Status and phase
Conditions
Treatments
About
The goal of this clinical trial is to evaluate the efficacy, safety and pharmacokinetics of elamipretide in subjects with dry age-related macular degeneration (AMD). The main questions it aims to answer are: what is the rate of change in the macular area of photoreceptor loss in subjects who receive a daily dose of elamipretide compared with those who receive a look-alike substance that contains no active drug, and what is the safety and tolerability of elamipretide daily subcutaneous injections. Participants will receive either once daily subcutaneous doses of 40mg elamipretide or placebo and the two treatment groups will be compared.
Full description
The ReNEW (SPIAM-301) trial is a phase 3, randomized, double-masked, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once daily subcutaneous (SC) injection of elamipretide in subjects who have dry AMD. Subjects will be randomized (2:1) to once daily SC doses of 40 mg elamipretide, or placebo for 96 weeks of treatment by a central randomization and stratified by SD-OCT device type (Heidelberg SPECTRALIS®, ZEISS CIRRUS®) and baseline macular area of photoreceptor loss, defined as an ellipsoid zone-retinal pigment epithelium (EZ-RPE) thickness of 0μm assessed by SD-OCT and ellipsoid zone (EZ) mapping (High Strata ≥ 5.1mm2, Low Strata <5.1mm2).
Primary Objective
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
A subject must meet all the inclusion criteria at the Screening and Baseline Visit (unless otherwise specified) to be eligible for inclusion in the trial.
Adults ≥ 55 years of age with at least 1 eye with dry AMD with photoreceptor loss, as determined at the Screening Visit by the presence of extrafoveal geographic atrophy (GA), as determined by the Reading Center primarily by fundus autofluorescence (FAF). For this trial, extrafoveal GA is defined as:
Ocular conditions - Study Eye:
GA in the study eye at the Screening Visit may be multi-focal, but the cumulative GA lesion and size (by FAF, as determined by the Reading Center) must:
BCVA by Early Treatment Diabetic Retinopathy Study (ETDRS) score of ≥ 55 letters in the study eye
LL BCVA by ETDRS score of ≥ 10 letters in the study eye
LLD (defined as the difference between BCVA and LL BCVA) of > 5 letters in the study eye
Sufficiently clear ocular media, adequate pupillary dilation, fixation to permit quality fundus imaging, and ability to cooperate sufficiently for adequate ophthalmic visual function testing and anatomic assessment in the study eye
Systemic and General Criteria:
Able to administer IMP or have an appropriate designee who can administer the IMP (i.e., a capable family member or a caregiver)
Able to provide informed consent and willing to comply with all site visits, examinations, daily IMP administrations and dosing diary entries, and other conditions of the trial protocol
Women of childbearing potential must agree to use 1 of the following methods of contraception from the date they sign the ICF until 28 days after the last dose of IMP:
Male subjects with female partners of childbearing potential must be willing to use a highly effective method of contraception (e.g., abstinence, dual method of contraception) from the date they sign the ICF until 28 days after the last dose of IMP
Exclusion criteria
Subjects who meet any of the following criteria at the Screening and Baseline Visit (unless otherwise specified) will be excluded from the trial:
Ocular Conditions - Study Eye:
The absence of observable hyper-FAF at the margins of the GA in the study eye at the Screening Visit by the Reading Center
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
Evidence of exudative AMD or CNV in the study eye by history or FA , as determined by the Reading Center
Presence of retinal vein occlusion in the study 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 and/or vitreomacular traction in the study eye that causes distortion of the retinal contour
Presence of any retinal pathology in the study eye that prohibits outer retinal quantification and EZ mapping, as determined at the Screening Visit by the Reading Center
At the Screening Visit, advanced glaucoma resulting in a cup to disc ratio of > 0.8 in the study eye
History of glaucoma filtration surgery or uncontrolled glaucoma at Baseline Visit in the opinion of the Investigator OR currently using ≥ 3 medications (Minimally invasive glaucoma surgeries (e.g., MIGS) are allowable) Note: Combination medications count as 2 medications.
Presence of visually significant cataract OR presence of significant posterior capsular opacity in the setting of pseudophakia Note: Significant cataract is defined as ≥ +3 nuclear sclerosis based upon the scale below or any Posterior Subcapsular Cataract in the study eye. The Sponsor, or its designee, will supply the clinical trial sites with a copy of the standard photographs. Grade Description
Presence of significant keratopathy or any other media or corneal opacity that would cause scattering of light or alter visual function, especially in LL conditions in the study eye
Ocular incisional or laser surgery (including cataract surgery) in the study eye within 90 days before the Baseline Visit
YAG laser capsulotomy in the study eye within 30 days before the Baseline Visit
Aphakia in the study eye
History of vitrectomy surgery, submacular surgery, or any vitreoretinal surgery in the study eye
Prior treatment with Visudyne® (verteporfin) ocular photodynamic therapy, external-beam radiation therapy (for intraocular conditions), or transpupillary thermotherapy in the study eye
History of subthreshold laser treatment or other forms of photobiomodulation for AMD in the study eye
Intravitreal drug delivery in the past 60 days or 5-half-lives from the Baseline Visit of the injected drug whichever is longer (e.g., intravitreal corticosteroid injection, anti-angiogenic drugs, or device implantation) in the study eye
Intravitreal drug delivery of a complement inhibitor in the past 6 months from the Baseline Visit in the study eye
Concurrent disease in the study eye that could require medical or surgical intervention during the trial
Ocular conditions - Either Eye:
Presence or a history of diabetic retinopathy in either eye (a history of diabetes mellitus without retinopathy is not a criterion for exclusion)
History of herpetic infection in either eye
Active uveitis and/or vitritis (grade trace or above) in either eye
History of idiopathic or autoimmune-associated uveitis in either eye
Active infectious conjunctivitis, keratitis, scleritis, or endophthalmitis in either eye
Systemic Conditions:
Has a history of a systemic eosinophilic illness and/or an eosinophil count >1,000 cells x106/L (equivalent to >1 cell x 103/μL) at the Screening Visit
History of solid organ transplant
Any disease or medical condition that in the opinion of the Investigator would prevent the subject from successfully participating in the trial or might confound trial results
Current use of medications known to be toxic to the lens, retina, or optic nerve (e.g., deferoxamine, chloroquine/hydroxychloroquine [Plaquenil®], tamoxifen, phenothiazines, ethambutol, digoxin, and aminoglycosides)
eGFR of < 30 mL/min at the Screening Visit (using the CKD-EPI 2021 formula)
General Conditions:
Participation in other investigational drug or device clinical trials within 30 days or 5 half-lives (whichever is longer) of Screening; or is currently enrolled in a non-interventional clinical trial that, in the opinion of the Investigator, may be potentially confounding to the results of the current trial
Women who are pregnant, planning to become pregnant, or breastfeeding/lactating
History of allergy to fluorescein that is not amenable to treatment
Inability to comply with trial or follow-up procedures
Inability to obtain CFP, FAF, and FA of sufficient quality to be analyzed and interpreted
Active malignancy or any other cancer from which the subject has been cancer-free for < 2 years. Localized squamous or non-invasive basal cell skin carcinomas are allowed, if appropriately treated prior to screening
History of allergic reaction to the investigational drug or any of its components
Prior participation in any elamipretide trial
Primary purpose
Allocation
Interventional model
Masking
360 participants in 2 patient groups, including a placebo group
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Central trial contact
Rekha Sathyanarayana
Data sourced from clinicaltrials.gov
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