ACTIVE_NOT_RECRUITING

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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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)

Quick Facts

Study Start:2024-05-30

Study Completion:2027-09

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT06373731

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:55 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
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) 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 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	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 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 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) 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

Inclusion Criteria

Exclusion Criteria

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)
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
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

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
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
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)
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

Contacts and Locations

Principal Investigator

Rekha Sathyanarayana

STUDY_DIRECTOR

Stealth BioTherapeutics

Study Locations (Sites)

Associated Retina Consultants

Phoenix, Arizona, 85020

United States

Barnet Dulaney Perkins Eye Center

Sun City, Arizona, 85351

United States

Retina Associates of Southern California

Huntington Beach, California, 92607

United States

Retina Consultants of San Diego

Poway, California, 92064

United States

Retinal Consultants Medical Group

Sacramento, California, 95825

United States

Orange County Retinal Medical Group

Santa Ana, California, 92705

United States

Bay Area Retina Associates

Walnut Creek, California, 94598

United States

Retina Consultants of Southern Colorado

Colorado Springs, Colorado, 80909

United States

Connecticut Eye Consultants, P.C.

Danbury, Connecticut, 06810

United States

Vitreo Retinal Associates

Gainesville, Florida, 32607

United States

Florida Retina Institute

Orlando, Florida, 32806

United States

Retina Vitreous Associates of Florida

St. Petersburg, Florida, 33711

United States

University Retina and Macula Associates

Oak Forest, Illinois, 60452

United States

Associated Vitreoretinal and Uveitis Consultants

Carmel, Indiana, 46290

United States

Mid Atlantic Retina Specialist

Hagerstown, Maryland, 21740

United States

Ophthalmic Consultants of Boston

Boston, Massachusetts, 02114

United States

Kellogg Eye Center

Ann Arbor, Michigan, 48105

United States

Retina Consultants of Minnesota

Minneapolis, Minnesota, 55435

United States

Mid Atlantic Retina

Cherry Hill, New Jersey, 08034

United States

NJ Retina

Teaneck, New Jersey, 07666

United States

Retina Vitreous Center

Edmond, Oklahoma, 73013

United States

Retina Northwest, PC

Portland, Oregon, 97221

United States

Retina Research Institute of Texas

Abilene, Texas, 79606

United States

Austin Clinical Research, LLC

Austin, Texas, 78750

United States

Retina Consultants of Texas

Bellaire, Texas, 77401

United States

Valley Retina Institute

McAllen, Texas, 78503

United States

Texas Retina Associates of Plano

Plano, Texas, 75075

United States

Medical Center Ophthalmology Associates

San Antonio, Texas, 78240

United States

Retina Consultants of Texas

The Woodlands, Texas, 77384

United States

Emerson Clinical Research Institute

Falls Church, Virginia, 22042

United States

Pacific Northwest Retina, PLLC

Silverdale, Washington, 98383

United States

Collaborators and Investigators

Sponsor: Stealth BioTherapeutics Inc.

Rekha Sathyanarayana, STUDY_DIRECTOR, Stealth BioTherapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-05-30

Study Completion Date2027-09

Study Record Updates

Study Start Date2024-05-30

Study Completion Date2027-09

Terms related to this study

Keywords Provided by Researchers

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

Additional Relevant MeSH Terms

Age Related Macular Degeneration (ARMD)