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| ID | Type | Description | Link |
|---|---|---|---|
| 18-EI-0067 |
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Background:
Age-related macular degeneration (AMD) is an eye disease. It is the leading cause of vision loss in people over 55 in the U.S. Changes in the eye can make it difficult for the eye to adjust to low light. This is known as dark adaptation. Identifying and watching the early to middle stages of AMD and changes in dark adaptation might help researchers develop new treatments to stop the disease before it becomes severe. Taking vitamin A might help improve vision in people with AMD.
Objectives:
To see if taking 16,000 IU of vitamin A per day improves vision in people with AMD. Also to improve understanding of AMD and associated dark adaptation.
Eligibility:
Adults ages 50 and older with AMD and normal liver function
Design:
Participants will be screened with:
Medical and eye disease history
Eye exam: The pupil will be dilated with eye drops. Pictures will be taken of the retina and the inside of the eye.
Including the screening visit, participants will have at least 5 visits. They will be about once a month over 6 months. Visits include:
Questions about eye problems in certain light
Eye exam
Blood and urine tests
Dark adaptation protocol: Participants will sit at a machine in a dark room. They will look into the machine and push a button when they see a light. This lasts 20-30 minutes.
Participants will take a vitamin A supplement by mouth once a day for 2 months. They will record when they take the pills in a diary.
Objective:
The objective of this study is to investigate the potential efficacy and safety of vitamin A palmitate dosing in improving dark adaptation in participants with age-related macular degeneration (AMD) and abnormal dark adaptation.
Study Population:
The first cohort consists of five participants with AMD who meet the eligibility criteria. The second cohort will consist of five participants with AMD who meet the eligibility criteria. Up to five additional participants may be accrued in the second cohort to account for participants who withdraw from the study prior to receiving one month of study supplementation for a reason unrelated to an adverse reaction. Up to 18 participants may be enrolled in this study.
Design:
This is a prospective, uncontrolled, single center, pilot study to investigate the potential efficacy and safety of vitamin A palmitate dosing in improving dark adaptation in participants with AMD and abnormal dark adaptation. Participants in the first cohort were instructed to take 16,000 IU of vitamin A palmitate daily for two months. Enrollment for Cohort 1 ended on May 24, 2019. Participants in the second cohort will be instructed to take 48,000 IU of vitamin A palmitate daily for one month. Participants in both cohorts will continue in the study for one month after ending Vitamin A supplementation. Participants in Cohort 1 may enroll into Cohort 2 as long as their last intake of vitamin A palmitate was greater than two months prior to their enrollment into Cohort 2.
Outcome Measures:
For each cohort, the primary outcome is the measurement of dark adaptation parameters (thresholds and kinetics) by the following: dark adaptation times as measured by the AdaptDx comparing before and after vitamin A palmitate supplementation and dark adaptation parameters as measured by the Medmont comparing before and after vitamin A palmitate supplementation. The primary outcome will be assessed at Month 2 in the first cohort and Month 1 in the second cohort. For both cohorts, the secondary outcomes include changes in low luminance visual acuity (LLVA) and changes in patient reported outcomes as measured by the low luminance questionnaire (LLQ). The secondary outcomes also include measurement of dark adaptation parameters (thresholds and kinetics) comparing baseline and one month after completing supplementation (Month 3 in Cohort 1 and Month 2 in Cohort 2).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Participants | Experimental | Participants with age-related macular degeneration |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Vitamin A palmitate | Drug | Provide vitamin A to participants with pre/post assessments of vision. |
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| Measure | Description | Time Frame |
|---|---|---|
| Change in Dark Adaptation Rod Intercept Time (RIT) From Baseline in the Study Eye at the Treatment Completion Visit (Month 2 for Cohort 1 and Month 1 for Cohort 2) | The mean change in rod intercept time (RIT) in the study eye at the treatment completion visit from baseline was descriptively summarized and assessed using a Student's paired t-test at a two-sided Type I error rate of 5%. | Baseline to Month 2 for Cohort 1 and Baseline to Month 1 for Cohort 2 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Dark Adaptation Rod Intercept Time (RIT) From Baseline in the Study Eye at the Post-Treatment Follow-Up Visit (Month 3 for Cohort 1 and Month 2 for Cohort 2). | The mean change in rod intercept time (RIT) in the study eye at the post-treatment follow-up visit from baseline was descriptively summarized and assessed using a Student's paired t-test at a two-sided Type I error rate of 5%. | Baseline to Month 3 for Cohort 1 and Baseline to Month 2 for Cohort 2 |
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To be eligible, the following inclusion criteria must be met, where applicable.
Participant must be 50 years of age or older.
Participant must understand and sign the protocol s informed consent document.
Any participant of childbearing potential must be willing to undergo urine pregnancy tests throughout the study.
Any participant of childbearing potential and any participant able to father children must have (or have a partner who has) had a hysterectomy or vasectomy, be completely abstinent from intercourse, or must agree to practice at least one acceptable method of contraception throughout the course of the study and for one week after study supplement discontinuation. Acceptable methods of contraception include:
Participants must agree to notify the study investigator or coordinator if any of their doctors initiate a new prescription medication during the course of this study.
Participant must agree to not take greater than or equal to 8000 IU vitamin A palmitate outside the study supplementation.
For supplementation eligibility, participant must have normal liver function as demonstrated by the Chemistry 20 panel, or have mild abnormalities not above grade 1 as defined by the Common Terminology Criteria for Adverse Events v4.0 (CTCAE).
EXCLUSION CRITERIA:
A participant is not eligible if any of the following exclusion criteria are present:
STUDY EYE ELIGIBILITY CRITERIA:
The participant must have at least one eye meeting all inclusion criteria and none of the exclusion criteria listed below.
STUDY EYE INCLUSION CRITERIA:
STUDY EYE EXCLUSION CRITERIA:
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| Name | Affiliation | Role |
|---|---|---|
| Emily Y Chew, M.D. | National Eye Institute (NEI) | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| National Institutes of Health Clinical Center | Bethesda | Maryland | 20892 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37315571 | Result | Pfau K, Jeffrey BG, Cukras CA. LOW-DOSE SUPPLEMENTATION WITH RETINOL IMPROVES RETINAL FUNCTION IN EYES WITH AGE-RELATED MACULAR DEGENERATION BUT WITHOUT RETICULAR PSEUDODRUSEN. Retina. 2023 Sep 1;43(9):1462-1471. doi: 10.1097/IAE.0000000000003840. |
| Label | URL |
|---|---|
| NIH Clinical Center Detailed Web Page | View source |
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Participants met all inclusion criteria and none of the exclusion criteria for at least on eye as defined in the protocol. If both eyes met the eligibility criteria, the eye with the better visual acuity was chosen as the study eye. Participants enrolled after May 24, 2019 were enrolled into Cohort 2. All eligible participants underwent treatment starting at Baseline. The IP used in the study was vitamin A palmitate and was not anticipated to differentially treat participant eyes.
Up to eighteen (18) participants (8 participants in Cohort 1 and up to 10 participants in Cohort 2) with age-related macular degeneration (AMD) and abnormal dark adaptation may be enrolled. At the National Eye Institute, enrollment for Cohort 1 closed on May 24, 2019 and enrollment for Cohort 2 was suspended on August 31, 2023.
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1 | Oral administration of vitamin A palmitate. Vitamin A palmitate: Participants in Cohort 1 were instructed to take 16,000 international unit of investigational product (IP) daily for two months. At Baseline and Month 1, a one month supply of IP was dispensed to the participant during the study visit. Participants were required to bring their bottles of IP to each appropriate visit for IP counts for compliance monitoring. Participants could complete participation in the study one month after IP supplementation (Month 3). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 6, 2023 | Apr 24, 2024 |
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| Change in Low Luminance Visual Acuity (LLVA) From Baseline in the Study Eye at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance visual acuity (LLVA) in the study eye from baseline at the treatment completion and post-treatment follow-up visits were descriptively summarized. | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
| Change in Low Luminance Questionnaire (LLQ) Composite Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) composite score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores; (weighted) subscale scores are averaged to produce a composite score. | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
| Change in Low Luminance Questionnaire (LLQ) Driving Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) driving subscale score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
| Change in Low Luminance Questionnaire (LLQ) Extreme Lighting Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) extreme lighting subscale score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
| Change in Low Luminance Questionnaire (LLQ) Mobility Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) mobility subscale score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
| Change in Low Luminance Questionnaire (LLQ) Emotional Distress Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) emotional distress subscale score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
| Change in Low Luminance Questionnaire (LLQ) General Dim Lighting Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) general dim lighting subscale score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
| Change in Low Luminance Questionnaire (LLQ) Peripheral Vision Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) peripheral vision subscale score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
| FG001 | Cohort 2 | Oral administration of vitamin A palmitate. Vitamin A palmitate: Participants in Cohort 2 were instructed to take 48,000 international unit of investigational product (IP) daily for one month. Participants in Cohort 1 could enroll into Cohort 2 as long as their last intake of vitamin A palmitate was greater than two months prior to their enrollment into Cohort 2. At Baseline, a one month supply of IP was dispensed to the participant during the study visit. Participants were required to bring their bottles of IP to each appropriate visit for IP counts for compliance monitoring. Participants could complete participation in the study one month after IP supplementation (Month 2). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol. |
| Completed Treatment Completion Visit |
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| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1 | Oral administration of vitamin A palmitate. Vitamin A palmitate: Participants in Cohort 1 were instructed to take 16,000 international unit of investigational product (IP) daily for two months. At Baseline and Month 1, a one month supply of IP was dispensed to the participant during the study visit. Participants were required to bring their bottles of IP to each appropriate visit for IP counts for compliance monitoring. Participants could complete participation in the study one month after IP supplementation (Month 3). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol. |
| BG001 | Cohort 2 | Oral administration of vitamin A palmitate. Vitamin A palmitate: Participants in Cohort 2 were instructed to take 48,000 international unit of investigational product (IP) daily for one month. Participants in Cohort 1 may enroll into Cohort 2 as long as their last intake of vitamin A palmitate was greater than two months prior to their enrollment into Cohort 2. At Baseline, a one month supply of IP was dispensed to the participant during the study visit. Participants were required to bring their bottles of IP to each appropriate visit for IP counts for compliance monitoring. Participants could complete participation in the study one month after IP supplementation (Month 2). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
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| Age, Categorical | Count of Participants | Participants |
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| Age, Continuous | Median | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Dark Adaptation Rod Intercept Time | Baseline measurement of dark adaptation rod intercept time (RIT) in the study eye. | Mean | Standard Deviation | minutes |
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| Low Luminance Visual Acuity (LLVA) Total Letters Read | Baseline measurement of low luminance visual acuity (LLVA) total letters read in the study eye. | Mean | Standard Deviation | letters read |
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| Low Luminance Questionnaire (LLQ) Composite Score | Low luminance questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores; (weighted) subscale scores are averaged to produce a composite score. | Mean | Standard Deviation | score on a scale |
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| Low Luminance Questionnaire (LLQ) Driving Subscale Score | Low luminance questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Mean | Standard Deviation | score on a scale |
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| Low Luminance Questionnaire (LLQ) Extreme Lighting Subscale Score | Low luminance questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Mean | Standard Deviation | score on a scale |
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| Low Luminance Questionnaire (LLQ) Mobility Subscale Score | Low luminance questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Mean | Standard Deviation | score on a scale |
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| Low Luminance Questionnaire (LLQ) Emotional Distress Subscale Score | Low luminance questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Mean | Standard Deviation | score on a scale |
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| Low Luminance Questionnaire (LLQ) General Dim Lighting Subscale Score | Low luminance questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Mean | Standard Deviation | score on a scale |
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| Low Luminance Questionnaire (LLQ) Peripheral Vision Subscale Score | Low luminance questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Mean | Standard Deviation | score on a scale |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Change in Dark Adaptation Rod Intercept Time (RIT) From Baseline in the Study Eye at the Treatment Completion Visit (Month 2 for Cohort 1 and Month 1 for Cohort 2) | The mean change in rod intercept time (RIT) in the study eye at the treatment completion visit from baseline was descriptively summarized and assessed using a Student's paired t-test at a two-sided Type I error rate of 5%. | Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit. | Posted | Mean | Standard Deviation | Minutes | Baseline to Month 2 for Cohort 1 and Baseline to Month 1 for Cohort 2 |
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| Secondary | Change in Dark Adaptation Rod Intercept Time (RIT) From Baseline in the Study Eye at the Post-Treatment Follow-Up Visit (Month 3 for Cohort 1 and Month 2 for Cohort 2). | The mean change in rod intercept time (RIT) in the study eye at the post-treatment follow-up visit from baseline was descriptively summarized and assessed using a Student's paired t-test at a two-sided Type I error rate of 5%. | Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit. | Posted | Mean | Standard Deviation | minutes | Baseline to Month 3 for Cohort 1 and Baseline to Month 2 for Cohort 2 |
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| Secondary | Change in Low Luminance Visual Acuity (LLVA) From Baseline in the Study Eye at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance visual acuity (LLVA) in the study eye from baseline at the treatment completion and post-treatment follow-up visits were descriptively summarized. | Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit. | Posted | Mean | Standard Deviation | letters read | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
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| Secondary | Change in Low Luminance Questionnaire (LLQ) Composite Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) composite score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores; (weighted) subscale scores are averaged to produce a composite score. | Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit. | Posted | Mean | Standard Deviation | score on a scale | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
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| Secondary | Change in Low Luminance Questionnaire (LLQ) Driving Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) driving subscale score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit. | Posted | Mean | Standard Deviation | score on a scale | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
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| Secondary | Change in Low Luminance Questionnaire (LLQ) Extreme Lighting Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) extreme lighting subscale score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit. | Posted | Mean | Standard Deviation | score on a scale | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
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| Secondary | Change in Low Luminance Questionnaire (LLQ) Mobility Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) mobility subscale score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit. | Posted | Mean | Standard Deviation | score on a scale | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
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| Secondary | Change in Low Luminance Questionnaire (LLQ) Emotional Distress Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) emotional distress subscale score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit. | Posted | Mean | Standard Deviation | score on a scale | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
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| Secondary | Change in Low Luminance Questionnaire (LLQ) General Dim Lighting Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) general dim lighting subscale score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit. | Posted | Mean | Standard Deviation | score on a scale | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
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| Secondary | Change in Low Luminance Questionnaire (LLQ) Peripheral Vision Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits | The mean change in low luminance questionnaire (LLQ) peripheral vision subscale score from baseline at the treatment completion and post-treatment completion visits were descriptively summarized. LLQ is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Questions are scored on a range from 0 (indicative of maximum difficulty) to 100 (indicative of no difficulty) in 25 point increments where higher scores indicate better functioning. Each question is assigned to one of six distinct subscales. Applicable questions are averaged to produce (weighted) subscale scores. | Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit. | Posted | Mean | Standard Deviation | score on a scale | Treatment Completion Visit: Month 2 for Cohort 1 and Month 1 for Cohort 2; Post-Treatment Follow-Up Visit: Month 3 for Cohort 1 and Month 2 for Cohort 2 |
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Participants enrolled are assessed for AEs starting at Baseline through study completion (Month 3 for Cohort 1 and Month 2 for Cohort 2).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1 | Oral administration of vitamin A palmitate. Vitamin A palmitate: Participants in Cohort 1 were instructed to take 16,000 international unit of investigational product (IP) daily for two months. At Baseline and Month 1, a one month supply of IP was dispensed to the participant during the study visit. Participants were required to bring their bottles of IP to each appropriate visit for IP counts for compliance monitoring. Participants could complete participation in the study one month after IP supplementation (Month 3). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol. | 0 | 5 | 0 | 5 | 2 | 5 |
| EG001 | Cohort 2 | Oral administration of vitamin A palmitate. Vitamin A palmitate: Participants in Cohort 2 were instructed to take 48,000 international unit of investigational product (IP) daily for one month. Participants in Cohort 1 may enroll into Cohort 2 as long as their last intake of vitamin A palmitate was greater than two months prior to their enrollment into Cohort 2. At Baseline, a one month supply of IP was dispensed to the participant during the study visit. Participants were required to bring their bottles of IP to each appropriate visit for IP counts for compliance monitoring. Participants could complete participation in the study one month after IP supplementation (Month 2). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol. | 0 | 3 | 0 | 3 | 2 | 3 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cataract | Eye disorders | MedDRA 25.1 | Non-systematic Assessment |
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| Eye irritation | Eye disorders | MedDRA 24.1 | Non-systematic Assessment |
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| Lacrimal disorder | Eye disorders | MedDRA 25.0 | Non-systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 25.0 | Non-systematic Assessment |
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| Swelling | General disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Blood pressure increased | Investigations | MedDRA 24.1 | Non-systematic Assessment |
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| Blood uric acid increased | Investigations | MedDRA 26.0 | Non-systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Non-systematic Assessment |
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| Prostatic mass | Reproductive system and breast disorders | MedDRA 25.1 | Non-systematic Assessment |
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| Visual Impairment | Eye disorders | MedDRA 24.1 | Non-systematic Assessment | Natural Progression of Disease |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Emily Chew, MD, Principal Investigator, NEI | National Institutes of Health | 3014966583 | emily.chew@nih.gov |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 11, 2024 | Apr 24, 2024 | SAP_001.pdf |
| ICF | No | No | Yes | Informed Consent Form | Sep 20, 2022 | Apr 24, 2024 | ICF_002.pdf |
| ID | Term |
|---|---|
| C014794 | retinol palmitate |
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| >=65 years |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Change from Baseline at the Treatment Completion Visit |
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| Analysis: Student's paired t-test on study eyes alone at a two-sided Type I error rate of 5%. Null hypothesis: the mean RIT at Baseline = the mean RIT at the treatment completion visit (Month 1 for Cohort 2). | Student's paired t-test | 0.818 | Result of statistical analyses were considered significant if the p-value from the two-sided test was <0.05. No adjustments for multiplicity were made. | Mean Difference (Final Values) | -0.27 | 2-Sided | 95 | -4.66 | 4.12 | The estimate was the difference in mean change in RIT between baseline and the treatment completion visit. | Superiority |
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| OG001 | Cohort 2 | Oral administration of vitamin A palmitate. Vitamin A palmitate: Participants in Cohort 2 were instructed to take 48,000 international unit of investigational product (IP) daily for one month. Participants in Cohort 1 may enroll into Cohort 2 as long as their last intake of vitamin A palmitate was greater than two months prior to their enrollment into Cohort 2. At Baseline, a one month supply of IP was dispensed to the participant during the study visit. Participants were required to bring their bottles of IP to each appropriate visit for IP counts for compliance monitoring. Participants could complete participation in the study one month after IP supplementation (Month 2). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol. |
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| OG001 | Cohort 2 | Oral administration of vitamin A palmitate. Vitamin A palmitate: Participants in Cohort 2 were instructed to take 48,000 international unit of investigational product (IP) daily for one month. Participants in Cohort 1 may enroll into Cohort 2 as long as their last intake of vitamin A palmitate was greater than two months prior to their enrollment into Cohort 2. At Baseline, a one month supply of IP was dispensed to the participant during the study visit. Participants were required to bring their bottles of IP to each appropriate visit for IP counts for compliance monitoring. Participants could complete participation in the study one month after IP supplementation (Month 2). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol. |
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| OG001 | Cohort 2 | Oral administration of vitamin A palmitate. Vitamin A palmitate: Participants in Cohort 2 were instructed to take 48,000 international unit of investigational product (IP) daily for one month. Participants in Cohort 1 may enroll into Cohort 2 as long as their last intake of vitamin A palmitate was greater than two months prior to their enrollment into Cohort 2. At Baseline, a one month supply of IP was dispensed to the participant during the study visit. Participants were required to bring their bottles of IP to each appropriate visit for IP counts for compliance monitoring. Participants could complete participation in the study one month after IP supplementation (Month 2). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol. |
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| OG001 | Cohort 2 | Oral administration of vitamin A palmitate. Vitamin A palmitate: Participants in Cohort 2 were instructed to take 48,000 international unit of investigational product (IP) daily for one month. Participants in Cohort 1 may enroll into Cohort 2 as long as their last intake of vitamin A palmitate was greater than two months prior to their enrollment into Cohort 2. At Baseline, a one month supply of IP was dispensed to the participant during the study visit. Participants were required to bring their bottles of IP to each appropriate visit for IP counts for compliance monitoring. Participants could complete participation in the study one month after IP supplementation (Month 2). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol. |
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| OG001 | Cohort 2 | Oral administration of vitamin A palmitate. Vitamin A palmitate: Participants in Cohort 2 were instructed to take 48,000 international unit of investigational product (IP) daily for one month. Participants in Cohort 1 may enroll into Cohort 2 as long as their last intake of vitamin A palmitate was greater than two months prior to their enrollment into Cohort 2. At Baseline, a one month supply of IP was dispensed to the participant during the study visit. Participants were required to bring their bottles of IP to each appropriate visit for IP counts for compliance monitoring. Participants could complete participation in the study one month after IP supplementation (Month 2). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol. |
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| OG001 | Cohort 2 | Oral administration of vitamin A palmitate. Vitamin A palmitate: Participants in Cohort 2 were instructed to take 48,000 international unit of investigational product (IP) daily for one month. Participants in Cohort 1 may enroll into Cohort 2 as long as their last intake of vitamin A palmitate was greater than two months prior to their enrollment into Cohort 2. At Baseline, a one month supply of IP was dispensed to the participant during the study visit. Participants were required to bring their bottles of IP to each appropriate visit for IP counts for compliance monitoring. Participants could complete participation in the study one month after IP supplementation (Month 2). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol. |
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| OG001 | Cohort 2 | Oral administration of vitamin A palmitate. Vitamin A palmitate: Participants in Cohort 2 were instructed to take 48,000 international unit of investigational product (IP) daily for one month. Participants in Cohort 1 may enroll into Cohort 2 as long as their last intake of vitamin A palmitate was greater than two months prior to their enrollment into Cohort 2. At Baseline, a one month supply of IP was dispensed to the participant during the study visit. Participants were required to bring their bottles of IP to each appropriate visit for IP counts for compliance monitoring. Participants could complete participation in the study one month after IP supplementation (Month 2). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol. |
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