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DHA and X-Linked Retinitis Pigmentosa

Primary Purpose

Retinitis Pigmentosa, X-linked Genetic Diseases

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
docosahexaenoic acid OR corn/soy oil placebo
Sponsored by
Retina Foundation of the Southwest
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Retinitis Pigmentosa focused on measuring retina, electroretinography, clinical trial, docosahexaenoic acid, omega-3 fatty acid, x-linked inheritance

Eligibility Criteria

7 Years - 32 Years (Child, Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria: 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 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. Exclusion Criteria: 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.

Sites / Locations

  • Retina Foundation of the Southwest

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

1.

2

Arm Description

Oral Docosahexaenoic acid, dosage based on body weight

corn/soy oil placebo; oil not containing DHA...dosage based on body weight

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.

Full Information

First Posted
December 27, 2004
Last Updated
March 4, 2015
Sponsor
Retina Foundation of the Southwest
Collaborators
Foundation Fighting Blindness, DSM Nutritional Products, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT00100230
Brief Title
DHA and X-Linked Retinitis Pigmentosa
Official Title
Investigation of Effectiveness and Safety of High Dose Docosahexaenoic Acid (DHA) in X-Linked Retinitis Pigmentosa
Study Type
Interventional

2. Study Status

Record Verification Date
March 2015
Overall Recruitment Status
Completed
Study Start Date
September 2004 (undefined)
Primary Completion Date
May 2012 (Actual)
Study Completion Date
August 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Retina Foundation of the Southwest
Collaborators
Foundation Fighting Blindness, DSM Nutritional Products, Inc.

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief 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). 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. 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
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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Retinitis Pigmentosa, X-linked Genetic Diseases
Keywords
retina, electroretinography, clinical trial, docosahexaenoic acid, omega-3 fatty acid, x-linked inheritance

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
78 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1.
Arm Type
Experimental
Arm Description
Oral Docosahexaenoic acid, dosage based on body weight
Arm Title
2
Arm Type
Placebo Comparator
Arm Description
corn/soy oil placebo; oil not containing DHA...dosage based on body weight
Intervention Type
Drug
Intervention Name(s)
docosahexaenoic acid OR corn/soy oil placebo
Other Intervention Name(s)
DHA; omega-3 fatty acid, OR RANDOMIZED TO corn/soy oil placebo
Intervention Description
daily intake of DHA based on body weight or corn/soy oil placebo(oil not containing DHA; 4 year trial
Primary Outcome Measure Information:
Title
Rate of LOSS of 31 Hertz Cone Electroretinographic Function
Description
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.
Time Frame
4 years
Secondary Outcome Measure Information:
Title
Rate of LOSS of Rod Electroretinographic Function
Description
Hypothesis #1: Elevation of red blood cell-docosahexaenoic acid levels will slow the progressive loss of rod electroretinographic response in this 4-year trial.
Time Frame
4 years
Other Pre-specified Outcome Measures:
Title
Loss of Peripheral Visual Fields
Description
Hypothesis: Elevation of red blood cell-docosahexaenoic acid levels will slow the progressive loss of peripheral visual fields in this 4-year trial.
Time Frame
4 years

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
7 Years
Maximum Age & Unit of Time
32 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 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. Exclusion Criteria: 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dennis R. Hoffman, Ph.D.
Organizational Affiliation
Retina Foundation of the Southwest
Official's Role
Principal Investigator
Facility Information:
Facility Name
Retina Foundation of the Southwest
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
24805262
Citation
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.
Results Reference
result
PubMed Identifier
25015354
Citation
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.
Results Reference
result
PubMed Identifier
26469750
Citation
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.
Results Reference
derived
PubMed Identifier
25342618
Citation
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.
Results Reference
derived
PubMed Identifier
23828615
Citation
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.
Results Reference
derived
Links:
URL
http://www.retinafoundation.org
Description
website for Retina Foundation of the Southwest

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DHA and X-Linked Retinitis Pigmentosa

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