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DHA and X-Linked Retinitis Pigmentosa

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ClinicalTrials.gov Identifier: NCT00100230
Recruitment Status : Completed
First Posted : December 28, 2004
Results First Posted : March 17, 2015
Last Update Posted : March 17, 2015
Sponsor:
Collaborators:
Foundation Fighting Blindness
DSM Nutritional Products, Inc.
Information provided by (Responsible Party):
Dennis Hoffman, Retina Foundation of the Southwest

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Prevention
Conditions Retinitis Pigmentosa
X-linked Genetic Diseases
Intervention Drug: docosahexaenoic acid OR corn/soy oil placebo
Enrollment 78
Recruitment Details  
Pre-assignment Details  
Arm/Group Title 1. Docosahexaenoic Acid (DHA) Arm Corn/Soy Oil Placebo Arm
Hide Arm/Group Description Docosahexaenoic acid (an omega-3 polyunsaturated fatty acid) taken orally at a dosage of 30 mg/kg body weight/day for a 4-year duration trial. Corn/soy oil placebo (oils not containing DHA); dosage based on body weight for 4-year trial
Period Title: Overall Study
Started 41 37
Completed 29 22
Not Completed 12 15
Arm/Group Title 1. Docosahexaenoic Acid (DHA) Arm Corn/Soy Oil Placebo Arm Total
Hide Arm/Group Description Docosahexaenoic acid (an omega-3 polyunsaturated fatty acid) taken orally at a dosage of 30 mg/kg body weight/day for a 4-year duration trial. Corn/soy oil placebo (oils not containing DHA); dosage based on body weight for 4-year trial Total of all reporting groups
Overall Number of Baseline Participants 41 37 78
Hide Baseline Analysis Population Description
males with X-linked retinitis pigmentosa
Age, Customized   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Age Number Analyzed 41 participants 37 participants 78 participants
16.7  (7.8) 16.2  (6.6) 16.7  (7.1)
[1]
Measure Description: Ages 7 to 32 years
Sex/Gender, Customized  
Measure Type: Number
Unit of measure:  Participants
male Number Analyzed 41 participants 37 participants 78 participants
41 37 78
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 41 participants 37 participants 78 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   2.4%
0
   0.0%
1
   1.3%
White
37
  90.2%
33
  89.2%
70
  89.7%
More than one race
3
   7.3%
4
  10.8%
7
   9.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 41 participants 37 participants 78 participants
Hispanic or Latino
4
   9.8%
1
   2.7%
5
   6.4%
Not Hispanic or Latino
37
  90.2%
36
  97.3%
73
  93.6%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 41 participants 37 participants 78 participants
United States 38 35 73
Canada 3 2 5
1.Primary Outcome
Title Rate of LOSS of 31 Hertz Cone Electroretinographic Function
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Number of participants completing at least one year of trial (i.e., modified intent to treat cohort)
Arm/Group Title DHA (Docosahexaenoic Acid) Placebo (Corn/Soy Oil)
Hide Arm/Group Description:

Oral Docosahexaenoic acid, dosage based on body weight

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

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

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

Overall Number of Participants Analyzed 33 27
Mean (Standard Error)
Unit of Measure: log microvolts/year
0.028  (0.001) 0.022  (0.002)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection DHA (Docosahexaenoic Acid), Placebo (Corn/Soy Oil)
Comments [Not Specified]
Type of Statistical Test Non-Inferiority or Equivalence
Comments Equivalence analysis: standard power calculation of 20%; used 2-sided t-test
Statistical Test of Hypothesis P-Value 0.30
Comments Threshold for significance: p<0.05
Method Mixed Models Analysis
Comments [Not Specified]
2.Secondary Outcome
Title Rate of LOSS of Rod Electroretinographic Function
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Number of participants completing at least one year of trial (i.e., modified intent to treat cohort)
Arm/Group Title DHA (Docosahexaenoic Acid) Placebo (Corn/Soy Oil)
Hide Arm/Group Description:

Oral Docosahexaenoic acid, dosage based on body weight

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

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

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

Overall Number of Participants Analyzed 33 27
Mean (Standard Error)
Unit of Measure: change in amplitude, log microvolts/year
0.010  (0.001) 0.023  (0.001)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection DHA (Docosahexaenoic Acid), Placebo (Corn/Soy Oil)
Comments [Not Specified]
Type of Statistical Test Non-Inferiority or Equivalence
Comments Equivalence analysis: standard power calculation of 20%; used 2-sided t-test
Statistical Test of Hypothesis P-Value 0.27
Comments Threshold for significance: p<0.05
Method Mixed Models Analysis
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection DHA (Docosahexaenoic Acid), Placebo (Corn/Soy Oil)
Comments [Not Specified]
Type of Statistical Test Non-Inferiority or Equivalence
Comments Equivalence analysis: standard power calculation of 20%; used 2-sided t-test
Statistical Test of Hypothesis P-Value 0.27
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
3.Other Pre-specified Outcome
Title Loss of Peripheral Visual Fields
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Only participants completing at least one year of trial
Arm/Group Title DHA (Docosahexaenoic Acid) ARM Placebo (Corn/Soy Oil) ARM
Hide Arm/Group Description:

Oral Docosahexaenoic acid, dosage based on body weight

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

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

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

Overall Number of Participants Analyzed 33 27
Mean (Standard Error)
Unit of Measure: decibels (dB)
50.4  (3.2) 112.8  (2.0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection DHA (Docosahexaenoic Acid) ARM, Placebo (Corn/Soy Oil) ARM
Comments [Not Specified]
Type of Statistical Test Non-Inferiority or Equivalence
Comments Equivalence analysis: standard power calculation of 20%; used 2-sided t-test
Statistical Test of Hypothesis P-Value 0.000008
Comments Threshold for significance: p<0.05
Method Mixed Models Analysis
Comments [Not Specified]
Time Frame 4 years
Adverse Event Reporting Description Adverse events criteria were based on: Division of AIDS (DAIDS) National Institutes of Health. Table for grading severity of adult and pediatric adverse events (Dec 2004; rev. 2009) (http://rsc.tech-res.com/Document/safetyandpharmacovigilance/Table_for_Grading_Severity_of_Adult_Pediatric_Adverse_Events.pdf). Date of last access: 3-19-2014.
 
Arm/Group Title 1. Docosahexaenoic Acid (DHA) Arm Corn/Soy Oil Placebo Arm
Hide Arm/Group Description Docosahexaenoic acid (an omega-3 polyunsaturated fatty acid) taken orally at a dosage of 30 mg/kg body weight/day for a 4-year duration trial. Corn/soy oil placebo (oils not containing DHA); dosage based on body weight for 4-year trial
All-Cause Mortality
1. Docosahexaenoic Acid (DHA) Arm Corn/Soy Oil Placebo Arm
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
1. Docosahexaenoic Acid (DHA) Arm Corn/Soy Oil Placebo Arm
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/33 (0.00%)      0/27 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
1. Docosahexaenoic Acid (DHA) Arm Corn/Soy Oil Placebo Arm
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   9/33 (27.27%)      16/27 (59.26%)    
Blood and lymphatic system disorders     
Low HDL cholesterol  1  2/33 (6.06%)  2 5/27 (18.52%)  5
elevated LDL cholesterol  1  2/33 (6.06%)  2 0/27 (0.00%)  0
elevated total cholesterol  1  2/33 (6.06%)  2 1/27 (3.70%)  1
elevated potassium  1  0/33 (0.00%)  0 4/27 (14.81%)  4
elevated glucose  1  0/33 (0.00%)  0 3/27 (11.11%)  3
Gastrointestinal disorders     
Stomach ache  1  2/33 (6.06%)  2 1/27 (3.70%)  1
Nausea  1  1/33 (3.03%)  1 2/27 (7.41%)  3
Indicates events were collected by systematic assessment
1
Term from vocabulary, DAIDS
A lower than expected event rate for the primary outcome measure was the major trial limitation.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Dennis Hoffman, Ph.D., Sr. Research Scientist
Organization: Retina Foundation of the Southwest
Phone: 214-363-3911 ext 125
Responsible Party: Dennis Hoffman, Retina Foundation of the Southwest
ClinicalTrials.gov Identifier: NCT00100230     History of Changes
Other Study ID Numbers: 2543
FD-R-002543
5R01FD002543 ( U.S. FDA Grant/Contract )
C-TX02-0704-0274 ( Other Grant/Funding Number: Foundation Fighting Blindness )
First Submitted: December 27, 2004
First Posted: December 28, 2004
Results First Submitted: August 25, 2014
Results First Posted: March 17, 2015
Last Update Posted: March 17, 2015