A Phase 2 Randomized, Double-Masked, Placebo-Controlled Clinical Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Elamipretide in Subjects With Age-Related Macular Degeneration With Non-central Geographic Atrophy
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- Age-related Macular Degeneration
- Sponsor
- Stealth BioTherapeutics Inc.
- Enrollment
- 176
- Locations
- 33
- Primary Endpoint
- GA Area Change From Baseline by OCT
- Status
- Completed
- Last Updated
- last year
Overview
Brief Summary
A randomized, double-masked, placebo-controlled study to evaluate the safety, efficacy and pharmacokinetics of elamipretide in subjects with Age-Related Macular Degeneration with non-central Geographic Atrophy.
Detailed Description
This was a randomized, double-masked, placebo controlled study using three periods to evaluate the safety, efficacy and pharmacokinetics of elamipretide in subjects with Age-Related Macular Degeneration with non-central Geographic Atrophy. The total duration of subject participation was up to 54 weeks, including a Screening Period (≤2 weeks), Treatment Period (48 weeks), and Follow-up (4 weeks). 176 eligible subjects were randomized in a 2:1 ratio (elamipretide:placebo) to receive 40 mg elamipretide or placebo. The study drug (i.e., elamipretide or placebo) was administered once daily via SC injection using the elamipretide delivery system during the 48 week Treatment Period. After completion of the 48-week Treatment Period subjects continued to be monitored for safety during the 4-week Follow-up Period and an end of study (EOS) Follow-up Visit was conducted at Week 52.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Adults ≥ 55 years of age with at least 1 eye with AMD with non-central GA as determined by FAF.
- •Ocular conditions-study eye
- •GA in the study eye at the Screening Visit may be multi-focal, but the cumulative GA lesion and size must:
- •be ≥ 0.05 mm2 and ≤ 10.16 mm2 and
- •reside completely within the FAF 30 or 35 degree image.
- •must be at least 150 μm from foveal center with preserved outer retinal structural details
- •No evidence of CNV by history, OCT or FA in the study eye.
- •BCVA by Early Treatment Diabetic Retinopathy Study (ETDRS) score of ≥ 55 letters (Snellen equivalent ≥ 20/70) in the study eye at the Screening Visit and Baseline Visit.
- •LL BCVA by ETDRS score of ≥ 10 letters in the study eye at the Screening Visit and Baseline Visit.
- •LL VA deficit (defined as difference the between BCVA and LL BCVA) of \> 5 letters in the study eye at Screening and Baseline Visits.
Exclusion Criteria
- •Ocular conditions-study eye
- •The absence of observable hyper-FAF at the margins of the GA in the study eye(only for lesions ≥ 0.25mm2)
- •Atrophic retinal disease of causality other than AMD including myopia-related maculopathy and monogenetic macular dystrophies including pattern dystrophy and adult-onset Stargardt disease in the study eye.
- •Presence or diagnosis of exudative AMD or CNV in the study eye.
- •Presence of retinal vein occlusion in the study eye.
- •Presence of diabetic retinopathy (a history of diabetes mellitus without retinopathy is not a criterion for exclusion) in either eye.
- •Presence of vitreous hemorrhage in the study eye.
- •History of retinal detachment in the study eye.
- •History of macular hole (stages 2 to 4) in the study eye.
- •Presence of an epiretinal membrane that causes distortion of the retinal contour in the study eye.
Outcomes
Primary Outcomes
GA Area Change From Baseline by OCT
Time Frame: Baseline and Weeks 12, 24, 36, 48
Geographic atrophy (GA) area: change from baseline as measured by optical coherence tomography (OCT) from Baseline to the end of treatment (EOT; Week 48)
LL BCVA Score Change From Baseline
Time Frame: Baseline and Weeks 4, 8, 12, 24, 36, 48
Change in low luminance best corrected visual acuity (LL BCVA) score from Baseline to the end of treatment (EOT; Week 48) assessment measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale. ETDRS charts present a series of five letters of equal difficulty on each row, with standardized spacing between letters and rows; there is a total of 14 lines (70 letters), with letter size increasing further geometrically and equivalently in every line by a factor of 1.2589 (or 0.1 log unit), moving up the chart. Minimum score of zero, maximum score of 100. Change from baseline: a more negative score is worse outcome, a more positive score is better outcome. A lower score means less letters were read correctly (worse outcome) and a higher score means more letters were read correctly (better outcome).
Secondary Outcomes
- LL RA Change From Baseline(Baseline and Weeks 4,12, 36, 48)
- BCVA Change From Baseline(Baseline and Weeks 4, 8, 12, 24, 36, 48)
- GA Area as Measured by Fundus Autofluorescence (FAF) Change From Baseline(Baseline and Weeks 12, 24, 36, 48)