An Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Intermediate Age-Related Macular Degeneration

NCT02848313 | Completed Phase 1 Results

• 1 other identifier: SPIAM-101
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

This is an open-label, Phase 1 single-center study in approximately 40 subjects who have 1 eye with intermediate AMD, including a high-risk drusen without geographic atrophy (GA) subgroup and a noncentral GA subgroup. Eligible subjects will receive 40 mg of elamipretide administered as a once daily 1.0 mL subcutaneous injection for 12 weeks.

Conditions

Age-Related Macular Degeneration

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA)

  Change from Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) at 4 meters, letters from Baseline to Day 7, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28.

  Day 0 (Baseline to Day 7, and to Weeks 4, 8, 12, 16, 20, 24, and 28.

Secondary Outcomes (13)

• Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA)

  Baseline to Day 7, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28.

• Change From Baseline in Mean Dark Adaptometry

  Baseline (Day 0) and Week 24

• Mean Treatment Compliance (%) of Administration of Subcutaneous Elamipretide

  Baseline through Week 28

• Mean Number of Home Health Visits to Administer Elamipretide

  Baseline (Day 0) through Week 24

• Change From Baseline in Mean Area of Geographic Atrophy by Fundus Autofluorescence

  Baseline (Day 0) to Week 24

Study Arms (2)

Intermediate AMD - HRD without GA

EXPERIMENTAL

Participants had one 1 eye with intermediate age-related macular degeneration with high-risk drusen without geographic atrophy \[GA\]), i.e. the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen. Participants received 40 mg dose of elamipretide administered once daily as a 1.0mL SC injection.

Drug: Elamipretide

Intermediate AMD with NCGA

EXPERIMENTAL

Participants had 1 eye with intermediate AMD with noncentral geographic atrophy \[NCGA\]; i.e. evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area\[DA\]) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium (RPE) and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Participants in this arm also received 40 mg dose of elamipretide administered once daily as a 1.0mL SC injection.

Drug: Elamipretide

Interventions

Elamipretide DRUG

40 mg dose of elamipretide administered once daily as a 1.0mL SC injection.

Also known as: MTP-131; Bendavia

Intermediate AMD - HRD without GA; Intermediate AMD with NCGA

Eligibility Criteria

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

You may qualify if:

• Intermediate AMD - noncentral GA disease group:
• Adults ≥ 55 years of age with 1 eye with intermediate AMD - noncentral GA.
• No evidence of choroidal neovascularization (active or prior history) in the study eye.
• Geographic atrophy may be multifocal, but the cumulative GA lesion size must be:
• ≥ 1.27 mm2 (approximately ≥ 0.5 DA) and ≤ 10.16 mm2 (approximately ≤ 4 DA).
• Must reside completely within the FAF imaging field (field 2 to 30-degree image centered on the fovea).
• Presence of measurable hyperautofluorescence adjacent to the discrete foci of GA.
• Intermediate AMD - high-risk drusen without GA disease group:
• ≥ 55 years of age with one eye with intermediate AMD - high-risk drusen without GA.
• High-risk drusen is defined as presence of either at least 1 large (≥ 125 µm) druse or multiple medium-size (between 63 and 124 µm) drusen.
• General (both disease groups):
• Able to provide informed consent and willing to comply with all study visits and examinations.
• Women of childbearing potential who are not pregnant or nursing and have a negative serum pregnancy test at screening.
• Best-corrected visual acuity assessed by ETDRS letters ≥ 55 letters (Snellen equivalent ≥ 20/70).
• Low-luminance visual acuity deficit (defined as difference between BCVA and LL visual acuity) \> 5 letters.

You may not qualify if:

• A subject with study eye who meets any of the following criteria will be excluded from the study:
• Ocular conditions - study eye
• Age-related macular degeneration with any evidence of central GA (i.e., involving the fovea).
• Atrophic retinal disease because of causes other than AMD.
• Presence or diagnosis of exudative AMD or choroidal neovascularization in the study eye.
• Presence of vitreous hemorrhage.
• History of retinal detachment or macular hole (stage 3 or 4) in the study eye.
• Presence of macular pucker.
• History of advanced guttae indicative of Fuchs endothelial dystrophy.
• Presence of visually significant cataract OR presence of significant posterior capsular opacity in the setting of Pseudophakia.
• Presence of significant keratopathy that would cause scattering of light or alter visual function, especially in LL conditions.
• Ocular incisional surgery (including cataract surgery) in the study eye within 3 months (i.e. 90 days) before Day 1.
• Aphakia.
• History of vitrectomy surgery, submacular surgery, or any vitreoretinal surgery.
• Prior treatment with Visudyne ® (verteporfin), external-beam radiation therapy (for intraocular conditions), or transpupillary thermotherapy.

Sponsors & Collaborators

• Stealth BioTherapeutics Inc.: lead

Study Sites (1)

Duke University School of Medicine / Dept. of Ophthalmology (Duke Eye Center)

Durham, North Carolina, 27710, United States

Location

MeSH Terms

Conditions

Macular Degeneration

Interventions

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

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

Results Point of Contact

• Title: Jim Carr, Pharm.D. Chief Clinical Development Officer
• Organization: Stealth BioTherapeutics, Inc

jim.carr@stealthbt.com

1-617-600-6888

Study Officials

• Scott W Cousins, MD

  Duke University School of Medicine / Dept. of Ophthalmology (Duke Eye Center)

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Both arms received 40 mg dose of elamipretide administered once daily as a 1.0mL SC injection.

Study Record Dates

• First Submitted: July 25, 2016
• First Posted: July 28, 2016
• Study Start: October 28, 2016
• Primary Completion: March 16, 2018
• Study Completion: April 10, 2018
• Last Updated: October 20, 2020
• Results First Posted: September 21, 2020

Record last verified: 2020-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)