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

> **NCT01793090** · PHASE2 · COMPLETED · sponsor: **Bambino Gesù Hospital and Research Institute** · enrollment: 30 (actual)

## Conditions studied

- Methylmalonic Aciduria and Homocystinuria,Cblc Type
- Genetic Disease
- Retinopathy

## Interventions

- **DRUG:** Epi-743
- **OTHER:** Placebo supplementation

## Key facts

- **NCT ID:** NCT01793090
- **Lead sponsor:** Bambino Gesù Hospital and Research Institute
- **Sponsor class:** OTHER
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2013-01
- **Primary completion:** 2013-07
- **Final completion:** 2017-02
- **Target enrollment:** 30 (ACTUAL)
- **Last updated:** 2017-04-25

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01793090

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01793090, "EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT01793090. Licensed CC0.

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