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Effect of DHA Supplements on Macular Function in Patients With Stargardt Macular Dystrophy and Stargardt-like Macular Dystrophy

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ClinicalTrials.gov Identifier: NCT00060749
Recruitment Status : Completed
First Posted : May 12, 2003
Last Update Posted : July 2, 2017
Sponsor:
Information provided by:
National Institutes of Health Clinical Center (CC)

Tracking Information
First Submitted Date  ICMJE May 9, 2003
First Posted Date  ICMJE May 12, 2003
Last Update Posted Date July 2, 2017
Study Start Date  ICMJE May 5, 2003
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE Not Provided
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00060749 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Effect of DHA Supplements on Macular Function in Patients With Stargardt Macular Dystrophy and Stargardt-like Macular Dystrophy
Official Title  ICMJE 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
Brief Summary

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:

  • Medical history and physical examination.
  • Blood test to measure levels of DHA and vitamins.
  • Eye examination: The patient's vision and eye pressure are tested, then the pupils are dilated to examine structures inside the eye. Photographs are also taken.
  • Visual field test: The patient looks at a tiny spot of light projected onto a white screen and is asked to note when other lights appear at other places on the screen.
  • Electroretinogram (ERG): An electrode (small silver disk) is taped to the patient's forehead. Drops are given to numb the eyes and special contact lenses are inserted in the eyes. For the first part of the test, the patient looks at the center of a black and white checkerboard screen that flickers for 30 seconds at a time. This is repeated 16 or more times. For the second part of the test, the patient looks inside a sphere, in which flashes of light flicker for 20 seconds at a time. This is repeated four or more times. The contact lenses sense small electrical signals generated by the retina during the tests.

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.

Detailed Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Primary Purpose: Treatment
Condition  ICMJE Macular Degeneration
Intervention  ICMJE Drug: Docosahexaenoic Acid (DHA) Dietary Supplement
Study Arms  ICMJE Not Provided
Publications * Zhang K, Kniazeva M, Han M, Li W, Yu Z, Yang Z, Li Y, Metzker ML, Allikmets R, Zack DJ, Kakuk LE, Lagali PS, Wong PW, MacDonald IM, Sieving PA, Figueroa DJ, Austin CP, Gould RJ, Ayyagari R, Petrukhin K. A 5-bp deletion in ELOVL4 is associated with two related forms of autosomal dominant macular dystrophy. Nat Genet. 2001 Jan;27(1):89-93.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Enrollment  ICMJE
 (submitted: May 3, 2006)
22
Original Enrollment  ICMJE
 (submitted: June 23, 2005)
20
Study Completion Date  ICMJE December 10, 2007
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE
  • INCLUSION CRITERIA:

To be eligible to enroll in this study, a prospective participant must satisfy the following inclusion criteria.

  1. Understand and sign the informed consent.
  2. 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):

  3. Have a pattern of inheritance that indicates autosomal recessive inheritance.
  4. 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):

  5. Have a pattern of inheritance that indicates autosomal dominant inheritance.
  6. Have a phenotype consistent with the diagnosis of Stargardt-like macular dystrophy that may include: fundus examination showing bilateral central maculopathy and fundus flecks confined to the central macula, or intravenous fluorescein angiogram.

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00060749
Other Study ID Numbers  ICMJE 030179
03-EI-0179
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE National Eye Institute (NEI)
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account National Institutes of Health Clinical Center (CC)
Verification Date December 10, 2007

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP