EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment

PHASE2CompletedINTERVENTIONAL
Enrollment

30

Participants

Timeline

Start Date

January 31, 2013

Primary Completion Date

July 31, 2013

Study Completion Date

February 28, 2017

Conditions
Methylmalonic Aciduria and Homocystinuria,Cblc TypeGenetic DiseaseRetinopathy
Interventions
DRUG

Epi-743

EPI- 743 in capsule or formulation comprised of USP/NF Sesame Oil at a potency of 100 mg EPI-743/ 1 mL total volume. Mode of Administration: Oral with meal or G-Tube infusion with food.

OTHER

Placebo supplementation

Placebo will be administered in the same formulation as the active comparator

Trial Locations (1)

00165

Bambino Gesù Hospital and Research Institute, Rome

All Listed Sponsors
collaborator

Catholic University of the Sacred Heart

OTHER

lead

Bambino Gesù Hospital and Research Institute

OTHER

NCT01793090 - EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment | Biotech Hunter | Biotech Hunter