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Evaluating elamipretide for treating dry age-related macular degeneration

ReNEW: A Phase 3, Double-Masked, Placebo-Controlled Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects Who Have Dry Age-Related Macular Degeneration (Dry AMD)

Phase 3 Interventional Stealth BioTherapeutics Inc. · NCT06373731

This study is testing if a new injection called elamipretide can help adults with dry age-related macular degeneration by slowing down vision loss over 96 weeks.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment360 (estimated)
Ages55 Years and up
SexAll
SponsorStealth BioTherapeutics Inc. Industry-sponsored
Drugs / interventionsradiation
Locations63 sites (Phoenix, Arizona and 62 other locations)
Trial IDNCT06373731 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the efficacy, safety, and pharmacokinetics of elamipretide, administered as a daily subcutaneous injection, in adults with dry age-related macular degeneration (AMD). Participants will be randomly assigned to receive either elamipretide or a placebo for 96 weeks, with the primary focus on measuring changes in the macular area of photoreceptor loss. The study will also evaluate the safety and tolerability of the treatment, as well as the pharmacokinetic profile of elamipretide and its metabolites.

Who should consider this trial

Good fit: Ideal candidates are adults aged 55 and older with at least one eye exhibiting dry AMD and photoreceptor loss.

Not a fit: Patients with advanced forms of AMD or those with significant ocular conditions affecting the study eye may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could slow or prevent vision loss in patients with dry age-related macular degeneration.

How similar studies have performed: Previous studies have shown promise with similar approaches, but this specific treatment is being evaluated in a phase 3 setting, indicating a significant step in its development.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

1. 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:

   1. well-demarcated area(s) of GA
   2. All GA lesions must be at least 150 μm from foveal center Note: The fellow eye may have any of the following: no AMD, AMD without GA, AMD with GA, CNV AMD, or foveal GA (ongoing treatment with anti-angiogenic therapies and/or complement inhibitor therapies in the fellow eye is allowable)

   Ocular conditions - Study Eye:
2. 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:

   1. be ≥ 0.50 mm2 and ≤ 10.16 mm2 AND
   2. reside completely within the FAF 30- or 35-degree image
3. BCVA by Early Treatment Diabetic Retinopathy Study (ETDRS) score of ≥ 55 letters in the study eye
4. LL BCVA by ETDRS score of ≥ 10 letters in the study eye
5. LLD (defined as the difference between BCVA and LL BCVA) of \> 5 letters in the study eye
6. 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:
7. Able to administer IMP or have an appropriate designee who can administer the IMP (i.e., a capable family member or a caregiver)
8. 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
9. 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:

   1. Abstinence, when it is in line with the preferred and usual lifestyle of the subject; Subject agrees to use a highly effective method of contraception should they become sexually active
   2. Relationships with male partners who have been surgically sterilized by vasectomy (the vasectomy procedure must have been conducted at least 60 days prior to the Screening Visit)
   3. Barrier method (e.g., condom or occlusive cap) with spermicidal foam/gel/film/cream AND either hormonal contraception (oral, implanted, or injectable) or an intrauterine device or system Note: Non-childbearing potential is defined as surgical sterilization (e.g., bilateral oophorectomy, hysterectomy, or tubal ligation) or postmenopausal (defined as permanent cessation of menstruation for at least 12 consecutive months prior to the Screening Visit).
10. 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:

1. The absence of observable hyper-FAF at the margins of the GA in the study eye at the Screening Visit by the Reading Center
2. 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
3. Evidence of exudative AMD or CNV in the study eye by history or FA , as determined by the Reading Center
4. Presence of retinal vein occlusion in the study eye
5. Presence of vitreous hemorrhage in the study eye
6. History of retinal detachment in the study eye
7. History of macular hole (stages 2 to 4) in the study eye
8. Presence of an epiretinal membrane and/or vitreomacular traction in the study eye that causes distortion of the retinal contour
9. 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
10. At the Screening Visit, advanced glaucoma resulting in a cup to disc ratio of \> 0.8 in the study eye
11. 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.
12. 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

    * 1 Opacity is absent
    * 2 Opacity is present, but less than Nuclear Standard Photograph #2
    * 3 Opacity is present, and as severe as or worse than Nuclear Standard Photograph #2 Source: (Chew 2010)
13. 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
14. Ocular incisional or laser surgery (including cataract surgery) in the study eye within 90 days before the Baseline Visit
15. YAG laser capsulotomy in the study eye within 30 days before the Baseline Visit
16. Aphakia in the study eye
17. History of vitrectomy surgery, submacular surgery, or any vitreoretinal surgery in the study eye
18. Prior treatment with Visudyne® (verteporfin) ocular photodynamic therapy, external-beam radiation therapy (for intraocular conditions), or transpupillary thermotherapy in the study eye
19. History of subthreshold laser treatment or other forms of photobiomodulation for AMD in the study eye
20. 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
21. Intravitreal drug delivery of a complement inhibitor in the past 6 months from the Baseline Visit in the study eye
22. Concurrent disease in the study eye that could require medical or surgical intervention during the trial

    Ocular conditions - Either Eye:
23. Presence or a history of diabetic retinopathy in either eye (a history of diabetes mellitus without retinopathy is not a criterion for exclusion)
24. History of herpetic infection in either eye
25. Active uveitis and/or vitritis (grade trace or above) in either eye
26. History of idiopathic or autoimmune-associated uveitis in either eye
27. Active infectious conjunctivitis, keratitis, scleritis, or endophthalmitis in either eye

    Systemic Conditions:
28. 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
29. History of solid organ transplant
30. 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
31. 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)
32. eGFR of \< 30 mL/min at the Screening Visit (using the CKD-EPI 2021 formula)

    General Conditions:
33. 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
34. Women who are pregnant, planning to become pregnant, or breastfeeding/lactating
35. History of allergy to fluorescein that is not amenable to treatment
36. Inability to comply with trial or follow-up procedures
37. Inability to obtain CFP, FAF, and FA of sufficient quality to be analyzed and interpreted
38. 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
39. History of allergic reaction to the investigational drug or any of its components
40. Prior participation in any elamipretide trial

Where this trial is running

Phoenix, Arizona and 62 other locations

+13 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Age Related Macular DegenerationdAMD, Dry AMD, dryAMDMacular degenerationnon-central geographic atrophy, GAMTP-131elamipretideNon- Intravitreal, Non- gene therapy
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.