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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06373731




Registration number
NCT06373731
Ethics application status
Date submitted
16/04/2024
Date registered
18/04/2024
Date last updated
26/11/2024

Titles & IDs
Public title
ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD)
Scientific 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)
Secondary ID [1] 0 0
SPIAM-301
Universal Trial Number (UTN)
Trial acronym
ReNEW
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Age Related Macular Degeneration (ARMD) 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Elamipretide
Treatment: Drugs - Placebo

Experimental: Elamipretide - Subjects will receive once daily subcutaneous doses of 40mg elamipretide for 96 weeks

Placebo comparator: Placebo - Subjects will receive once daily subcutaneous doses of placebo for 96 weeks


Treatment: Drugs: Elamipretide
Subjects will receive once daily SC doses of 40 mg elamipretide for 96 weeks

Treatment: Drugs: Placebo
Subjects will receive once daily SC doses of Placebo 96 weeks
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Week 48 Rate of change in the macular area of photoreceptor loss
Timepoint [1] 0 0
Baseline, Week 48
Secondary outcome [1] 0 0
Week 72 Rate of change in the Macular area of photoreceptor loss
Timepoint [1] 0 0
Baseline, Week 72
Secondary outcome [2] 0 0
Week 96 Rate of change in the Macular area of photoreceptor loss
Timepoint [2] 0 0
Baseline, Week 96
Secondary outcome [3] 0 0
Proportion of subjects gaining = 10 letters (2 lines) in Low Luminance Best-Corrected Visual Acuity (LL BCVA)
Timepoint [3] 0 0
Baseline, Week 48
Secondary outcome [4] 0 0
Proportion of subjects gaining = 15 letters in Low Luminance Best-Corrected Visual Acuity (LL BCVA)
Timepoint [4] 0 0
Baseline, Week 48

Eligibility
Key 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
Minimum age
55 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 by history or FA in the study eye, 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 > 2 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 > +2 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 of diabetic retinopathy (a history of diabetes mellitus without retinopathy is not a criterion for exclusion) in either eye
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 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/05/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2027
Actual
Sample size
Target
360
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Connecticut
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Indiana
Country [8] 0 0
United States of America
State/province [8] 0 0
Maryland
Country [9] 0 0
United States of America
State/province [9] 0 0
Massachusetts
Country [10] 0 0
United States of America
State/province [10] 0 0
Michigan
Country [11] 0 0
United States of America
State/province [11] 0 0
Minnesota
Country [12] 0 0
United States of America
State/province [12] 0 0
New Jersey
Country [13] 0 0
United States of America
State/province [13] 0 0
Ohio
Country [14] 0 0
United States of America
State/province [14] 0 0
Oklahoma
Country [15] 0 0
United States of America
State/province [15] 0 0
Oregon
Country [16] 0 0
United States of America
State/province [16] 0 0
Texas
Country [17] 0 0
United States of America
State/province [17] 0 0
Virginia
Country [18] 0 0
United States of America
State/province [18] 0 0
Washington
Country [19] 0 0
Hungary
State/province [19] 0 0
Pécs
Country [20] 0 0
New Zealand
State/province [20] 0 0
Christchurch
Country [21] 0 0
New Zealand
State/province [21] 0 0
Wellington
Country [22] 0 0
United Kingdom
State/province [22] 0 0
Bristol
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Leicester
Country [24] 0 0
United Kingdom
State/province [24] 0 0
London
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Salisbury
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Sunderland

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Stealth BioTherapeutics Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT06373731
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Rekha Sathyanarayana
Address 0 0
Stealth BioTherapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Rekha Sathyanarayana
Address 0 0
Country 0 0
Phone 0 0
617-762-2579
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06373731