Trial Outcomes & Findings for Vitamin A Palmitate Supplementation in Patients With Reticular Pseudodrusen (RPD) and Delayed Dark Adaptation (NCT NCT03478878)
NCT ID: NCT03478878
Last Updated: 2025-04-16
Results Overview
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%.
Recruitment status
COMPLETED
Study phase
EARLY_PHASE1
Target enrollment
9 participants
Primary outcome timeframe
Baseline to Month 2 for Cohort 1 and Baseline to Month 1 for Cohort 2
Results posted on
2025-04-16
Participant Flow
Up to eighteen (18) participants (8 participants in Cohort 1 and up to 10 participants in Cohort 2) with reticular pseudodrusen (RPD) and abnormal dark adaptation may be enrolled. At the National Eye Institute, enrollment for Cohort 1 closed on January 10, 2019 and enrollment for Cohort 2 was suspended on August 31, 2023.
Participants met all inclusion criteria and none of the exclusion criteria for at least one 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 January 10, 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.
Participant milestones
| Measure |
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.
|
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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|---|---|---|
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Overall Study
STARTED
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7
|
2
|
|
Overall Study
Completed Treatment Completion Visit
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7
|
2
|
|
Overall Study
COMPLETED
|
7
|
2
|
|
Overall Study
NOT COMPLETED
|
0
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0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
The exact age of participants over 89 years of age are not provided as this information is considered Protected Health Information. Therefore, participants over 89 years of age (n=1) are excluded.
Baseline characteristics by cohort
| Measure |
Cohort 1
n=7 Participants
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.
|
Cohort 2
n=2 Participants
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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Total
n=9 Participants
Total of all reporting groups
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|---|---|---|---|
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Age, Continuous
|
71.5 years
STANDARD_DEVIATION 9.61 • n=6 Participants • The exact age of participants over 89 years of age are not provided as this information is considered Protected Health Information. Therefore, participants over 89 years of age (n=1) are excluded.
|
75.0 years
STANDARD_DEVIATION 15.73 • n=2 Participants • The exact age of participants over 89 years of age are not provided as this information is considered Protected Health Information. Therefore, participants over 89 years of age (n=1) are excluded.
|
72.4 years
STANDARD_DEVIATION 9.58 • n=8 Participants • The exact age of participants over 89 years of age are not provided as this information is considered Protected Health Information. Therefore, participants over 89 years of age (n=1) are excluded.
|
|
Age, Customized
Age, Categorical · <= 18 years
|
0 Participants
n=7 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=9 Participants
|
|
Age, Customized
Age, Categorical · Between 18 and 65 years
|
3 Participants
n=7 Participants
|
0 Participants
n=2 Participants
|
3 Participants
n=9 Participants
|
|
Age, Customized
Age, Categorical · >= 65 years and <= 89 years
|
3 Participants
n=7 Participants
|
2 Participants
n=2 Participants
|
5 Participants
n=9 Participants
|
|
Age, Customized
Age, Categorical · > 89 years
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1 Participants
n=7 Participants
|
0 Participants
n=2 Participants
|
1 Participants
n=9 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=7 Participants
|
1 Participants
n=2 Participants
|
5 Participants
n=9 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=7 Participants
|
1 Participants
n=2 Participants
|
4 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=7 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
7 Participants
n=7 Participants
|
2 Participants
n=2 Participants
|
9 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=7 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=7 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=7 Participants
|
0 Participants
n=2 Participants
|
1 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=7 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=7 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=7 Participants
|
2 Participants
n=2 Participants
|
8 Participants
n=9 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=7 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=7 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=9 Participants
|
|
Region of Enrollment
United States
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7 participants
n=7 Participants
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2 participants
n=2 Participants
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9 participants
n=9 Participants
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Dark Adaptation Rod Intercept Time
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16.83 minutes
STANDARD_DEVIATION 11.894 • n=7 Participants
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24.30 minutes
STANDARD_DEVIATION 16.405 • n=2 Participants
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18.49 minutes
STANDARD_DEVIATION 12.272 • n=9 Participants
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Low Luminance Visual Acuity (LLVA) Total Letters Read
|
66.0 letters read
STANDARD_DEVIATION 21.01 • n=7 Participants
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77.0 letters read
STANDARD_DEVIATION 4.24 • n=2 Participants
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68.4 letters read
STANDARD_DEVIATION 18.89 • n=9 Participants
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Low Luminance Questionnaire (LLQ) Composite Score
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74.87 score on a scale
STANDARD_DEVIATION 20.180 • n=7 Participants
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93.54 score on a scale
STANDARD_DEVIATION 5.500 • n=2 Participants
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79.02 score on a scale
STANDARD_DEVIATION 19.416 • n=9 Participants
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Low Luminance Questionnaire (LLQ) Driving Subscale Score
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57.86 score on a scale
STANDARD_DEVIATION 32.385 • n=7 Participants
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87.50 score on a scale
STANDARD_DEVIATION 10.607 • n=2 Participants
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64.44 score on a scale
STANDARD_DEVIATION 31.169 • n=9 Participants
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|
Low Luminance Questionnaire (LLQ) Extreme Lighting Subscale Score
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64.29 score on a scale
STANDARD_DEVIATION 27.884 • n=7 Participants
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86.46 score on a scale
STANDARD_DEVIATION 10.312 • n=2 Participants
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69.21 score on a scale
STANDARD_DEVIATION 26.307 • n=9 Participants
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Low Luminance Questionnaire (LLQ) Mobility Subscale Score
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82.74 score on a scale
STANDARD_DEVIATION 16.035 • n=7 Participants
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97.50 score on a scale
STANDARD_DEVIATION 3.536 • n=2 Participants
|
86.02 score on a scale
STANDARD_DEVIATION 15.387 • n=9 Participants
|
|
Low Luminance Questionnaire (LLQ) Emotional Distress Subscale Score
|
84.82 score on a scale
STANDARD_DEVIATION 13.432 • n=7 Participants
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96.88 score on a scale
STANDARD_DEVIATION 4.419 • n=2 Participants
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87.50 score on a scale
STANDARD_DEVIATION 12.885 • n=9 Participants
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Low Luminance Questionnaire (LLQ) General Dim Lighting Subscale Score
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76.19 score on a scale
STANDARD_DEVIATION 23.535 • n=7 Participants
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92.92 score on a scale
STANDARD_DEVIATION 4.125 • n=2 Participants
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79.91 score on a scale
STANDARD_DEVIATION 21.724 • n=9 Participants
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Low Luminance Questionnaire (LLQ) Peripheral Vision Subscale Score
|
83.33 score on a scale
STANDARD_DEVIATION 18.002 • n=7 Participants
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100.00 score on a scale
STANDARD_DEVIATION 0.000 • n=2 Participants
|
87.04 score on a scale
STANDARD_DEVIATION 17.236 • n=9 Participants
|
PRIMARY outcome
Timeframe: Baseline to Month 2 for Cohort 1 and Baseline to Month 1 for Cohort 2Population: Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit.
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%.
Outcome measures
| Measure |
Cohort 1
n=7 Participants
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.
|
Cohort 2
n=2 Participants
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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|---|---|---|
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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)
Baseline Visit
|
16.83 minutes
Standard Deviation 11.894
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24.30 minutes
Standard Deviation 16.405
|
|
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)
Treatment Completion Visit
|
18.66 minutes
Standard Deviation 14.622
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26.10 minutes
Standard Deviation 19.658
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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)
Change from Baseline at the Treatment Completion Visit
|
1.83 minutes
Standard Deviation 12.193
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1.80 minutes
Standard Deviation 3.253
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SECONDARY outcome
Timeframe: Baseline to Month 3 for Cohort 1 and Baseline to Month 2 for Cohort 2Population: Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit.
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%.
Outcome measures
| Measure |
Cohort 1
n=7 Participants
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.
|
Cohort 2
n=2 Participants
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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|---|---|---|
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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 Visit
|
16.83 minutes
Standard Deviation 11.894
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24.30 minutes
Standard Deviation 16.405
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|
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)
Post-Treatment Follow-Up Visit
|
18.70 minutes
Standard Deviation 11.563
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26.60 minutes
Standard Deviation 18.809
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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)
Change from Baseline at the Post-Treatment Follow-Up Visit
|
1.87 minutes
Standard Deviation 10.244
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2.30 minutes
Standard Deviation 2.404
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SECONDARY outcome
Timeframe: 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 2Population: Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit.
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.
Outcome measures
| Measure |
Cohort 1
n=7 Participants
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.
|
Cohort 2
n=2 Participants
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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|---|---|---|
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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
Baseline Visit
|
66.0 letters read
Standard Deviation 21.01
|
77.0 letters read
Standard Deviation 4.24
|
|
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
|
70.9 letters read
Standard Deviation 10.76
|
76.5 letters read
Standard Deviation 2.12
|
|
Change in Low Luminance Visual Acuity (LLVA) From Baseline in the Study Eye at the Treatment Completion and Post-Treatment Follow-Up Visits
Post-Treatment Follow-Up Visit
|
70.0 letters read
Standard Deviation 10.98
|
78.5 letters read
Standard Deviation 2.12
|
|
Change in Low Luminance Visual Acuity (LLVA) From Baseline in the Study Eye at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Treatment Completion Visit
|
4.9 letters read
Standard Deviation 14.36
|
-0.5 letters read
Standard Deviation 2.12
|
|
Change in Low Luminance Visual Acuity (LLVA) From Baseline in the Study Eye at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Post-Treatment Follow-Up Visit
|
4.0 letters read
Standard Deviation 13.00
|
1.5 letters read
Standard Deviation 2.12
|
SECONDARY outcome
Timeframe: 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 2Population: Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit.
Low Luminance Questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Individual questions are assigned to one of six distinct subscales and are scored in 25-point increments (range=0 to 100, higher values=better outcome). Applicable questions are averaged to produce (weighted) subscale scores (range=0 to 100, higher values=better outcome). (Weighted) Subscale scores are averaged to produce a composite score (range=0 to 100, higher values=better outcome). The mean change in LLQ composite score from baseline at the treatment completion and post-treatment completion visits are descriptively summarized. A positive score change represents improvement in functioning from baseline (i.e., higher values=better outcome), whereas a negative score change represents a decline in functioning from baseline (i.e., lower values=worse outcome).
Outcome measures
| Measure |
Cohort 1
n=7 Participants
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.
|
Cohort 2
n=2 Participants
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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|---|---|---|
|
Change in Low Luminance Questionnaire (LLQ) Composite Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Baseline Visit
|
74.87 score on a scale
Standard Deviation 20.180
|
93.54 score on a scale
Standard Deviation 5.500
|
|
Change in Low Luminance Questionnaire (LLQ) Composite Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Treatment Completion Visit
|
74.19 score on a scale
Standard Deviation 23.676
|
92.74 score on a scale
Standard Deviation 5.647
|
|
Change in Low Luminance Questionnaire (LLQ) Composite Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Post-Treatment Follow-Up Visit
|
76.95 score on a scale
Standard Deviation 16.717
|
90.64 score on a scale
Standard Deviation 8.614
|
|
Change in Low Luminance Questionnaire (LLQ) Composite Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Treatment Completion Visit
|
-0.68 score on a scale
Standard Deviation 7.194
|
-0.80 score on a scale
Standard Deviation 0.147
|
|
Change in Low Luminance Questionnaire (LLQ) Composite Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Post-Treatment Follow-Up Visit
|
2.08 score on a scale
Standard Deviation 9.926
|
-2.90 score on a scale
Standard Deviation 3.115
|
SECONDARY outcome
Timeframe: 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 2Population: Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit.
Low Luminance Questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Individual questions are assigned to one of six distinct subscales and are scored in 25-point increments (range=0 to 100, higher values=better outcome). Applicable questions are averaged to produce (weighted) subscale scores (range=0 to 100, higher values=better outcome). The mean change in LLQ driving subscale score from baseline at the treatment completion and post-treatment completion visits are descriptively summarized. A positive score change represents improvement in functioning from baseline (i.e., higher values=better outcome), whereas a negative score change represents a decline in functioning from baseline (i.e., lower values=worse outcome).
Outcome measures
| Measure |
Cohort 1
n=7 Participants
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.
|
Cohort 2
n=2 Participants
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.
|
|---|---|---|
|
Change in Low Luminance Questionnaire (LLQ) Driving Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Baseline Visit
|
57.86 score on a scale
Standard Deviation 32.385
|
87.50 score on a scale
Standard Deviation 10.607
|
|
Change in Low Luminance Questionnaire (LLQ) Driving Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Treatment Completion Visit
|
60.00 score on a scale
Standard Deviation 37.193
|
80.0 score on a scale
Standard Deviation 21.213
|
|
Change in Low Luminance Questionnaire (LLQ) Driving Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Post-Treatment Follow-Up Visit
|
62.14 score on a scale
Standard Deviation 36.384
|
85.00 score on a scale
Standard Deviation 14.142
|
|
Change in Low Luminance Questionnaire (LLQ) Driving Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Treatment Completion Visit
|
2.14 score on a scale
Standard Deviation 11.852
|
-7.50 score on a scale
Standard Deviation 10.607
|
|
Change in Low Luminance Questionnaire (LLQ) Driving Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Post-Treatment Follow-Up Visit
|
4.29 score on a scale
Standard Deviation 13.048
|
-2.50 score on a scale
Standard Deviation 3.536
|
SECONDARY outcome
Timeframe: 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 2Population: Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit.
Low Luminance Questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Individual questions are assigned to one of six distinct subscales and are scored in 25-point increments (range=0 to 100, higher values=better outcome). Applicable questions are averaged to produce (weighted) subscale scores (range=0 to 100, higher values=better outcome). The mean change in LLQ extreme lighting subscale score from baseline at the treatment completion and post-treatment completion visits are descriptively summarized. A positive score change represents improvement in functioning from baseline (i.e., higher values=better outcome), whereas a negative score change represents a decline in functioning from baseline (i.e., lower values=worse outcome).
Outcome measures
| Measure |
Cohort 1
n=7 Participants
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.
|
Cohort 2
n=2 Participants
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.
|
|---|---|---|
|
Change in Low Luminance Questionnaire (LLQ) Extreme Lighting Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Baseline Visit
|
64.29 score on a scale
Standard Deviation 27.884
|
86.46 score on a scale
Standard Deviation 10.312
|
|
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
|
70.54 score on a scale
Standard Deviation 23.514
|
89.38 score on a scale
Standard Deviation 6.187
|
|
Change in Low Luminance Questionnaire (LLQ) Extreme Lighting Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Post-Treatment Follow-Up Visit
|
67.11 score on a scale
Standard Deviation 24.003
|
86.16 score on a scale
Standard Deviation 10.733
|
|
Change in Low Luminance Questionnaire (LLQ) Extreme Lighting Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Treatment Completion Visit
|
6.25 score on a scale
Standard Deviation 9.200
|
2.92 score on a scale
Standard Deviation 4.125
|
|
Change in Low Luminance Questionnaire (LLQ) Extreme Lighting Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Post-Treatment Follow-Up Visit
|
2.83 score on a scale
Standard Deviation 14.923
|
-0.30 score on a scale
Standard Deviation 0.421
|
SECONDARY outcome
Timeframe: 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 2Population: Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit.
Low Luminance Questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Individual questions are assigned to one of six distinct subscales and are scored in 25-point increments (range=0 to 100, higher values=better outcome). Applicable questions are averaged to produce (weighted) subscale scores (range=0 to 100, higher values=better outcome). The mean change in LLQ mobility subscale score from baseline at the treatment completion and post-treatment completion visits are descriptively summarized. A positive score change represents improvement in functioning from baseline (i.e., higher values=better outcome), whereas a negative score change represents a decline in functioning from baseline (i.e., lower values=worse outcome).
Outcome measures
| Measure |
Cohort 1
n=7 Participants
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.
|
Cohort 2
n=2 Participants
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.
|
|---|---|---|
|
Change in Low Luminance Questionnaire (LLQ) Mobility Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Baseline Visit
|
82.74 score on a scale
Standard Deviation 16.035
|
97.50 score on a scale
Standard Deviation 3.536
|
|
Change in Low Luminance Questionnaire (LLQ) Mobility Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Treatment Completion Visit
|
83.93 score on a scale
Standard Deviation 16.567
|
91.67 score on a scale
Standard Deviation 5.893
|
|
Change in Low Luminance Questionnaire (LLQ) Mobility Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Post-Treatment Follow-Up Visit
|
81.55 score on a scale
Standard Deviation 13.145
|
92.50 score on a scale
Standard Deviation 10.607
|
|
Change in Low Luminance Questionnaire (LLQ) Mobility Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Treatment Completion Visit
|
1.19 score on a scale
Standard Deviation 8.578
|
-5.83 score on a scale
Standard Deviation 2.357
|
|
Change in Low Luminance Questionnaire (LLQ) Mobility Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Post-Treatment Follow-Up Visit
|
-1.19 score on a scale
Standard Deviation 6.682
|
-5.00 score on a scale
Standard Deviation 7.071
|
SECONDARY outcome
Timeframe: 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 2Population: Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit.
Low Luminance Questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Individual questions are assigned to one of six distinct subscales and are scored in 25-point increments (range=0 to 100, higher values=better outcome). Applicable questions are averaged to produce (weighted) subscale scores (range=0 to 100, higher values=better outcome). The mean change in LLQ emotional distress subscale score from baseline at the treatment completion and post-treatment completion visits are descriptively summarized. A positive score change represents improvement in functioning from baseline (i.e., higher values=better outcome), whereas a negative score change represents a decline in functioning from baseline (i.e., lower values=worse outcome).
Outcome measures
| Measure |
Cohort 1
n=7 Participants
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.
|
Cohort 2
n=2 Participants
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.
|
|---|---|---|
|
Change in Low Luminance Questionnaire (LLQ) Emotional Distress Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Baseline Visit
|
84.82 score on a scale
Standard Deviation 13.432
|
96.88 score on a scale
Standard Deviation 4.419
|
|
Change in Low Luminance Questionnaire (LLQ) Emotional Distress Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Treatment Completion Visit
|
79.46 score on a scale
Standard Deviation 20.321
|
100.00 score on a scale
Standard Deviation 0.000
|
|
Change in Low Luminance Questionnaire (LLQ) Emotional Distress Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Post-Treatment Follow-Up Visit
|
93.75 score on a scale
Standard Deviation 9.547
|
96.88 score on a scale
Standard Deviation 4.419
|
|
Change in Low Luminance Questionnaire (LLQ) Emotional Distress Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Treatment Completion Visit
|
-5.36 score on a scale
Standard Deviation 12.721
|
3.13 score on a scale
Standard Deviation 4.419
|
|
Change in Low Luminance Questionnaire (LLQ) Emotional Distress Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Post-Treatment Follow-Up Visit
|
8.93 score on a scale
Standard Deviation 14.369
|
0.00 score on a scale
Standard Deviation 0.000
|
SECONDARY outcome
Timeframe: 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 2Population: Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit.
Low Luminance Questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Individual questions are assigned to one of six distinct subscales and are scored in 25-point increments (range=0 to 100, higher values=better outcome). Applicable questions are averaged to produce (weighted) subscale scores (range=0 to 100, higher values=better outcome). The mean change in LLQ general dim lighting subscale score from baseline at the treatment completion and post-treatment completion visits are descriptively summarized. A positive score change represents improvement in functioning from baseline (i.e., higher values=better outcome), whereas a negative score change represents a decline in functioning from baseline (i.e., lower values=worse outcome).
Outcome measures
| Measure |
Cohort 1
n=7 Participants
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.
|
Cohort 2
n=2 Participants
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.
|
|---|---|---|
|
Change in Low Luminance Questionnaire (LLQ) General Dim Lighting Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Baseline Visit
|
76.19 score on a scale
Standard Deviation 23.535
|
92.92 score on a scale
Standard Deviation 4.125
|
|
Change in Low Luminance Questionnaire (LLQ) General Dim Lighting Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Treatment Completion Visit
|
73.81 score on a scale
Standard Deviation 25.877
|
95.42 score on a scale
Standard Deviation 0.589
|
|
Change in Low Luminance Questionnaire (LLQ) General Dim Lighting Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Post-Treatment Follow-Up Visit
|
75.00 score on a scale
Standard Deviation 23.323
|
83.33 score on a scale
Standard Deviation 11.785
|
|
Change in Low Luminance Questionnaire (LLQ) General Dim Lighting Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Treatment Completion Visit
|
-2.38 score on a scale
Standard Deviation 4.725
|
2.50 score on a scale
Standard Deviation 3.536
|
|
Change in Low Luminance Questionnaire (LLQ) General Dim Lighting Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Post-Treatment Follow-Up Visit
|
-1.19 score on a scale
Standard Deviation 10.949
|
-9.58 score on a scale
Standard Deviation 7.660
|
SECONDARY outcome
Timeframe: 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 2Population: Primary Analysis Population: Participants enrolled who received IP through the treatment completion visit.
Low Luminance Questionnaire (LLQ) is a 32-item questionnaire relating to difficulty with visual function in low luminance settings. Individual questions are assigned to one of six distinct subscales and are scored in 25-point increments (range=0 to 100, higher values=better outcome). Applicable questions are averaged to produce (weighted) subscale scores (range=0 to 100, higher values=better outcome). The mean change in LLQ peripheral vision subscale score from baseline at the treatment completion and post-treatment completion visits are descriptively summarized. A positive score change represents improvement in functioning from baseline (i.e., higher values=better outcome), whereas a negative score change represents a decline in functioning from baseline (i.e., lower values=worse outcome).
Outcome measures
| Measure |
Cohort 1
n=7 Participants
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.
|
Cohort 2
n=2 Participants
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.
|
|---|---|---|
|
Change in Low Luminance Questionnaire (LLQ) Peripheral Vision Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Baseline Visit
|
83.33 score on a scale
Standard Deviation 18.002
|
100.00 score on a scale
Standard Deviation 0.000
|
|
Change in Low Luminance Questionnaire (LLQ) Peripheral Vision Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Treatment Completion Visit
|
77.38 score on a scale
Standard Deviation 28.753
|
100.00 score on a scale
Standard Deviation 0.000
|
|
Change in Low Luminance Questionnaire (LLQ) Peripheral Vision Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Post-Treatment Follow-Up Visit
|
82.14 score on a scale
Standard Deviation 13.113
|
100.00 score on a scale
Standard Deviation 0.000
|
|
Change in Low Luminance Questionnaire (LLQ) Peripheral Vision Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Treatment Completion Visit
|
-5.95 score on a scale
Standard Deviation 14.203
|
0.00 score on a scale
Standard Deviation 0.000
|
|
Change in Low Luminance Questionnaire (LLQ) Peripheral Vision Subscale Score From Baseline at the Treatment Completion and Post-Treatment Follow-Up Visits
Change from Baseline at the Post-Treatment Follow-Up Visit
|
-1.19 score on a scale
Standard Deviation 15.537
|
0.00 score on a scale
Standard Deviation 0.000
|
Adverse Events
Cohort 1
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Cohort 2
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Cohort 1
n=7 participants at risk
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.
|
Cohort 2
n=2 participants at risk
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.
|
|---|---|---|
|
Eye disorders
Eye pruritus
|
14.3%
1/7 • Number of events 1 • Participants enrolled are assessed for AEs starting at Baseline through study completion (Month 3 for Cohort 1 and Month 2 for Cohort 2).
|
0.00%
0/2 • Participants enrolled are assessed for AEs starting at Baseline through study completion (Month 3 for Cohort 1 and Month 2 for Cohort 2).
|
|
Infections and infestations
Pneumonia
|
14.3%
1/7 • Number of events 1 • Participants enrolled are assessed for AEs starting at Baseline through study completion (Month 3 for Cohort 1 and Month 2 for Cohort 2).
|
0.00%
0/2 • Participants enrolled are assessed for AEs starting at Baseline through study completion (Month 3 for Cohort 1 and Month 2 for Cohort 2).
|
|
Skin and subcutaneous tissue disorders
Rash papular
|
14.3%
1/7 • Number of events 1 • Participants enrolled are assessed for AEs starting at Baseline through study completion (Month 3 for Cohort 1 and Month 2 for Cohort 2).
|
0.00%
0/2 • Participants enrolled are assessed for AEs starting at Baseline through study completion (Month 3 for Cohort 1 and Month 2 for Cohort 2).
|
Additional Information
Emily Chew, MD, Principal Investigator, NEI
National Institutes of Health
Phone: 3014966583
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place