mRNA-3704-P101 Patients with Isolated MMA due to MUT Deficiency
Research type
Research Study
Full title
A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients with Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency
IRAS ID
264475
Contact name
Dr Stephanie Grunewald
Contact email
Sponsor organisation
ModernaTx, Inc
Eudract number
2019-001061-32
Duration of Study in the UK
6 years, 0 months, 31 days
Research summary
The study is a first in human, open label, dose escalation study to evaluate the safety, pharmacodynamics (how a drug works in the body) and pharmacokinetics (how a drug moves through the body) of the study drug, mRNA-3704 in participants with Isolated Methylmalonic Acidemia (MMA) due to Methylmalonyl-CoA Mutase Deficiency (MUT).
Isolated methylmalonic acidemia or aciduria (MMA) is an ultra-rare, serious, life-threatening inherited metabolic disorder which occurs in approximately 1 in 50,000 to 100,000 people. The disorder mainly affects children and is diagnosed during early infancy.The first part of the study will consist of a dose escalation phase, looking at three different doses of mRNA-3704. In the second part of the study, the study drug dose given to patients will be based on the dose selected in the first part of the study.
Patients in both parts of the study will participate in a pre-dosing observational period, followed by a treatment period, and then a follow-up period which includes withdrawal of treatment.
The study drug, mRNA-3704, is given by IV (intravenously or through a vein) infusion.
Up to 34 patients will be enrolled globally in the study. The study sponsor is ModernaTX, Inc.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
19/LO/1200
Date of REC Opinion
25 Oct 2019
REC opinion
Further Information Favourable Opinion