Alpha Lipoic Acid in Geographic Atrophy
Condition: Age-Related Macular Degeneration
MeSH terms: Macular Degeneration; Geographic Atrophy; Atrophy
Study type: Interventional
Phase: Phase 1/Phase 2

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.
    

Intervention name(s): alpha lipoic acid; Placebo
Intervention type(s): Drug; Drug
Other intervention name(s): thioctic acid
Intervention MeSH terms: Thioctic Acid
Allocation: Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment