Trial Outcomes & Findings for DHA and X-Linked Retinitis Pigmentosa (NCT NCT00100230)
NCT ID: NCT00100230
Last Updated: 2015-03-17
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
78 participants
Primary outcome timeframe
4 years
Results posted on
2015-03-17
Participant Flow
Participant milestones
| Measure |
1. Docosahexaenoic Acid (DHA) Arm
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 Arm
Corn/soy oil placebo (oils not containing DHA); dosage based on body weight for 4-year trial
|
|---|---|---|
|
Overall Study
STARTED
|
41
|
37
|
|
Overall Study
COMPLETED
|
29
|
22
|
|
Overall Study
NOT COMPLETED
|
12
|
15
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
DHA and X-Linked Retinitis Pigmentosa
Baseline characteristics by cohort
| Measure |
1. Docosahexaenoic Acid (DHA) Arm
n=41 Participants
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 Arm
n=37 Participants
Corn/soy oil placebo (oils not containing DHA); dosage based on body weight for 4-year trial
|
Total
n=78 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
Age
|
16.7 years
STANDARD_DEVIATION 7.8 • n=5 Participants
|
16.2 years
STANDARD_DEVIATION 6.6 • n=7 Participants
|
16.7 years
STANDARD_DEVIATION 7.1 • n=5 Participants
|
|
Sex/Gender, Customized
male
|
41 participants
n=5 Participants
|
37 participants
n=7 Participants
|
78 participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
37 Participants
n=5 Participants
|
33 Participants
n=7 Participants
|
70 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
37 Participants
n=5 Participants
|
36 Participants
n=7 Participants
|
73 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
38 participants
n=5 Participants
|
35 participants
n=7 Participants
|
73 participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
3 participants
n=5 Participants
|
2 participants
n=7 Participants
|
5 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 4 yearsPopulation: Number of participants completing at least one year of trial (i.e., modified intent to treat cohort)
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.
Outcome measures
| Measure |
DHA (Docosahexaenoic Acid)
n=33 Participants
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
|
Placebo (Corn/Soy Oil)
n=27 Participants
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
|
|---|---|---|
|
Rate of LOSS of 31 Hertz Cone Electroretinographic Function
|
0.028 log microvolts/year
Standard Error 0.001
|
0.022 log microvolts/year
Standard Error 0.002
|
SECONDARY outcome
Timeframe: 4 yearsPopulation: Number of participants completing at least one year of trial (i.e., modified intent to treat cohort)
Hypothesis #1: Elevation of red blood cell-docosahexaenoic acid levels will slow the progressive loss of rod electroretinographic response in this 4-year trial.
Outcome measures
| Measure |
DHA (Docosahexaenoic Acid)
n=33 Participants
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
|
Placebo (Corn/Soy Oil)
n=27 Participants
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
|
|---|---|---|
|
Rate of LOSS of Rod Electroretinographic Function
|
0.010 change in amplitude, log microvolts/year
Standard Error 0.001
|
0.023 change in amplitude, log microvolts/year
Standard Error 0.001
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 4 yearsPopulation: Only participants completing at least one year of trial
Hypothesis: Elevation of red blood cell-docosahexaenoic acid levels will slow the progressive loss of peripheral visual fields in this 4-year trial.
Outcome measures
| Measure |
DHA (Docosahexaenoic Acid)
n=33 Participants
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
|
Placebo (Corn/Soy Oil)
n=27 Participants
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
|
|---|---|---|
|
Loss of Peripheral Visual Fields
|
50.4 decibels (dB)
Standard Error 3.2
|
112.8 decibels (dB)
Standard Error 2.0
|
Adverse Events
1. Docosahexaenoic Acid (DHA) Arm
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Corn/Soy Oil Placebo Arm
Serious events: 0 serious events
Other events: 16 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
1. Docosahexaenoic Acid (DHA) Arm
n=33 participants at risk
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 Arm
n=27 participants at risk
Corn/soy oil placebo (oils not containing DHA); dosage based on body weight for 4-year trial
|
|---|---|---|
|
Gastrointestinal disorders
Stomach ache
|
6.1%
2/33 • Number of events 2 • 4 years
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.
|
3.7%
1/27 • Number of events 1 • 4 years
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.
|
|
Gastrointestinal disorders
Nausea
|
3.0%
1/33 • Number of events 1 • 4 years
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.
|
7.4%
2/27 • Number of events 3 • 4 years
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.
|
|
Blood and lymphatic system disorders
Low HDL cholesterol
|
6.1%
2/33 • Number of events 2 • 4 years
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.
|
18.5%
5/27 • Number of events 5 • 4 years
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.
|
|
Blood and lymphatic system disorders
elevated LDL cholesterol
|
6.1%
2/33 • Number of events 2 • 4 years
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.
|
0.00%
0/27 • 4 years
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.
|
|
Blood and lymphatic system disorders
elevated total cholesterol
|
6.1%
2/33 • Number of events 2 • 4 years
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.
|
3.7%
1/27 • Number of events 1 • 4 years
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.
|
|
Blood and lymphatic system disorders
elevated potassium
|
0.00%
0/33 • 4 years
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.
|
14.8%
4/27 • Number of events 4 • 4 years
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.
|
|
Blood and lymphatic system disorders
elevated glucose
|
0.00%
0/33 • 4 years
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.
|
11.1%
3/27 • Number of events 3 • 4 years
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.
|
Additional Information
Dennis Hoffman, Ph.D., Sr. Research Scientist
Retina Foundation of the Southwest
Phone: 214-363-3911
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place