Macular DegenerationPhase 1NCT00060749
Effect of DHA Supplements on Macular Function in Patients With Stargardt Macular Dystrophy and Stargardt-like Macular Dystrophy
A Phase 1 study testing Docosahexaenoic Acid (DHA) Dietary Supplement in people with Macular Degeneration, run by National Eye Institute (NEI) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Macular Degeneration
- What is being tested?
- Docosahexaenoic Acid (DHA) Dietary Supplement
- How many people
- 22 participants
- Where
- 1 siteUnited States
- Study dates
- 5 May 2003to 10 Dec 2007
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- To be eligible to enroll in this study, a prospective participant must satisfy the following inclusion criteria.
- Understand and sign the informed consent.
- 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 pattern of inheritance that indicates autosomal recessive inheritance.
- 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.
Show all 9Show fewer
- Autosomal Dominant Stargardt-like Macular Dystrophy Participants (must be observed in at least one study eye):
- Have a pattern of inheritance that indicates autosomal dominant inheritance.
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
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
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:
Read more
- 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.
Read the 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.
- Conditions
- Macular Degeneration
- Being tested
- Docosahexaenoic Acid (DHA) Dietary Supplement
- Keywords
- Stargardt, Macular Dystrophy, ELOVL4, DHA, Stargardt's Disease, Autosomal Dominant Macular Dystrophy, Autosomal Recessive Macular Dystrophy
- Medical subject headings
- Macular Degeneration, Erythrokeratodermia with ataxia, Stargardt Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Docosahexaenoic Acid (DHA) Dietary SupplementDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 May 2003 (estimated)
- Study start5 May 2003
- Study completion10 Dec 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Macular DegenerationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Enrolment
- 22 participants
Sponsor and investigators
- Lead sponsor
- National Eye Institute (NEI)
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00060749
- Sponsor's protocol ID
- 030179
- Other IDs
- 03-EI-0179
- Status last verified
- 10 Dec 2007
- First submitted
- 9 May 2003
- Last updated
- 2 Jul 2017
- Results posted
- No
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. doi: 10.1038/83817.
PubMed 11138005
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00060749
- Acronym / sponsor ID
- 030179
- Conditions
- Macular Degeneration
- Keywords
- Stargardt, Macular Dystrophy, ELOVL4, DHA, Stargardt's Disease, Autosomal Dominant Macular Dystrophy, Autosomal Recessive Macular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants
- Lead sponsor
- National Eye Institute (NEI)US National Institutes of Health
- Study start
- 5 May 2003
- Study completion
- 10 Dec 2007
- First posted
- 12 May 2003 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- To be eligible to enroll in this study, a prospective participant must satisfy the following inclusion criteria.
- Understand and sign the informed consent.
- 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 pattern of inheritance that indicates autosomal recessive inheritance.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Docosahexaenoic Acid (DHA) Dietary Supplement | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2017.
- Background: PubMed 11138005Zhang 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. doi: 10.1038/83817.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Docosahexaenoic Acid (DHA) Dietary Supplement in people with Macular Degeneration, run by National Eye Institute (NEI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00060749, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jul 2017.