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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02848313
Recruitment Status : Unknown
Verified September 2017 by Stealth BioTherapeutics Inc..
Recruitment status was:  Active, not recruiting
First Posted : July 28, 2016
Last Update Posted : September 6, 2017
Sponsor:
Information provided by (Responsible Party):
Stealth BioTherapeutics Inc.

Tracking Information
First Submitted Date  ICMJE July 25, 2016
First Posted Date  ICMJE July 28, 2016
Last Update Posted Date September 6, 2017
Actual Study Start Date  ICMJE November 3, 2016
Estimated Primary Completion Date March 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 7, 2017)
The incidence and severity of Adverse Events [ Time Frame: Baseline through Week 28 ]
Original Primary Outcome Measures  ICMJE
 (submitted: July 25, 2016)
  • The incidence and severity of Adverse Events [ Time Frame: Baseline through Week 16 ]
  • Compliance of self-administration of subcutaneous elamipretide [ Time Frame: Baseline through Week 12 ]
Change History Complete list of historical versions of study NCT02848313 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 7, 2017)
  • Change in fundus hyperautofluorescence [ Time Frame: Assessed at screening, baseline, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28 ]
  • Change from Baseline in best-corrected low-luminance (LL) visual acuity [ Time Frame: Assessed at screening, baseline, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28 ]
  • Change from Baseline in mesopic light sensitivity by microperimetry [ Time Frame: Assessed at screening, baseline, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28 ]
  • Change in dark adaptometry [ Time Frame: Assessed at screening, baseline, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28 ]
  • Change in drusen volume ("retinal pigment epithelium - drusen complex", as measured by automated segmentation) by SD-OCT [ Time Frame: Assessed at screening, baseline, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28 ]
  • Change in fundus photography [ Time Frame: Assessed at screening and Week 24 ]
  • Change in best-corrected visual acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study scale [ Time Frame: Assessed at screening, baseline, Day 7, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28 ]
  • Change in speed reading at standard light [ Time Frame: Assessed at baseline, Week 4, Week 8, Week 12, and Week 16 ]
  • Change is speed reading at low light [ Time Frame: Assessed at screening, baseline, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28 ]
  • Change in LL visual function by the Low-Luminance Questionnaire [ Time Frame: Assessed at screening, baseline, Week 12, Week 24, and Week 28 ]
  • Change in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) score [ Time Frame: Assessed at baseline, Week 12, Week 24, and Week 28 ]
  • Compliance of administration of subcutaneous elamipretide [ Time Frame: Baseline through Week 24 ]
  • Number of home health visits necessary for subject or caregiver to learn how to inject elamipretide [ Time Frame: Baseline through Week 24 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: July 25, 2016)
  • Change in fundus hyperautofluorescence for both noncentral geographic atrophy (GA) and High Risk Drusen Without GA [ Time Frame: Assessed at screening, baseline, Day 7, Week 4, Week 8, Week 12, and Week 16 ]
  • Change from Baseline in best-corrected low-luminance (LL) visual acuity for both noncentral GA and High Risk Drusen Without GA [ Time Frame: Assessed at screening, baseline, Day 7, Week 4, Week 8, Week 12, and Week 16 ]
  • Change from Baseline in mesopic light sensitivity by microperimetry for both noncentral GA and High Risk Drusen Without GA [ Time Frame: Assessed at screening, baseline, Day 7, Week 4, Week 8, Week 12, and Week 16 ]
  • Change in dark adaptometry for both noncentral GA and High Risk Drusen Without GA [ Time Frame: Assessed at screening, baseline, Day 7, Week 4, Week 8, Week 12, and Week 16 ]
  • Change in drusen volume ("retinal pigment epithelium - drusen complex", as measured by automated segmentation) by SD-OCT for both noncentral GA and High Risk Drusen Without GA [ Time Frame: Assessed at screening, baseline, Day 7, Week 4, Week 8, Week 12, and Week 16 ]
  • Change in fundus photography for both noncentral GA and High Risk Drusen Without GA [ Time Frame: Assessed at screening, Week 4, Week 12 ]
  • Change in best-corrected visual acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study scale for both noncentral GA and High Risk Drusen Without GA [ Time Frame: Assessed at screening, baseline, Day 7, Week 4, Week 8, Week 12, and Week 16 ]
  • Change is speed reading for both noncentral GA and High Risk Drusen Without GA [ Time Frame: Assessed at screening, baseline, Week 4, Week 8, Week 12, and Week 16 ]
  • Change in LL visual function by the Low-Luminance Questionnaire for both noncentral GA and High Risk Drusen Without GA [ Time Frame: Assessed at screening, Week 4, Week 8, Week 12, and Week 16 ]
  • Change in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) score for both noncentral GA and High Risk Drusen Without GA [ Time Frame: Assessed at screening, Week 4, Week 8, Week 12, and Week 16 ]
  • Change in drusen volume ("retinal pigment epithelium - drusen complex", as measured by automated segmentation) by SD-OCT for High Risk Drusen Without GA [ Time Frame: Assessed at screening, baseline, Day 7, Week 4, Week 8, Week 12, and Week 16 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Intermediate Age-Related Macular Degeneration
Official Title  ICMJE An Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Intermediate Age-Related Macular Degeneration
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Age-Related Macular Degeneration
Intervention  ICMJE Drug: Elamipretide
40 mg dose of elamipretide administered once daily as a 1.0mL SC injection.
Other Name: MTP-131; Bendavia
Study Arms  ICMJE Experimental: Elamipretide
40 mg dose of elamipretide administered once daily as a 1.0mL SC injection
Intervention: Drug: Elamipretide
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Actual Enrollment  ICMJE
 (submitted: July 25, 2016)
40
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2018
Estimated Primary Completion Date March 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

For this study, only 1 eye of an eligible subject will be included and designated as the study eye. However, all specified ophthalmic testing will be performed on both eyes at each time point. A potential subject must meet the following criteria to be eligible for inclusion in the study:

Intermediate AMD - noncentral GA disease group:

  1. Adults ≥ 55 years of age with 1 eye with intermediate AMD - noncentral GA.
  2. No evidence of choroidal neovascularization (active or prior history) in the study eye.
  3. Geographic atrophy may be multifocal, but the cumulative GA lesion size must be:

    1. ≥ 1.27 mm2 (approximately ≥ 0.5 DA) and ≤ 10.16 mm2 (approximately ≤ 4 DA).
    2. Must reside completely within the FAF imaging field (field 2 to 30-degree image centered on the fovea).
  4. Presence of measurable hyperautofluorescence adjacent to the discrete foci of GA.

    OR

    Intermediate AMD - high-risk drusen without GA disease group:

  5. ≥ 55 years of age with one eye with intermediate AMD - high-risk drusen without GA.
  6. 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):

  7. Able to provide informed consent and willing to comply with all study visits and examinations.
  8. Women of childbearing potential who are not pregnant or nursing and have a negative serum pregnancy test at screening.
  9. Best-corrected visual acuity assessed by ETDRS letters ≥ 55 letters (Snellen equivalent ≥ 20/70).
  10. Low-luminance visual acuity deficit (defined as difference between BCVA and LL visual acuity) > 5 letters.
  11. Has at least two Low-Luminance Questionnaire sub scale results, in which one of the abnormal subscales is either general dim light vision or dim light reading.
  12. The fellow eye may have intermediate AMD without noncentral GA (i.e., high-risk drusen), intermediate AMD with noncentral GA, NV AMD, or central GA. Ongoing treatment with antiangiogenic therapies in the fellow eye is allowable.
  13. No evidence of visually significant cataract OR pseudophakia without evidence of posterior capsular opacity.
  14. Sufficiently clear ocular media, adequate pupillary dilation, fixation to permit quality fundus imaging, and able to cooperate sufficiently for adequate ophthalmic visual function testing and anatomic assessment.
  15. Able to administer SC study drug solution as demonstrated at screening or able to have a care provider or appropriate designee who can administer the study drug (i.e., a capable family member or home health nursing aide).
  16. If of childbearing potential or in a relationship with a partner of childbearing potential, are able to abstain from sex or use acceptable contraception during the study and for 3 months after dosing.

    1. For men: Abstinence is only acceptable if it is in line with the preferred and usual lifestyle of the subject. The subject also agrees to use an acceptable method of contraception should they become sexually active. Subjects must use a condom with spermicide from the date of informed consent until at least 3 months after the last dose of study drug. Periodic abstinence (e.g.calendar, ovulation, symptothermal, or postovulation methods) and withdrawal are not acceptable methods of contraception.
    2. For women: abstinence is only acceptable when it is in line with the preferred and usual lifestyle of the subject. The subject agrees to use an acceptable method of contraception should they become sexually active. Maintenance of a monogamous relationship with a male partner who has been surgically sterilized by vasectomy (the vasectomy procedure must have been conducted at least 60 days before the Screening visit or confirmed via sperm analysis), 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 are acceptable methods.
  17. Ability and willingness to undertake all scheduled visits and assessments.

Exclusion Criteria:

A subject with study eye who meets any of the following criteria will be excluded from the study:

Ocular conditions - study eye

  1. Age-related macular degeneration with any evidence of central GA (i.e., involving the fovea).
  2. Atrophic retinal disease because of causes other than AMD.
  3. Presence or diagnosis of exudative AMD or choroidal neovascularization in the study eye.
  4. History of diabetic retinopathy (a history of diabetes mellitus without retinopathy is not a criterion for exclusion).
  5. Presence of vitreous hemorrhage.
  6. History of retinal detachment or macular hole (stage 3 or 4) in the study eye.
  7. Presence of macular pucker.
  8. History of uncontrolled glaucoma, defined as advanced cup-to-disc ratio > 0.7 and IOP > 25, with or without topical antihypertensive eye drops; treatment of ocular hypertension or controlled glaucoma are not criteria for exclusion.
  9. History of advanced guttae indicative of Fuchs endothelial dystrophy.
  10. Presence of visually significant cataract OR presence of significant posterior capsular opacity in the setting of Pseudophakia.
  11. Presence of significant keratopathy that would cause scattering of light or alter visual function, especially in LL conditions.
  12. Ocular incisional surgery (including cataract surgery) in the study eye within 3 months (i.e. 90 days) before Day 1.
  13. Aphakia.
  14. History of vitrectomy surgery, submacular surgery, or any vitreoretinal surgery.
  15. Prior treatment with Visudyne ® (verteporfin), external-beam radiation therapy (for intraocular conditions), or transpupillary thermotherapy.
  16. History of prophylactic subthreshold laser treatment for retinal disease.
  17. Previous intravitreal drug delivery (e.g., intravitreal corticosteroid injection, anti-angiogenic drugs, or device implantation) in the study eye.

    Ocular conditions - either eye

  18. Active uveitis and/or vitritis (grade trace or above) in either eye.
  19. History of idiopathic or autoimmune-associated uveitis in either eye.
  20. Active, infectious conjunctivitis, keratitis, scleritis, or endophthalmitis in either eye.

    Systemic conditions

  21. Known to be immunocompromised or receiving systemic immunosuppression.
  22. Any disease or medical condition that in the opinion of the Investigator would prevent the subject from participating in the study or might confound study results.
  23. Estimated glomerular filtration rate < 30 mL/minute, by MDRD.
  24. Presence or history of clinically significant allergy disease requiring treatment, as judged by the Investigator. Hay fever is allowed unless it is active.

    General

  25. Participation in other investigational drug or device clinical trials within 30 days before enrollment, or planning to participate in any other investigational drug or device clinical trials within 30 days of study completion.
  26. History of allergy to fluorescein that is not amenable to treatment.
  27. Inability to comply with study or follow-up procedures.
  28. Inability to obtain color fundus photograph, FAF, and fluorescein angiography of sufficient quality to be analyzed and interpreted.
  29. History of allergic reaction to the investigational drug or any of its components.
  30. Current use of or likely need for any excluded medication.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 55 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02848313
Other Study ID Numbers  ICMJE SPIAM-101
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Stealth BioTherapeutics Inc.
Study Sponsor  ICMJE Stealth BioTherapeutics Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Scott W Cousins, MD Duke University School of Medicine / Dept. of Ophthalmology (Duke Eye Center)
PRS Account Stealth BioTherapeutics Inc.
Verification Date September 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP