Phase 1/2 study of mRNA-3927 in participants with PA previously enrolled in the mRNA-3927-P101
Research type
Research Study
Full title
A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101Study
IRAS ID
1003761
Contact name
Tal Zaks
Contact email
Sponsor organisation
ModernaTX, Inc
Eudract number
2020-005009-14
Research summary
Propionic acidaemia (PA) is a metabolic disorder which prevents the body from breaking down specific parts of proteins and fats. It is caused by changes to genes that leads to a problem with an enzyme called propionyl-CoA carboxylase (PCC). PCC is normally responsible for this breakdown, so when it’s missing or cannot work properly, it leads to the build-up of some substances which can be harmful (toxins) and makes it harder for cells to create energy from food. Current treatments for PA manage the symptoms, but do not fix the cause of the disease.
The study is designed to evaluate long-term safety, tolerability and impact of mRNA-3927 on the change in levels of toxins in participants. Treatment will continue unless one of the following occurs: mRNA-3927 receives marketing approval and reimbursement, the participant discontinues study drug, the participant is no longer receiving clinical benefit, or ModernaTX discontinues the development of mRNA-3927.
Participants with PA who were previously enrolled and completed the end-of-treatment (EOT) Visit of the mRNA-3927-P101 study will have the option to enrol into this extension study provided all eligibility criteria have been met. The study will include 2 periods: Treatment Period and Follow-up Period (up to 2 years after the last dose of study drug). During the Treatment Period, participants will attend outpatient and homecare visits as appropriate. In both periods, participants will have multiple types of assessments/ questionnaires.
Participants will be treated with mRNA-3927 by intravenous infusion during the treatment period. All participants will enter the study receiving mRNA-3927 at the same dose and at the same dosing interval as last received in the mRNA-3927-P101 study. The total duration of the study will be approximately 96 months.
Participants who transition to receiving commercially available mRNA-3927will not be followed up but will complete the EOT assessments.REC name
North East - York Research Ethics Committee
REC reference
21/NE/0082
Date of REC Opinion
20 Jul 2021
REC opinion
Further Information Favourable Opinion