Clinical trial NCT00000116: Randomized Trial of DHA for Retinitis Pigmentosa Patients Receiving Vitamin A
Official title: Clinical Trial of Docosahexaenoic Acid (DHA) in Patients With Retinitis Pigmentosa Receiving Vitamin A Treatment
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Retinitis Pigmentosa
Lead sponsor: National Eye Institute (NEI)
Sponsor class: NIH
First posted: 1999-09-24
Enrollment: 221
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this trial is to determine whether a nutritional supplement in addition to vitamin A will slow the course of retinitis pigmentosa.

Detailed description: Retinitis pigmentosa (RP) is a group of inherited retinal degenerations with a worldwide prevalence of approximately 1 in 4,000. Patients typically report night blindness and difficulty with midperipheral visual field in adolescence. As the condition progresses, they lose far peripheral visual field. Most patients have reductions in central vision by age 50 to 80 years. Based on electroretinograms (ERGs), the course of the disease can be slowed on average among adults on 15,000 IU/day of vitamin A palmitate. While conducting the trial on the effects of vitamin A on RP, it became apparent that another substance in the diet could be affecting the course of the disease. This prompted the present randomized, controlled trial.

This study is a randomized, controlled, double-masked trial with a planned duration of 5 years. Patients with the common forms of RP are assigned to either a test or a control group. All receive 15,000 IU/day of vitamin A palmitate in addition to either 1200 mg/d of docosahexaenoic acid or control capsules. Participants will not know the contents of the supplement or the group to which they have been assigned until the end of the trial. The main outcome measurement is the total point score on the Humphrey Field Analyzer (HFA). In addition, computer-averaged 30-Hz cone ERG amplitudes and visual acuity are measured annually.

Eligibility criteria: Inclusion Criteria:

Ocular Criteria:

Retinitis Pigmentosa, typical (non-syndromic) forms Best corrected visual acuity Greater than or equal to (GE )20/100 HFA 30-2 total point score GE 250 dB (decibels) 30 (Hertz) Hz ERG cone amplitude GE 0.68 microvolts

Dietary Criteria:

Dark fish intake Less than or equal to (LE) five servings per week Dietary omega-3 fatty acid intake LE 0.41 g/d Preformed Vitamin A intake in diet and supplements LE 10,000 IU/d Supplement intake LE 5000 IU/d of Vitamin A and LE 30 IU/d Vitamin E Consumption LE 3 alcoholic beverages per day

Medical and other criteria:

Body Mass Index Less than (LT )40 and weight GE 5th percentile for age, sex, and height Serum retinol level LE 100 mg/dl and serum retinyl ester levels LE 380 nm/L Serum cholesterol level LT 300 mg/dL and serum triglyceride levels LT 400 mg/dL Agree not to know study capsule content

Exclusion criteria:

Ocular criteria:

No confounding ocular disease

Dietary criteria:

No intake of cod liver oil or omega-3 capsules

Medical and other criteria:

Not pregnant or planning to become pregnant No clinically significant abnormal result on Complete Blood Count or serum liver function profile No other disease which might affect absorption or metabolism of DHA or Vitamin A

Primary outcomes:
measure: Change in Humphrey Field Analyzer (HFA) total point score 30-2 program
timeFrame: Annual percent change per year at each of 4 years of followup
Secondary outcomes:
measure: Change in Humphrey Field Analyzer (HFA) total point score 30 -2 plus 30/60-2 programs combined
timeFrame: Annual percent change per year at each of four years of followup
measure: Change in 30Hz Electroretinogram ( ERG) amplitude
timeFrame: Annual percent change per year at each of four years of followup
measure: Change in Early Treatment of Diabetic Retinopathy(EDTRS) Visual Acuity
timeFrame: Change in number of letters read per year at each of four years of followup.
Other outcomes:
Locations:
city: Boston
country: United States
facility: Berman-Gund Laboratory for the Study of Retinal Degenerations, Harvard Medical School, Massachusetts Eye and Ear Infirmary
state: Massachusetts
Study officials:
affiliation: Berman-Gund Laboratory for the Study of Retinal Degenerations, Harvard Medical School
name: Eliot Berson (Deceased), MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Berson EL, Rosner B, Sandberg MA, Hayes KC, Nicholson BW, Weigel-DiFranco C, Willett W. A randomized trial of vitamin A and vitamin E supplementation for retinitis pigmentosa. Arch Ophthalmol. 1993 Jun;111(6):761-72. doi: 10.1001/archopht.1993.01090060049022.
citation: Berson EL, Rosner B, Sandberg MA, Weigel-DiFranco C, Moser A, Brockhurst RJ, Hayes KC, Johnson CA, Anderson EJ, Gaudio AR, Willett WC, Schaefer EJ. Clinical trial of docosahexaenoic acid in patients with retinitis pigmentosa receiving vitamin A treatment. Arch Ophthalmol. 2004 Sep;122(9):1297-305. doi: 10.1001/archopht.122.9.1297.
citation: Berson EL, Rosner B, Sandberg MA, Weigel-DiFranco C, Moser A, Brockhurst RJ, Hayes KC, Johnson CA, Anderson EJ, Gaudio AR, Willett WC, Schaefer EJ. Further evaluation of docosahexaenoic acid in patients with retinitis pigmentosa receiving vitamin A treatment: subgroup analyses. Arch Ophthalmol. 2004 Sep;122(9):1306-14. doi: 10.1001/archopht.122.9.1306.