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Clinical trial

A Prospective, Longitudinal, Observational, Multi-centre Study of Pediatric Participants up to 16 Years of Age With Methylmalonyl-CoA Mutase Deficiency Caused by Mutations in the MMUT Gene That Results in a Diagnosis of Isolated Methylmalonic Acidemia (MMA).

NCT07432880 is registered in ClinicalTrials.gov with Genespire Srl as lead sponsor.

NCT NCT07432880
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Registry record

As filed in ClinicalTrials.gov.

Title
A Prospective, Longitudinal, Observational, Multi-centre Study of Pediatric Participants up to 16 Years of Age With Methylmalonyl-CoA Mutase Deficiency Caused by Mutations in the MMUT Gene That Results in a Diagnosis of Isolated Methylmalonic Acidemia (MMA).
Lead sponsor
Genespire Srl
Phase
Observational
Overall status
Recruiting
Start date
August 13, 2026
Completion date
August 31, 2030
Enrollment
30 (estimated)
Sites
8

Conditions

As the registry lists them; a condition with its own page is linked.

methylmalonic acidemia (mma)