NCT02613572

Alpha Lipoic Acid in Geographic Atrophy

Evaluation of Lipoic Acid as a Treatment for Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD): Phase I- Tolerability Study and Phase II Pilot- Determine the Effects of ALA on the Progression of Geographic Atrophy (GA) in Patients With Age-related Macular Degeneration (AMD).

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 68 (actual)
• Sponsor: University of Pennsylvania · Academic / Other
• Sex: All
• Age: 55 Years – 90 Years
• Healthy volunteers: Accepted

Summary

Because of its iron-chelating and antioxidant properties, alpha lipoic acid may be a treatment for geographic atrophy (GA) secondary to age-related macular degeneration. There is ample published data about the safety and pharmacokinetics of alpha lipoic acid in adults. However, there is not much data on the safety and tolerability of higher doses of alpha lipoic acid in the elderly population. The purpose of Phase I of this protocol is to determine if there are safety/tolerability concerns seen when higher doses of alpha lipoic acid are taken by subjects 65 years of age or older. The objective of Phase 2 of this protocol is to determine the effects of ALA on the progression of GA in subjects with AMD. The central hypothesis, based on the existing literature, is that oral ALA reduces the rate of enlargement of GA in AMD subjects. The rationale is that the antioxidant and iron chelating effects of ALA will slow down one of the major pathways responsible for GA progression.

Detailed description

Phase I (Apr 2016 completed): 15 subjects, 65 years of age or older will take alpha lipoic acid on the following schedule: 600 mg once daily with a meal for 5 days. If tolerated, then the subject will then take 800 mg once daily with a meal for 5 days. If tolerated, then the subject will then take 1200 mg once daily with a meal for 5 days. Phase II: Randomized, double-blind placebo controlled pilot trial. Upon the completion of the dose tolerability test, we plan to enroll 50 subjects into a randomized, double-blind, placebo-controlled trial. Subjects will be randomized (1:1) into one of two study arms: placebo capsules and ALA 1200 mg orally once daily, assuming that 1200 mg is well tolerated by subjects in Phase 1. If 1200 mg is not well-tolerated based on Phase 1 data, then the highest tolerable dose will be used. Four clinical sites are planned and the enrollment period is estimated to be 6 months. The primary endpoint is the mean rate of change of the area of GA in the study eye from baseline to 18 months as evaluated by fundus autofluorescence. Subjects will have a refracted electronic visual acuity and dilated exam at baseline, 6 months, 12 months, and 18 months. The study will be conducted on an outpatient basis and study visits will last approximately 2-3 hours. Two weeks after the 18 months study visit, the subject will be contacted to share with the investigators adverse events that developed after completing the 18 month visit. The Investigator shall ensure each subject has a follow-up eye exam scheduled within 6 months.

Conditions

• Age-Related Macular Degeneration

Interventions

Type	Name	Description
DRUG	alpha lipoic acid
DRUG	Placebo

Timeline

• Start date: 2015-11-01
• Primary completion: 2019-02-15
• Completion: 2019-03-01
• First posted: 2015-11-24
• Last updated: 2020-08-06
• Results posted: 2020-08-06

Locations

5 sites across 1 country: United States