Participants with Adverse Events (AEs), Serious AEs (SAEs), and AEs Leading to Discontinuation
Time frame: Baseline through End of Study Visit (up to 8 years)
Annualized Frequency of Investigator-reported Metabolic Decompensation Events (MDEs)
Time frame: Baseline through End of Study Visit (up to 8 years)
Annualized Frequency of Investigator-reported MDE-related Hospitalizations
Time frame: Baseline through End of Study Visit (up to 8 years)
Annualized Frequency of Investigator-reported PA-related Hospitalizations
Time frame: Baseline through End of Study Visit (up to 8 years)
Annualized Frequency of Investigator-reported PA-related Urgent Healthcare Encounters
Time frame: Baseline through End of Study Visit (up to 8 years)
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Icahn School of Medicine at Mount Sinai
New York, New York, United States
RECRUITINGDuke University Medical System (Duke Health)
Durham, North Carolina, United States
RECRUITINGThe Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
RECRUITINGTexas Children's Hospital
Houston, Texas, United States
RECRUITINGHospital For Sick Children
Toronto, Ontario, Canada
RECRUITINGFujita Health University Hospital
Toyoake-shi, Akita, Japan
NOT_YET_RECRUITINGWillink Biochemical Genetics Unit - Manchester
Manchester, England, United Kingdom
RECRUITINGUniversity Hospital Birmingham NHS Foundation Trust
Birmingham, West Midlands, United Kingdom
RECRUITING...and 2 more locations