Vitamin A Palmitate Supplementation in People With Age-Related Macular Degeneration (and Without Reticular Pseudodrusen) and Delayed Dark Adaptation

NCT03478865 | Completed Early Phase 1 Results FDA Drug

• 2 other identifiers: 18006718-EI-0067
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

AMD

Keywords

Eye Examinations; Retinal Pigment Epithelium

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (9)

• 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).

  Baseline to Month 3 for Cohort 1 and Baseline to Month 2 for Cohort 2

• Change in Low Luminance Visual Acuity (LLVA) From Baseline in the Study Eye at the Treatment Completion and Post-Treatment Follow-Up Visits

  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

  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

  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

  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

Study Arms (1)

Participants

EXPERIMENTAL

Participants with age-related macular degeneration

Drug: Vitamin A palmitate

Interventions

Vitamin A palmitate DRUG

Provide vitamin A to participants with pre/post assessments of vision.

Participants

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Hormonal contraception (i.e. birth control pills, injected hormones, dermal patch or vaginal ring),
• Intrauterine device,
• Barrier methods (diaphragm, condom) with spermicide, or
• Surgical sterilization (tubal ligation).
• 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).

You may not qualify if:

• Participant is in another investigational study and actively receiving study therapy.
• Participant is unable to comply with study procedures or follow-up visits.
• Participant is already taking vitamin A palmitate supplements greater than or equal to 8,000 IU.
• Participant has a history of vitamin A deficiency.
• Participant has a condition that, in the opinion of the investigator, would preclude participation in the study (e.g., unstable medical status including blood pressure and glycemic control).
• Participant has a history of hepatitis or liver failure.
• Participant has chronic gastrointestinal disease.
• Participant will be excluded if the participant has serologic evidence of an active hepatitis infection.
• Participant was in Cohort 1 and took his/her last dose of vitamin A palmitate less than two months prior to enrolling in Cohort 2.
• STUDY EYE ELIGIBILITY CRITERIA:
• The eye must have a best-corrected ETDRS visual acuity score better than or equal to 20/80 (i.e., equal to or better than 54 letters).
• Participant must have at least one large druse.
• Presence of advanced macular degeneration with central geographic atrophy or choroidal neovascularization.
• Presence of definite reticular pseudodrusen.
• An ocular condition is present (other than retinal vein occlusion) that, in the opinion of the investigator, might alter visual acuity during the course of the study (e.g., vein occlusion, uveitis or other ocular inflammatory disease, neovascular glaucoma, Irvine-Gass Syndrome, etc.).

Sponsors & Collaborators

• National Eye Institute (NEI): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

Related Publications (1)

• 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.

  PMID: 37315571 RESULT

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Interventions

retinol palmitate

Results Point of Contact

• Title: Emily Chew, MD, Principal Investigator, NEI
• Organization: National Institutes of Health

emily.chew@nih.gov

3014966583

Study Officials

• Emily Y Chew, M.D.

  National Eye Institute (NEI)

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 15, 2018
• First Posted: March 27, 2018
• Study Start: April 20, 2018
• Primary Completion: June 16, 2023
• Study Completion: June 16, 2023
• Last Updated: April 15, 2025
• Results First Posted: May 31, 2024

Record last verified: 2024-03

Locations

US (1)