Investigating the Effect of Vitamin A Supplementation on Retinitis Pigmentosa

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT00065455
• Date of registration: 23/07/2003
• Prospective Registration: No
• Primary sponsor: National Eye Institute (NEI)
• Public title: Investigating the Effect of Vitamin A Supplementation on Retinitis Pigmentosa
• Scientific title: Pilot Study on the Effect of Vitamin A Supplementation on Cone Function in Retinitis Pigmentosa
• Date of first enrolment: July 17, 2003
• Target sample size: 11
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00065455
• Study type: Interventional
• Study design: Primary purpose: Treatment.
• Phase: Phase 1

Countries of recruitment

United States

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

All participants must meet the following criteria to participate in the study.

1. Men and women age 18 years of age and older. (Children will be excluded since there is
a higher incidence of vitamin A toxicity in the pediatric population.)

2. Diagnoses of typical RP of all genetic subtypes (simplex, autosomal dominant,
autosomal recessive, and X-linked), as determined primarily by abnormally reduced ERG
rod response amplitudes that are relatively more affected than cone ERG amplitudes.

3. Mutation in the RHO1 gene as determined by genotyping.

4. Participants in whom flicker ERG can be measured reliably (standard flicker ERG
amplitude greater than 2 microV).

5. Willingness to use contraception for the duration of the study.

6. Understood and signed consent.

EXCLUSION CRITERIA:

Participants with the following conditions will be excluded from study.

1. Participants with syndromic RP (i.e., Ushers syndrome).

2. Have abnormal liver function (greater than ULN ALT, AST, Alkaline Phosphate, or Total
Bilirubin).

3. Hematocrit greater than 1.5 x ULN

4. Have abnormal kidney function (greater than1.5 mg/dL serum creatinine).

5. Currently or has taken greater than 15,000 IU/day vitamin A supplementation within 6
months of the first screening visit.

6. Is pregnant or lactating (due to evidence that sugests excessive intake of vitamin A
could be tertogenic in humans and affect the content of breast milk).

7. Currently or has taken greater than 400 IU/day of vitamin E supplementation within 6
months of enrollment.

8. Vitamin A serum level exceeding 150 microg/dL.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Retinitis Pigmentosa

Intervention(s)

Drug: Vitamin A

Secondary ID(s)

030255

03-EI-0255

Source(s) of Monetary Support

Please refer to primary and secondary sponsors