Elamipretide for Age-Related Macular Degeneration
(ReNEW Trial)
Recruiting in Palo Alto (17 mi)
+61 other locations
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: Stealth BioTherapeutics Inc.
Must not be taking: Plaquenil, Tamoxifen, Digoxin, others
Disqualifiers: Diabetic retinopathy, Organ transplant, Cancer, others
Pivotal Trial (Near Approval)
Prior Safety Data
Approved in 1 Jurisdiction
Trial Summary
What is the purpose of this trial?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.
Do I need to stop my current medications for this trial?
The trial does not specify if you need to stop taking your current medications. However, you cannot participate if you are using medications known to be toxic to the eye, like chloroquine or tamoxifen.
What data supports the effectiveness of the drug Elamipretide for age-related macular degeneration?
The Phase 1 Clinical Trial of Elamipretide in Dry Age-Related Macular Degeneration and Noncentral Geographic Atrophy (ReCLAIM NCGA Study) assessed the safety and feasibility of Elamipretide, with exploratory analyses on changes in visual function, suggesting potential benefits in visual improvement.
12345How is the drug Elamipretide unique for treating age-related macular degeneration?
Elamipretide is unique because it targets mitochondria (the energy-producing parts of cells) to improve their function, which may help slow or reverse visual decline in age-related macular degeneration. Unlike other treatments that focus on reducing inflammation or preventing blood vessel growth, Elamipretide specifically enhances mitochondrial efficiency, potentially offering a novel approach to managing this condition.
26789Eligibility Criteria
This trial is for people with dry age-related macular degeneration (AMD). Participants should have a specific level of vision loss and clear enough eyes for imaging. They must be able to take daily injections or have someone who can administer them. Those with certain other eye conditions or treatments are excluded.Inclusion Criteria
Able to provide informed consent and willing to comply with all trial conditions
I can take the study medication myself or have someone who can do it for me.
I agree to use birth control if I can have children.
+8 more
Exclusion Criteria
Absence of observable hyper-FAF at the margins of the GA in the study eye
My vision loss is not due to AMD.
My kidney function is severely reduced.
+10 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive once daily subcutaneous doses of 40mg elamipretide or placebo for 96 weeks
96 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
Participant Groups
The study tests the effectiveness and safety of elamipretide, administered through daily shots, against a placebo in slowing down photoreceptor loss in the retina due to dry AMD. The comparison will help determine if elamipretide can benefit patients with this condition.
2Treatment groups
Experimental Treatment
Placebo Group
Group I: ElamipretideExperimental Treatment1 Intervention
Subjects will receive once daily subcutaneous doses of 40mg elamipretide for 96 weeks
Group II: PlaceboPlacebo Group1 Intervention
Subjects will receive once daily subcutaneous doses of placebo for 96 weeks
Elamipretide is already approved in United States for the following indications:
🇺🇸 Approved in United States as Elamipretide for:
- Pending FDA approval for Barth syndrome and primary mitochondrial myopathy
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Barnet Dulaney Perkins Eye CenterSun City, AZ
Retina Associates of Southern CaliforniaHuntington Beach, CA
Orange County Retinal Medical GroupSanta Ana, CA
Blue Ocean Clinical Research CenterClearwater, FL
More Trial Locations
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Who Is Running the Clinical Trial?
Stealth BioTherapeutics Inc.Lead Sponsor
References
Impact of Modifying Abicipar Manufacturing Process in Patients with Neovascular Age-Related Macular Degeneration: MAPLE Study Results. [2023]To evaluate the impact of modifying the abicipar pegol (abicipar) manufacturing process on the safety and treatment effect of abicipar in patients with neovascular age-related macular degeneration (nAMD).
Phase 1 Clinical Trial of Elamipretide in Dry Age-Related Macular Degeneration and Noncentral Geographic Atrophy: ReCLAIM NCGA Study. [2023]Assess the safety, tolerability, and feasibility of subcutaneous administration of the mitochondrial-targeted drug elamipretide in patients with dry age-related macular degeneration (AMD) and noncentral geographic atrophy (NCGA) and to perform exploratory analyses of change in visual function.
Evaluation of Abicipar Pegol (an Anti-VEGF DARPin Therapeutic) in Patients With Neovascular Age-Related Macular Degeneration: Studies in Japan and the United States. [2019]To evaluate comparability of abicipar pegol (abicipar) effects in patients with treatment-naïve neovascular age-related macular degeneration (nAMD) in Japan and the United States.
HIGH-DOSE HIGH-FREQUENCY AFLIBERCEPT FOR RECALCITRANT NEOVASCULAR AGE-RELATED MACULAR DEGENERATION. [2019]To determine the efficacy of monthly (0.1 mL/4 mg) aflibercept for refractory neovascular age-related macular degeneration (wet age-related macular degeneration).
Long-term outcome of intravitreal pegaptanib sodium as maintenance therapy in Japanese patients with neovascular age-related macular degeneration. [2022]To evaluate the results of a 3-year follow-up of intravitreal pegaptanib sodium injection as maintenance therapy for the treatment of neovascular age-related macular degeneration (AMD) in Japanese patients.
Repurposing Drugs for Treatment of Age-Related Macular Degeneration. [2023]The need for new drugs to treat dry forms of age-related macular degeneration remains high. A promising approach is repurposing of FDA-approved medications to treat AMD. Databases containing medical and drug records allow for retroactive identification of drugs whose use correlates with reduced AMD diagnosis. This short review summarizes progress in several classes of drugs considered for repurposing: GPR-143 agonists (L-DOPA), anti-diabetic drugs (metformin, acarbose, empagliflozin, fenofibrate), mitochondrial activators (PU-91), and serotonin pathway drugs (fluoxetine, flibanserin, xaliproden, buspirone). The promises and caveats of repurposing are discussed herein.
Treatment of age-related visual impairment with a peptide acting on mitochondria. [2022]Age-related visual decline and disease due to neural dysfunction are major sources of disability that have resisted effective treatment. In light of evidence that visual impairment and mitochondrial dysfunction advance with age, we characterized age-related decline of spatial visual function in mice and investigated whether treatment of aged mice with the mitochondrion-penetrating peptide elamipretide that has been reported to improve mitochondrial function, would improve it. Impaired photopic acuity measured by using a virtual optokinetic system emerged near 18 months and declined to ∼40% below normal by 34 months. Daily application of the synthetic peptide elamipretide, which has high selectivity for mitochondrial membranes that contain cardiolipin and promotes efficient electron transfer, was able to mitigate visual decline from 18 months onwards. Daily application from 24 months onwards, i.e. when acuity had reduced by ∼16%, reversed visual decline and normalized function within 2 months. Recovered function persisted for at least 3 months after treatment was withdrawn and a single treatment at 24 months delayed subsequent visual decline. Elamipretide applied daily from 32 months onwards took longer to take effect, but substantial improvement was found within 2 months. The effects of age and elamipretide treatment on contrast sensitivity were similar to those on acuity, systemic and eye drop applications of elamipretide had comparable effects, scotopic spatial visual function was largely unaffected by age or treatment, and altered function was independent of variation in optical clarity. These data indicate that elamipretide treatment adaptively alters the aging visual system. They also provide a rationale to investigate whether mitochondrial dysfunction is a treatable pathophysiology of human visual aging and age-related visual disease.
Pegaptanib: the first antiangiogenic agent approved for neovascular macular degeneration. [2019]Age-related macular degeneration is the leading cause of irreversible visual loss in the industrialised world. Angiogenesis underlies the neovascularisation, and vascular endothelial growth factor (VEGF) is an angiogenesis growth factor. In the VEGF Inhibition Study in Ocular Neovascularisation-1 (VISION-1) trial, pegaptanib (an aptamer inhibitor of VEGF) was tested in neovascular age-related macular degeneration. The 1186 patients received a sham injection or intravitreous injection of pegaptanib (0.3, 1.0 or 3.0 mg) every 6 weeks over a period of 48 weeks. The primary end point was the proportion of patients who lost
New approaches and potential treatments for dry age-related macular degeneration. [2022]Emerging treatments for dry age-related macular degeneration (AMD) and geographic atrophy focus on two strategies that target components involved in physiopathological pathways: prevention of photoreceptors and retinal pigment epithelium loss (neuroprotection induction, oxidative damage prevention, and visual cycle modification) and suppression of inflammation. Neuroprotective drugs, such as ciliary neurotrophic factor, brimonidine tartrate, tandospirone, and anti-amyloid β antibodies, aim to prevent apoptosis of retinal cells. Oxidative stress and depletion of essential micronutrients are targeted by the Age-Related Eye Disease Study (AREDS) formulation. Visual cycle modulators reduce the activity of the photoreceptors and retinal accumulation of toxic fluorophores and lipofuscin. Eyes with dry age-related macular degeneration present chronic inflammation and potential treatments include corticosteroid and complement inhibition. We review the current concepts and rationale of dry age-related macular degeneration treatment that will most likely include a combination of drugs targeting different pathways involved in the development and progression of age-related macular degeneration.