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A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide (MTP-131) Topical Ophthalmic Solution for the Treatment of Leber's Hereditary Optic Neuropathy

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ClinicalTrials.gov Identifier: NCT02693119
Recruitment Status : Active, not recruiting
First Posted : February 26, 2016
Last Update Posted : September 11, 2017
Sponsor:
Information provided by (Responsible Party):
Stealth BioTherapeutics Inc.

Brief Summary:
This is a Phase 2, prospective, randomized, double-masked, vehicle controlled, single-center study in approximately 12 subjects with LHON to evaluate safety, tolerability and efficacy of elamipretide (MTP-131) topical ophthalmic solution in this patient population. At the conclusion of 52 weeks of treatment, subjects will be offered the opportunity to enter an Open Label Extension for up to 48 additional weeks of treatment.

Condition or disease Intervention/treatment Phase
Leber's Hereditary Optic Neuropathy Drug: elamipretide (MTP-131) 1% topical ophthalmic solution Drug: Vehicle topical ophthalmic solution Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Prospective, Randomized, Double-Masked, Vehicle Controlled, Phase 2 Clinical Study to Evaluate the Safety, Tolerability and Efficacy of Elamipretide (MTP-131) Topical Ophthalmic Solution in Subjects With Leber's Hereditary Optic Neuropathy (LHON)
Study Start Date : March 2016
Estimated Primary Completion Date : May 2018
Estimated Study Completion Date : May 2018


Arm Intervention/treatment
Experimental: Group 1
One drop elamipretide (MTP-131) 1% topical ophthalmic solution BID applied to the left eye (OS) and one drop of vehicle topical ophthalmic solution
Drug: elamipretide (MTP-131) 1% topical ophthalmic solution
Drug: Vehicle topical ophthalmic solution
Experimental: Group 2
One drop elamipretide (MTP-131) 1% topical ophthalmic solution BID applied to the right eye (OD) and one drop of vehicle topical ophthalmic solution
Drug: elamipretide (MTP-131) 1% topical ophthalmic solution
Drug: Vehicle topical ophthalmic solution
Experimental: Group 3
One drop elamipretide (MTP-131) 1% topical ophthalmic solution BID applied to both eyes (OU)
Drug: elamipretide (MTP-131) 1% topical ophthalmic solution
Experimental: OLE
One drop elamipretide (MTP-131) 1% topical ophthalmic solution BID applied to both eyes (OU)
Drug: elamipretide (MTP-131) 1% topical ophthalmic solution



Primary Outcome Measures :
  1. Incidence and severity of adverse events [ Time Frame: Assessed at each visit from Baseline to Week 56 (follow-up visit) ]

Secondary Outcome Measures :
  1. Change in photopic negative response electroretinography (PhNR-ERG) response pattern [ Time Frame: Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. ]
  2. Change in visual field MD as measured by Humphrey automated visual field testing stimulus III [ Time Frame: Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. ]
  3. Change in best corrected visual acuity (BCVA) using the using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale [ Time Frame: Assessed at each visit from Baseline to Week 56 (follow-up visit). ]
  4. Change in Visual Function Questionnaire (VFQ-39) score [ Time Frame: Assessed at Baseline, Week 36 (end-of-treatment visit) and Week 56 (follow-up visit) ]
  5. Change in mean retinal nerve fiber layer thickness by spectral domain optical coherence tomography (SD-OCT) [ Time Frame: Assessed at Baseline and Week 52 (end-of-treatment visit) ]
  6. Change in mean retinal ganglion cell layer thickness by spectral domain optical coherence tomography (SD-OCT) [ Time Frame: Assessed at Baseline and Week 52 (end-of-treatment visit) ]
  7. Change in color discrimination/contrast sensitivity by Ishihara Color Test [ Time Frame: Assessed at each visit from Baseline to Week 56 (follow up visit) ]


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Ages Eligible for Study:   18 Years to 50 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Adults ≥18 and ≤ 50 years old at the time of loss of vision in the second eye.
  • Able to provide informed consent and willing to comply with all study visits and examinations
  • Diagnosis of LHON based on clinical and ophthalmic functional/anatomic test findings, and satisfactory documentation of the mitochondrial DNA mutation m.11778G>A
  • Loss of vision in both eyes of ≥1 year and ≤10 years at the time of the Screening Visit and current clinically stable visual function (as assessed by the Investigator)
  • Able to self-administer eye drops as demonstrated at screening or having a care provider who can do so
  • Documentation of having satisfactorily completed at least two previous Humphrey automated visual field tests prior to screening.
  • Women of childbearing potential must agree to use birth control as specified in the protocol from the date they sign the informed consent form

Exclusion Criteria:

  • Any other ocular pathology requiring treatment with prescription topical ophthalmic drops (e.g., glaucoma, dry eye)
  • Cup to disc ratio of > 0.8 in either eye
  • Media opacity, suboptimal pupillary dilatation, or refractive error that interferes with adequate retinal imaging
  • Known to be immunocompromised or receiving systemic immunosuppression
  • 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
  • Participation in other investigational drug or device clinical trials within 30 days prior to enrollment, or planning to participate in any other investigational drug or device clinical trials within 30 days of study completion
  • Women who are pregnant or lactating

Other protocol-defined inclusion/exclusion criteria may apply.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02693119


Locations
United States, California
Doheny Eye Center
Pasadena, California, United States
Sponsors and Collaborators
Stealth BioTherapeutics Inc.
Investigators
Principal Investigator: Alfredo Sadun, MD, PhD University of California, Los Angeles

Responsible Party: Stealth BioTherapeutics Inc.
ClinicalTrials.gov Identifier: NCT02693119     History of Changes
Other Study ID Numbers: SPILH-201
First Posted: February 26, 2016    Key Record Dates
Last Update Posted: September 11, 2017
Last Verified: September 2017

Keywords provided by Stealth BioTherapeutics Inc.:
LHON
Ocuvia™
elamipretide
MTP-131

Additional relevant MeSH terms:
Optic Nerve Diseases
Optic Atrophy, Hereditary, Leber
Cranial Nerve Diseases
Nervous System Diseases
Eye Diseases
Optic Atrophies, Hereditary
Optic Atrophy
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Pharmaceutical Solutions
Ophthalmic Solutions