Retinitis PigmentosaPhase 3NCT00346333
Clinical Trial of Lutein for Patients With Retinitis Pigmentosa Receiving Vitamin A
A Phase 3 study testing Lutein in people with Retinitis Pigmentosa, run by National Eye Institute (NEI) at 1 site in United States.
Trial summary
The purpose of this trial is to determine whether lutein in addition to vitamin A will slow the course of retinitis pigmentosa. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- Lutein
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 240 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2003to Dec 2008
- Phase
- Phase 3
- 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 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Ocular Criteria
- RP, typical forms(i.e. elevated final dark adaptation threshold,retinal arteriolar narrowing,and reduced and delayed full-field ERGs).
- Best-corrected visual acuity 20/100 or better
- HFA program 30-2 total point score >= 250 decibels(dB)to a size V white test light
- No confounding ocular disease such as glaucoma,uveitis,diabetic retinopathy,posterior subcapsular cataract more than 11% of total lens area (ie equivalent to P3 on Lens Opacity Classification System III)and pupil diameter after dilation less than 6 mm.
- Dietary Criteria
Show all 24Show fewer
- Fruit and vegetable intake < 10 servings/d
- Spinach or kale intake < 1 serving/d, i.e. <1/2 cup of cooked spinach or kale per day
- Dietary lutein intake <=5.4 mg/d as estimated from food frequency questionnaire
- No intake of cod liver oil or omega-3 capsules
- Dietary preformed vitamin A intake <= 10,000 IU/d
- Supplement intake <= 5,000 IU/d of Vitamin A and <= 30 IU/d of Vitamin E
- Consumption <= 3 alcoholic beverages/d
- Medical and other criteria
- Age 18-60 y
- Body mass index < 40 and weight >= 5th percentile for age,gender,and height
- Serum retinol level <= 100 micrograms/deciliter and serum retinyl ester level <= 380 nanomoles/Liter
- Serum cholesterol < 300 micrograms/deciliter and serum triglyceride level <400 micrograms/deciliter
- No clinically significant abnormality on blood cell count, glucose level, blood urea nitrogen level, serum lipid panel results or serum liver function profile.
- Not pregnant or planning to become pregnant
- Not smoking currently
- Agreed not to know tablet content or course of condition until the end of the trial.
- No other disease which might affect absorption or metabolism of lutein or vitamin A.
- Only one patient per family was accepted into the study.
Who cannot join
- Women who are pregnant or planning to become pregnant (Vitamin A supplements can increase the risk of birth defects.)
- Current participation in another clinical trial for RP
- Patients with atypical forms such as paravenous RP, pericentral RP, sector RP,unilateral RP,Refsum disease, Bardet-Biedl syndrome, retinitis punctata albescens and cone-rod dystrophy were excluded as were patients with RP and profound congenital deafness.
Trial locations and contacts
1 site in 1 country.
United States1 site
Berman Gund Laboratory for the Study of Retinal Degenerations, Harvard Medical School ,Massachusetts Eye & Ear Infirmary
Boston, Massachusetts, 02114
About this study
Official title: Randomized Clinical Trial for Retinitis Pigmentosa
The purpose of this trial is to determine whether lutein in addition to vitamin A will slow the course of retinitis pigmentosa.
Read the detailed description
Retinitis pigmentosa (RP) is a group of inherited retinal degenerations with a worldwide prevalence of approximately 1 in 4,000. Patients typically report night blindness and difficulty with mid-peripheral visual field in adolescence. As the condition progresses, they lose far peripheral visual field. Most patients have reductions in central vision by 60 years if left untreated. Vitamin A palmitate, 15,000 International Units (IU)/d and an omega-3 rich diet have been shown to slow the progression of this condition among adults with the typical forms.(see Archives of Ophthalmology,111:761-772,1993 ; Archives of Ophthalmology 122: 1306-1314, 2004; Archives of Ophthalmology 130(6):701-711,2013).
The present study was a randomized, controlled, double-masked trial with a planned duration of 5 years.Two hundred and forty adults with the typical forms of RP were assigned to either lutein 12mg/d or a control group. Patients in both groups received 15,000 IU/day of vitamin A palmitate in addition to the supplement under study. Participants agreed not to know the contents of the supplement or their group assignment until the end of the trial. The main outcome measurement was the total point score for the 30-2 program of the Humphrey Field analyzer (HFA). In addition,the total point score for the 60-4 program ,the total point score of the 30-2 and 60-4 programs combined, computer-averaged 30-Hz cone Electroretinogram (ERG) amplitude and Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity were measured annually as secondary endpoints.
- Conditions
- Retinitis Pigmentosa
- Being tested
- Lutein, Cornstarch control
- Keywords
- Retinitis Pigmentosa, Inherited Retinal Degeneration
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LuteinDrug
12mg/d
Cornstarch controlDietary supplement
Daily intake of cornstarch control plus 15,000 IU/d Vitamin A palmitate
| Study group | Role | What they receive |
|---|---|---|
| Lutein plus 15,000 IU/d Vitamin A | Experimental | Daily intake of 12mg of Lutein plus 15,000 IU/d of Vitamin A palmitate Receives: Drug: Lutein |
| Control plus 15,000 IU/d Vitamin A | Placebo comparator | Daily intake of cornstarch control plus 15,000 IU/d Vitamin A palmitate Receives: Dietary Supplement: Cornstarch control |
What is being measured
The primary outcome is the main question the study is designed to answer.
Central Visual Field (vf) Change Assessed Using the 30-2 Program of the Humphrey Field Analyzer (HFA).
Sum of visual field sensitivity readings in decibel(dB) to a size V target out to the 30 degree meridian in each direction of the visual field. The value presented represents annual change in dB over 4 years.
Measured: assessed at each of 4 annual visits after baseline
Secondary outcomes (4)
Mid-peripheral Field Change Assessed With the 60-4 Program of the Humphrey Field Analyzer.
Sum of visual field sensitivity readings in dB to a size V target from the 30 degree meridian to the 60 degree meridian in each direction of the visual field. The value presented represents annual change in dB over 4 years.
Measured: assessed at each of 4 annual visits after baseline
Total Field Change Assessed by the Combined 30-2 and 60-4 Programs of the Humphrey Field Analyzer.
Sum of visual field sensitivity readings in dB to a size V target obtained with the 30-2 and 60-4 programs of the Humphrey Field Analyzer combined for those patients on whom both measures were available.
Measured: assessed at each of 4 annual visits after baseline
Annual Change in 30 Hertz(Hz)Electroretinogram(ERG )Amplitude in Natural Log (ln) Microvolts/yr Over a 4 Year Period.
Computer averaged 30 Hz ERG amplitudes in microvolts for those with initial amplitudes of >= 0.68 microvolts. Presented on the ln scale.
Measured: assessed at each of 4 annual visits after baseline
Early Treatment Diabetic Retinopathy Study (ETDRS) Visual Acuity
Annual change in number of letters read.
Measured: assessed at each of 4 annual visits after baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jun 2006 (estimated)
- Study startJul 2003
- Primary completionDec 2008
- Study completionDec 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Retinitis PigmentosaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 240 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Eye Institute (NEI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Eliot L Berson, MD, Study chair, Harvard Medical School (HMS and HSDM)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00346333
- Sponsor's protocol ID
- NEI-126
- Status last verified
- Dec 2013
- First submitted
- 27 Jun 2006
- Last updated
- 31 Jan 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Berson EL, Rosner B, Sandberg MA, Weigel-DiFranco C, Brockhurst RJ, Hayes KC, Johnson EJ, Anderson EJ, Johnson CA, Gaudio AR, Willett WC, Schaefer EJ. Clinical trial of lutein in patients with retinitis pigmentosa receiving vitamin A. Arch Ophthalmol. 2010 Apr;128(4):403-11. doi: 10.1001/archophthalmol.2010.32.
PubMed 20385935Comander J, Weigel DiFranco C, Sanderson K, Place E, Maher M, Zampaglione E, Zhao Y, Huckfeldt RM, Bujakowska KM, Pierce E. Natural history of retinitis pigmentosa based on genotype, vitamin A/E supplementation, and an electroretinogram biomarker. JCI Insight. 2023 Aug 8;8(15):e167546. doi: 10.1172/jci.insight.167546.
PubMed 37261916
Study at a glance
From the registry record, last updated by the sponsor on 31 Jan 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Clinical Trial for Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT00346333
- Acronym / sponsor ID
- NEI-126
- Conditions
- Retinitis Pigmentosa
- Keywords
- Retinitis Pigmentosa, Inherited Retinal Degeneration
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 240 participants (actual)
- Lead sponsor
- National Eye Institute (NEI)US National Institutes of Health
- Study start
- Jul 2003
- Primary completion
- Dec 2008 (actual)
- Study completion
- Dec 2008 (actual)
- First posted
- 29 Jun 2006 (estimated)
- Results
- Posted 31 Jan 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this trial is to determine whether lutein in addition to vitamin A will slow the course of retinitis pigmentosa.
Detailed description
Retinitis pigmentosa (RP) is a group of inherited retinal degenerations with a worldwide prevalence of approximately 1 in 4,000. Patients typically report night blindness and difficulty with mid-peripheral visual field in adolescence. As the condition progresses, they lose far peripheral visual field. Most patients have reductions in central vision by 60 years if left untreated. Vitamin A palmitate, 15,000 International Units (IU)/d and an omega-3 rich diet have been shown to slow the progression of this condition among adults with the typical forms.(see Archives of Ophthalmology,111:761-772,1993 ; Archives of Ophthalmology 122: 1306-1314, 2004; Archives of Ophthalmology 130(6):701-711,2013).
The present study was a randomized, controlled, double-masked trial with a planned duration of 5 years.Two hundred and forty adults with the typical forms of RP were assigned to either lutein 12mg/d or a control group. Patients in both groups received 15,000 IU/day of vitamin A palmitate in addition to the supplement under study. Participants agreed not to know the contents of the supplement or their group assignment until the end of the trial. The main outcome measurement was the total point score for the 30-2 program of the Humphrey Field analyzer (HFA). In addition,the total point score for the 60-4 program ,the total point score of the 30-2 and 60-4 programs combined, computer-averaged 30-Hz cone Electroretinogram (ERG) amplitude and Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity were measured annually as secondary endpoints.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Lutein plus 15,000 IU/d Vitamin A | Experimental | Drug: LuteinDaily intake of 12mg of Lutein plus 15,000 IU/d of Vitamin A palmitate |
| Control plus 15,000 IU/d Vitamin A | Placebo comparator | Dietary Supplement: Cornstarch controlDaily intake of cornstarch control plus 15,000 IU/d Vitamin A palmitate |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (24 listed)
- Ocular Criteria
- RP, typical forms(i.e. elevated final dark adaptation threshold,retinal arteriolar narrowing,and reduced and delayed full-field ERGs).
- Best-corrected visual acuity 20/100 or better
- HFA program 30-2 total point score >= 250 decibels(dB)to a size V white test light
- No confounding ocular disease such as glaucoma,uveitis,diabetic retinopathy,posterior subcapsular cataract more than 11% of total lens area (ie equivalent to P3 on Lens Opacity Classification System III)and pupil diameter after dilation less than 6 mm.
Key exclusion criteria (3 listed)
- Women who are pregnant or planning to become pregnant (Vitamin A supplements can increase the risk of birth defects.)
- Current participation in another clinical trial for RP
- Patients with atypical forms such as paravenous RP, pericentral RP, sector RP,unilateral RP,Refsum disease, Bardet-Biedl syndrome, retinitis punctata albescens and cone-rod dystrophy were excluded as were patients with RP and profound congenital deafness.
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 |
|---|---|---|
| Lutein12mg/d | Drug | Lutein plus 15,000 IU/d Vitamin A |
| Cornstarch controlDaily intake of cornstarch control plus 15,000 IU/d Vitamin A palmitate | Dietary supplement | Control plus 15,000 IU/d Vitamin A |
Primary outcome
Central Visual Field (vf) Change Assessed Using the 30-2 Program of the Humphrey Field Analyzer (HFA).
Time frame: assessed at each of 4 annual visits after baseline
Sum of visual field sensitivity readings in decibel(dB) to a size V target out to the 30 degree meridian in each direction of the visual field. The value presented represents annual change in dB over 4 years.
4 secondary outcomes
Mid-peripheral Field Change Assessed With the 60-4 Program of the Humphrey Field Analyzer.
Time frame: assessed at each of 4 annual visits after baseline
Total Field Change Assessed by the Combined 30-2 and 60-4 Programs of the Humphrey Field Analyzer.
Time frame: assessed at each of 4 annual visits after baseline
Annual Change in 30 Hertz(Hz)Electroretinogram(ERG )Amplitude in Natural Log (ln) Microvolts/yr Over a 4 Year Period.
Time frame: assessed at each of 4 annual visits after baseline
Early Treatment Diabetic Retinopathy Study (ETDRS) Visual Acuity
Time frame: assessed at each of 4 annual visits after baseline
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jan 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 20385935Berson EL, Rosner B, Sandberg MA, Weigel-DiFranco C, Brockhurst RJ, Hayes KC, Johnson EJ, Anderson EJ, Johnson CA, Gaudio AR, Willett WC, Schaefer EJ. Clinical trial of lutein in patients with retinitis pigmentosa receiving vitamin A. Arch Ophthalmol. 2010 Apr;128(4):403-11. doi: 10.1001/archophthalmol.2010.32.
- Derived from this study: PubMed 37261916Comander J, Weigel DiFranco C, Sanderson K, Place E, Maher M, Zampaglione E, Zhao Y, Huckfeldt RM, Bujakowska KM, Pierce E. Natural history of retinitis pigmentosa based on genotype, vitamin A/E supplementation, and an electroretinogram biomarker. JCI Insight. 2023 Aug 8;8(15):e167546. doi: 10.1172/jci.insight.167546.
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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.
Eliot L Berson, MD study chair
Harvard Medical School (HMS and HSDM)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Lutein in people with Retinitis Pigmentosa, 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 24 inclusion and 3 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 31 Jan 2014. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 31 Jan 2014). 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?
Central Visual Field (vf) Change Assessed Using the 30-2 Program of the Humphrey Field Analyzer (HFA). (time frame: assessed at each of 4 annual visits after baseline). 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 NCT00346333, which also lists 2 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 31 Jan 2014.