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Sponsored by: |
National Eye Institute (NEI) |
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Information provided by: | National Institutes of Health Clinical Center (CC) |
ClinicalTrials.gov Identifier: | NCT00060749 |
This study will evaluate whether docosahexaenoic acid (DHA) dietary supplementation can improve macular function in patients with Stargardt macular dystrophy and Stargardt-like macular dystrophy. Stargardt macular dystrophy is a recessive inherited trait that causes a severe form of macular degeneration. (The macula is the center part of the retina in the back of the eye that is responsible for fine vision.) The disorder begins in late childhood and progresses to a significant decrease in central vision. One of the earliest signs of the disorder is accumulation in and under the macula of a fatty pigment called lipofuscin. Stargardt-like macular dystrophy is a dominant inherited trait involving loss of central vision, but it begins later than Stargardt macular dystrophy, and the accumulation of lipofuscin extends beyond the central region of the macula. DHA is a fatty acid that is essential for normal brain and eye development. It is normally found in the diet, but not in large amounts. Supplements may help prevent or slow the progression of some eye diseases.
Patients with autosomal dominant Stargardt-like macular dystrophy or autosomal recessive Stargardt macular dystrophy are eligible for this study.
Candidates will be screened with the following tests and procedures:
Participants will begin taking DHA capsules or a placebo (look-alike capsules with no active ingredient) from 1 week to 3 months after enrolling in the study and will repeat several of the screening tests at follow-up visits scheduled 3, 6, 9, 12, and 15 months after they start taking the capsules. They will also be interviewed about any treatment side effects.
Condition | Intervention | Phase |
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Macular Degeneration |
Drug: Docosahexaenoic Acid (DHA) Dietary Supplement |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment |
Official Title: | Investigation of the Effect of Dietary Docosahexaenoic Acid (DHA) Supplementation on Macular Function in Subjects With Autosomal Dominant Stargardt-Like and Autosomal Recessive Stargardt Macular Dystrophy |
Estimated Enrollment: | 22 |
Study Start Date: | May 2003 |
Estimated Study Completion Date: | December 2007 |
We propose to undertake a double-masked, randomized, placebo-controlled, crossover study on the effect of docosahexaenoic acid (DHA) dietary supplementation in subjects with macular dystrophy to determine whether DHA can improve macular function. Subjects will receive either oral DHA supplementation (5x200 mg BID, 2,000 mg/day) or placebo. Subjects will 'crossover' to the opposite treatment twice during this study. Primary outcomes will measure the change in macular function during periods with and without DHA supplementation.
Zhang and colleagues found a mutation in the gene, ELOVL4 (elongation of the very long chain fatty acid-4), in individuals with Stargardt-like macular dystrophy. The gene is presumed to function in the pathway of synthesis of very long chain polyunsaturated fatty acids, including DHA. DHA is the major very long chain polyunsaturated fatty acid of the retina. As our North American diet is poor in DHA, we hypothesize that a DHA dietary supplement might improve macular function in individuals with the ELOVL4 mutation. Since the effect of DHA supplementation may be non-specific, we propose to study a second cohort with Stargardt macular dystrophy, which has a different genotype involving a different metabolic pathway in the eye, but presents with a similar phenotype. Two cohorts of up to 10 subjects for analysis will be recruited from patients with either Stargardt-like macular dystrophy or Stargardt macular dystrophy.
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
To be eligible to enroll in this study, a prospective participant must satisfy the following inclusion criteria.
Able to comply with all study procedures (likely to exclude participants less than 10 years of age, but not necessarily).
Autosomal Recessive Stargardt Macular Dystrophy Participants (must be observed in at least one study eye):
Have a phenotype consistent with the diagnosis of autosomal recessive Stargardt macular dystrophy including the following clinical features: fundus examination showing bilateral central maculopathy and/or fundus flecks, or characteristic changes on an intravenous fluorescein angiogram.
Autosomal Dominant Stargardt-like Macular Dystrophy Participants (must be observed in at least one study eye):
EXCLUSION CRITERIA:
To be eligible to enroll in this study, a prospective participant must not satisfy any of the following exclusion criteria.
1. Have a non-recordable multi-focal ERG.
Study ID Numbers: | 030179, 03-EI-0179 |
Study First Received: | May 9, 2003 |
Last Updated: | December 11, 2007 |
ClinicalTrials.gov Identifier: | NCT00060749 History of Changes |
Health Authority: | United States: Federal Government |
Stargardt Macular Dystrophy ELOVL4 DHA Stargardt's Disease |
Autosomal Dominant Macular Dystrophy Autosomal Recessive Macular Dystrophy Stargardt's Disease Autosomal Dominant Macular Dystrophy Autosomal Recessive Macular Dystrophy |
Eye Diseases Retinal Degeneration Macular Degeneration Retinal Diseases |
Eye Diseases Retinal Degeneration Macular Degeneration Retinal Diseases |