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Retinitis PigmentosaPhase 2NCT00100230

DHA and X-Linked Retinitis Pigmentosa

A Phase 2 study of Retinitis Pigmentosa and X-linked Genetic Diseases, run by Retina Foundation of the Southwest at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Purpose: Retinitis pigmentosa (RP) is characterized by progressive loss of visual function due to specific genetic mutations. This trial is focused on patients with one of the most severe forms of the disease, X-linked inherited RP (XLRP). Read more about this study

Who is it for?
Ages 7 to 32 years, men only
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
78 participants
Where
1 siteUnited States
Study dates
Sep 2004to Aug 2014
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 7 to 32 years
  • Men only
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Diagnosis of RP by a retinal specialist
  • Clinical diagnosis consistent with X-linked inheritance
  • Enrolling minors and young adults (early onset of X-linked disease; ages 7 to 32)
  • Measurable cone ERG responses --patients with less than 0.64 microvolt response to 31-Hz flicker will be excluded as they are more likely to become undetectable during the study
  • Both eyes must meet entry criteria as both will be tested (i.e., no cataracts requiring surgery or retinal detachments).
  • Media clarity sufficient for fundus photography
Show all 10Show fewer
  • Able to return to study site at yearly intervals
  • Willing to supply blood samples at 6-month intervals
  • Judiciously take the placebo or DHA supplement for the 4-year study duration
  • Patient/parent/guardian understands and signs consent form.

Who cannot join

  • Excessive fish consumption (e.g., cold water fish such as salmon, tuna, sardines) and/or fish oil supplementation (or other oil containing DHA)
  • Baseline RBC-DHA levels showing evidence of supplementation (a typical level of RBC-DHA in normals is about 3.8%)
  • Chronic metabolic disease that may interfere with fatty acid metabolism or require anti-coagulant medication
  • No ethnic or racial groups will be excluded.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Retina Foundation of the Southwest

    Dallas, Texas, 75231

Other trials in United States

About this study

Official title: Investigation of Effectiveness and Safety of High Dose Docosahexaenoic Acid (DHA) in X-Linked Retinitis Pigmentosa

Purpose:

Retinitis pigmentosa (RP) is characterized by progressive loss of visual function due to specific genetic mutations. This trial is focused on patients with one of the most severe forms of the disease, X-linked inherited RP (XLRP). This disease is characterized by early onset (typically loss of night vision as a child) followed by loss of peripheral vision as a teenager and young adult. There is no male-to-male transmission of the disease in the family.

Read more

There is no cure for RP and treatment options are limited. Two clinical trials have not found a benefit from nutritional supplementation with the long-chain polyunsaturated fatty acid, docosahexaenoic acid (DHA), at low daily doses although there is evidence that it slows disease progression in certain instances. In this clinical trial, we propose that a high dose nutritional DHA supplement will slow the loss of visual function and preserve usable vision in patients with XLRP.

This study is a 4-year placebo-controlled randomized clinical trial meaning that patients have a 50-50 chance of receiving placebo or experimental treatment. A total of 66 patients will be enrolled; 33 will receive placebo and 33 will receive the treatment. Entry criteria include diagnosis of XLRP by an ophthalmologist, age 7 to 32 years, male, sufficient visual function such that disease progression can be followed for the entire duration of the trial, and a willingness to visit the testing site (Dallas, TX) once a year.

Annual visual function testing includes ETDRS visual acuity, full-field and multifocal electroretinography (ERG), static peripheral visual fields, and fundus photography. Cone ERG function is the primary outcome measure.

Funding Source - FDA, Foundation Fighting Blindness, DSM Nutritionals

Read the detailed description

Location \& Contact Information:

Retina Foundation of the Southwest, 9600 N. Central Expressway, Suite 200, Dallas, TX 75231 Contact: Dr. D. Hoffman (dhoffman@retinafoundation.org) or Dr. D. Birch (dbirch@retinafoundation.org).

Being tested
docosahexaenoic acid OR corn/soy oil placebo
Keywords
retina, electroretinography, clinical trial, docosahexaenoic acid, omega-3 fatty acid, x-linked inheritance
Medical subject headings
Retinitis Pigmentosa, Genetic Diseases, X-Linked

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • docosahexaenoic acid OR corn/soy oil placeboDrug

    Also called DHA; omega-3 fatty acid, OR RANDOMIZED TO corn/soy oil placebo

    daily intake of DHA based on body weight or corn/soy oil placebo(oil not containing DHA; 4 year trial

Study groups in NCT00100230
Study groupRoleWhat they receive
1.ExperimentalOral Docosahexaenoic acid, dosage based on body weight
Receives: Drug: docosahexaenoic acid OR corn/soy oil placebo
2Placebo comparatorcorn/soy oil placebo; oil not containing DHA...dosage based on body weight
Receives: Drug: docosahexaenoic acid OR corn/soy oil placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Rate of LOSS of 31 Hertz Cone Electroretinographic Function

    Hypothesis #1: Elevation of red blood cell-docosahexaenoic acid levels will slow the progressive loss of 31 hertz cone electroretinographic response in this 4-year trial.

    Measured: 4 years

Secondary outcomes (1)

  1. Rate of LOSS of Rod Electroretinographic Function

    Hypothesis #1: Elevation of red blood cell-docosahexaenoic acid levels will slow the progressive loss of rod electroretinographic response in this 4-year trial.

    Measured: 4 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Dec 2004 (estimated)
  2. Study startSep 2004
  3. Primary completionMay 2012
  4. Study completionAug 2014

Resources

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 2
Primary purpose
Prevention
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
78 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00100230
Sponsor's protocol ID
2543
Other IDs
FD-R-002543, 5R01FD002543, C-TX02-0704-0274
Status last verified
Mar 2015
First submitted
27 Dec 2004
Last updated
17 Mar 2015
Data monitoring committee
Yes

Publications

  • Hoffman DR, Hughbanks-Wheaton DK, Pearson NS, Fish GE, Spencer R, Takacs A, Klein M, Locke KG, Birch DG. Four-year placebo-controlled trial of docosahexaenoic acid in X-linked retinitis pigmentosa (DHAX trial): a randomized clinical trial. JAMA Ophthalmol. 2014 Jul;132(7):866-73. doi: 10.1001/jamaophthalmol.2014.1634.

    PubMed 24805262

  • Hughbanks-Wheaton DK, Birch DG, Fish GE, Spencer R, Pearson NS, Takacs A, Hoffman DR. Safety assessment of docosahexaenoic acid in X-linked retinitis pigmentosa: the 4-year DHAX trial. Invest Ophthalmol Vis Sci. 2014 Jul 11;55(8):4958-66. doi: 10.1167/iovs.14-14437.

    PubMed 25015354

  • Hoffman DR, Hughbanks-Wheaton DK, Spencer R, Fish GE, Pearson NS, Wang YZ, Klein M, Takacs A, Locke KG, Birch DG. Docosahexaenoic Acid Slows Visual Field Progression in X-Linked Retinitis Pigmentosa: Ancillary Outcomes of the DHAX Trial. Invest Ophthalmol Vis Sci. 2015 Oct;56(11):6646-53. doi: 10.1167/iovs.15-17786.

    PubMed 26469750

  • Cai CX, Locke KG, Ramachandran R, Birch DG, Hood DC. A comparison of progressive loss of the ellipsoid zone (EZ) band in autosomal dominant and x-linked retinitis pigmentosa. Invest Ophthalmol Vis Sci. 2014 Oct 23;55(11):7417-22. doi: 10.1167/iovs.14-15013.

    PubMed 25342618

  • Birch DG, Locke KG, Wen Y, Locke KI, Hoffman DR, Hood DC. Spectral-domain optical coherence tomography measures of outer segment layer progression in patients with X-linked retinitis pigmentosa. JAMA Ophthalmol. 2013 Sep;131(9):1143-50. doi: 10.1001/jamaophthalmol.2013.4160.

    PubMed 23828615

Questions about this trial

What is this clinical trial studying?

A Phase 2 study of Retinitis Pigmentosa and X-linked Genetic Diseases, run by Retina Foundation of the Southwest 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 10 inclusion and 4 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 17 Mar 2015. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 Mar 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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.

What does the primary outcome measure?

Rate of LOSS of 31 Hertz Cone Electroretinographic Function (time frame: 4 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00100230, which also lists 5 linked publications.

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 17 Mar 2015.

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