ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD)

NCT06373731 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: SPIAM-301
• Study Type: interventional
• Target: 313
• Locations: 8 countries
• Sites: 53

Brief Summary

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.

Conditions

Age Related Macular Degeneration (ARMD)

Keywords

dAMD, Dry AMD, dryAMD; Macular degeneration; non-central geographic atrophy, GA; MTP-131; elamipretide; Non- Intravitreal, Non- gene therapy

Outcome Measures

Primary Outcomes (1)

• Week 48 Rate of change in the macular area of photoreceptor loss

  Rate of change in the macular area of photoreceptor loss (defined as an EZ-RPE thickness of 0μm) assessed by SD-OCT and EZ mapping at Week 48

  Baseline, Week 48

Secondary Outcomes (4)

• Week 72 Rate of change in the Macular area of photoreceptor loss

  Baseline, Week 72

• Week 96 Rate of change in the Macular area of photoreceptor loss

  Baseline, Week 96

• Proportion of subjects gaining ≥ 10 letters (2 lines) in Low Luminance Best-Corrected Visual Acuity (LL BCVA)

  Baseline, Week 48

• Proportion of subjects gaining ≥ 15 letters in Low Luminance Best-Corrected Visual Acuity (LL BCVA)

  Baseline, Week 48

Study Arms (2)

Elamipretide

EXPERIMENTAL

Subjects will receive once daily subcutaneous doses of 40mg elamipretide for 96 weeks

Drug: Elamipretide

Placebo

PLACEBO COMPARATOR

Subjects will receive once daily subcutaneous doses of placebo for 96 weeks

Drug: Placebo

Interventions

Elamipretide DRUG

Subjects will receive once daily SC doses of 40 mg elamipretide for 96 weeks

Also known as: Elamipretide HCL

Elamipretide

Placebo DRUG

Subjects will receive once daily SC doses of Placebo 96 weeks

Placebo

Eligibility Criteria

• Age: 55 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• well-demarcated area(s) of GA
• 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:
• 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:
• be ≥ 0.50 mm2 and ≤ 10.16 mm2 AND
• reside completely within the FAF 30- or 35-degree image
• 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:

You may not qualify if:

• 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
• Opacity is absent

Sponsors & Collaborators

• Stealth BioTherapeutics Inc.: lead

Study Sites (53)

Associated Retina Consultants

Phoenix, Arizona, 85020, United States

Location

Barnet Dulaney Perkins Eye Center

Sun City, Arizona, 85351, United States

Location

Retina Associates of Southern California

Huntington Beach, California, 92607, United States

Location

Retina Consultants of San Diego

Poway, California, 92064, United States

Location

Retinal Consultants Medical Group

Sacramento, California, 95825, United States

Location

Orange County Retinal Medical Group

Santa Ana, California, 92705, United States

Location

Bay Area Retina Associates

Walnut Creek, California, 94598, United States

Location

Retina Consultants of Southern Colorado

Colorado Springs, Colorado, 80909, United States

Location

Connecticut Eye Consultants, P.C.

Danbury, Connecticut, 06810, United States

Location

Vitreo Retinal Associates

Gainesville, Florida, 32607, United States

Location

Florida Retina Institute

Orlando, Florida, 32806, United States

Location

Retina Vitreous Associates of Florida

St. Petersburg, Florida, 33711, United States

Location

University Retina and Macula Associates

Oak Forest, Illinois, 60452, United States

Location

Associated Vitreoretinal and Uveitis Consultants

Carmel, Indiana, 46290, United States

Location

Mid Atlantic Retina Specialist

Hagerstown, Maryland, 21740, United States

Location

Ophthalmic Consultants of Boston

Boston, Massachusetts, 02114, United States

Location

Kellogg Eye Center

Ann Arbor, Michigan, 48105, United States

Location

Retina Consultants of Minnesota

Minneapolis, Minnesota, 55435, United States

Location

Mid Atlantic Retina

Cherry Hill, New Jersey, 08034, United States

Location

NJ Retina

Teaneck, New Jersey, 07666, United States

Location

Retina Vitreous Center

Edmond, Oklahoma, 73013, United States

Location

Retina Northwest, PC

Portland, Oregon, 97221, United States

Location

Retina Research Institute of Texas

Abilene, Texas, 79606, United States

Location

Austin Clinical Research, LLC

Austin, Texas, 78750, United States

Location

Retina Consultants of Texas

Bellaire, Texas, 77401, United States

Location

Valley Retina Institute

McAllen, Texas, 78503, United States

Location

Texas Retina Associates of Plano

Plano, Texas, 75075, United States

Location

Medical Center Ophthalmology Associates

San Antonio, Texas, 78240, United States

Location

Retina Consultants of Texas

The Woodlands, Texas, 77384, United States

Location

Emerson Clinical Research Institute

Falls Church, Virginia, 22042, United States

Location

Pacific Northwest Retina, PLLC

Silverdale, Washington, 98383, United States

Location

Axon Clinical, s.r.o.

Prague, Czechia

Location

Augenzentrum am St. Franziskus-Hospital

Münster, Germany

Location

Klinik und Poliklinik für Augenheilkunde- Universitätsklinik Regensburg

Regensburg, Germany

Location

Department für Augenheilkunde

Tübingen, Germany

Location

University Of Debrecen Eye Center

Debrecen, Hungary

Location

Ganglion Medical Center

Pécs, Hungary

Location

University of Szeged, Department of Ophthalmology

Szeged, Hungary

Location

Hospital Luigi Sacco Ophthalmology Dept

Milan, Italy

Location

IRRCS Ospendale San Raffaele

Milan, Italy

Location

Policlinico Milano

Milan, Italy

Location

Fondazione Policlinico Gemelli

Roma, Italy

Location

Department of Ophthalmology, Azienda SanitariaUniversitaria Friuli Centrale

Udine, Italy

Location

Southern Eye Specialists

Christchurch, 8013, New Zealand

Location

Capital Eye Specialists

Wellington, 6011, New Zealand

Location

Centro de Oftalmologia Barraquer

Barcelona, Spain

Location

OMIQ Research

Barcelona, Spain

Location

Fundacion Aiken de la Comunitat Valenciana

Valencia, Spain

Location

Oftalvist

Valencia, Spain

Location

University Hospitals Bristol NHS Foundation Trust - Bristol Eye Hospital

Bristol, United Kingdom

Location

University Hospitals of Leicester, Leicester Royal Infirmary

Leicester, United Kingdom

Location

Macular Services, Central Middlesex Hospital, NHS Foundation Trust

London, United Kingdom

Location

South Tyneside and Sunderland NHS Foundation Trust - Sunderland Eye Infirmary

Sunderland, United Kingdom

Location

MeSH Terms

Conditions

Macular Degeneration

Interventions

arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Officials

• Rekha Sathyanarayana

  Stealth BioTherapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Trial personnel and subjects will be masked to treatment until the database is locked at the end of the trial, unless noted below. The Investigator will contact the Sponsor Medical Monitor prior to unmasking any subject's treatment sequence unless in the instance of a medical emergency. In case of an immediate medical emergency, or if directed by the Sponsor, and only if the information is required by the Investigator to manage a subject's AE, a subject's treatment assignment may be unmasked prematurely using the computerized system. The Sponsor must be notified as soon as possible regarding the reason for unmasking.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Subjects will be randomized (2:1) to once daily 40 mg SC of elamipretide or placebo for 96 weeks of treatment by a central randomization

Study Record Dates

• First Submitted: April 16, 2024
• First Posted: April 18, 2024
• Study Start: May 30, 2024
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): September 1, 2027
• Last Updated: November 17, 2025

Record last verified: 2025-11

Locations

HU (3); IT (5); GB (4); CZ (1); ES (4); US (31); NZ (2); DE (3)