Trial · NCT01793090
EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment
NCT01793090, Phase 2, Completed, Bambino Gesù Hospital and Research Institute
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment. Trials, record NCT01793090. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01793090
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment
- NCT number
- NCT01793090
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 30 participants, actual
- Lead sponsor
- Bambino Gesù Hospital and Research Institute
- Conditions
- Methylmalonic Aciduria and Homocystinuria,Cblc Type, Genetic Disease, Retinopathy
- Interventions
- Epi-743, Placebo supplementation
- First posted
- 2013-02-15
- Last updated
- 2017-04-25
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Visual Function | Baseline, six months, twelve months | Visual acuity: - Patients age 0-2: Durand acuity cards procedure: Improvement from baseline or nadir by greater than 2 lines when converted to EDTRS values.-Patients age 2-4: LEA Symbols for crowding binocular acuity: Improvement from baseline or nadir by greater than 2 lines when converted to EDTRS values; -Patients age \> 4 years: Cambridge acuity cards: Improved from baseline or nadir by greater than 2 lines on the EDTRS acuity testing chart at 4 meters. Eye-hand coordination: -Patients age 0-2: Improvement over baseline of 20% on Griffiths Mental Development Scale subscales D,E; - Patients age \> 2: Improvement over baseline of 20% on Movement Assessment Battery for Children |
| Secondary | Change in steady-state luminance Visual Evoked Potentials | Baseline, six months, twelve months | Steady-state luminance VEPs to sinusoidal flicker at the optimal frequency of 8 Hz. |
| Secondary | Evaluation of neurological function | Baseline, six months, twelve months | Evaluation of neurological function with Gross motor function measure, movement ABC |
| Other | Biomarkers of redox state | Baseline, six months, twelve months | Glutathione species in blood cells, Antioxidant enzymes expression, redox proteomic studies |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers7
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01793090
GET https://conductscience.org/api/trials/v1/NCT01793090/registered