Propionic Acidemia
Conditions
Keywords
mRNA-3927, Propionic Aciduria, Metabolism, Inborn Errors, Genetic Diseases, Inborn Amino Acid Metabolism, Inborn Errors, Acidosis, Acid-Base Imbalance, Metabolic Diseases, Organic Acidemias, Moderna, mRNA
Brief summary
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).
Detailed description
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.
Interventions
BIOLOGICALmRNA-3927
mRNA-3927 dispersion for IV infusion
Sponsors
ModernaTX, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participated in Study mRNA-3927-P101. * Completed the EOT/ET visit in Study mRNA-3927-P101 and enroll in this study such that the first dose in this study is planned to be within 14±3 days of the last dose of mRNA-3927 in the mRNA-3927-P101 study.
Exclusion criteria
* Not expected to receive clinical benefit from continued mRNA-3927 administration, in the opinion of the Investigator. * Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the individual at increased risk by participating in this study. * History of liver and/or kidney transplant.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants with Adverse Events (AEs), Serious AEs (SAEs), and AEs Leading to Discontinuation | Baseline through End of Study Visit (up to 8 years) |
Secondary
| Measure | Time frame |
|---|---|
| Annualized Frequency of Investigator-reported Metabolic Decompensation Events (MDEs) | Baseline through End of Study Visit (up to 8 years) |
| Annualized Frequency of Investigator-reported MDE-related Hospitalizations | Baseline through End of Study Visit (up to 8 years) |
| Annualized Frequency of Investigator-reported PA-related Hospitalizations | Baseline through End of Study Visit (up to 8 years) |
| Annualized Frequency of Investigator-reported PA-related Urgent Healthcare Encounters | Baseline through End of Study Visit (up to 8 years) |
Countries
Canada, France, Japan, Netherlands, Saudi Arabia, Spain, United Kingdom, United States
Contacts
CONTACTModerna WeCare Team
Outcome results
None listed