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Trial intelligence / Profile preview
mitoFolat
2023-503730-45-00Phase 2Authorised
- Sponsor
- Universitätsmedizin Göttingen
- Start
- Oct 2023
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Phase 2, randomised, single-blind trial of folinic acid in Kearns-Sayre syndrome (KSS) and cerebral folate deficiency (CFD) (2 arms), sponsored by Universitätsmedizin Göttingen. Primary endpoint: International Pediatric Mitochondrial Disease Scale (IPMDS) baseline-adjusted mean difference at 6 months. Planned start Oct 2023; status: authorised.
02
Drugs
folinic acid
03
Conditions
Kearns-Sayre syndrome (KSS)cerebral folate deficiency (CFD)
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