A Study of MTP-131 Topical Ophthalmic Solution in Subjects With Diabetic Macular Edema and Non-Exudative Intermediate Age-related Macular Degeneration

Phase 1

Completed

• Conditions: Age-related Macular Degeneration; Diabetic Macular Edema
• Interventions: Drug: MTP-131

• Registration Number: NCT02314299
• Lead Sponsor: Stealth BioTherapeutics Inc.

Brief Summary

This is a Phase 1/2, open-label, dose-escalation clinical study, enrolling 21 subjects in one of the two treatment groups to determine the effects of topical ocular administration of low dose or high dose MTP-131 given twice a day in subjects with Diabetic Macular Edema and Age-Related Macular Degeneration.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-11
• Study First Submitted: 2014-12-02
• Study First Submitted QC: 2014-12-10
• Study First Posted: 2014-12-11
• Primary Completion: 2015-05
• Study Completion: 2015-06
• Status Verified: 2015-10
• Disposition First Submitted: 2015-10-02
• Disposition First Submitted QC: 2015-10-02
• Last Update Submitted: 2015-10-02
• Disposition First Posted: 2015-10-26
• Last Update Posted: 2015-10-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

• General:

  1. Adults aged ≥18 and ≤ 80 years
  2. Women of childbearing potential must have a negative urine pregnancy test at Baseline and agree to use a highly effective method of contraception throughout the study and 60 days after the last dose of study drug.

• DME Treatment Group:

  1. Central subfield thickness (CST) ≥325 microns as measured by spectral domain optical coherence tomography (SD-OCT) at Screening and Baseline Visits
  2. BCVA (ETDRS) no better than 20/25 in the study eye
  3. BCVA (ETDRS) no worse than 20/640 in either eye

• AMD Treatment Group:

  1. Non-exudative, intermediate age-related macular degeneration defined as an eye(s) having extensive intermediate drusen (10 or more drusen 63μm - 124μm) or large drusen (1 or more ≥125 μm) with no evidence of advanced AMD (i.e., no choroidal neovascularization or macular atrophy)
  2. BCVA (ETDRS) no worse than 20/400 in either eye

Exclusion Criteria

• General:

  1. Inability to self-administer eye drops
  2. Current use of or likely need for systemic medications known to be toxic to the lens, retina or optic nerve (for example deferoxamine, chloroquine/hydroxychloroquine [Plaquenil], tamoxifen, phenothiazines and ethambutol)
  3. Any medical condition that, in the opinion of the investigator, would preclude safe participation in the study or completion of all study requirements and examinations
  4. Have a glycosylated hemoglobin (HbA1c) ≥ 12%

• Ocular Conditions:

  1. Any ocular condition in the study eye that in the opinion of the investigator would prevent improvement in visual acuity or successful completion of any study examination

  2. Ocular hypertension or glaucoma, dry eye, and any other ocular pathology in the study eye requiring treatment with topical ophthalmic drops

     • Other protocol-defined inclusion/exclusion criteria may apply

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
High Dose Regimen	MTP-131	MTP-131 (High Dose) sterile topical ophthalmic solution twice a day into the study eye
Low Dose Regimen	MTP-131	MTP-131 (Low Dose) sterile topical ophthalmic solution twice a day into the study eye

Primary Outcome Measures

Name	Time	Method
Incidence and severity of systemic and ocular adverse events	From Baseline to Day 28
Change from Baseline in vital sign measurements	From Baseline to Day 28
Change from Baseline in best corrected visual acuity (BCVA) as measured with the early treatment diabetic retinopathy study (ETDRS) scale	From Baseline to Day 28
Change from Baseline in slit lamp examinations	From Baseline to Day 28
Change from Baseline in intraocular pressure	From Baseline to Day 28
Change from Baseline in dilated fundoscopic examinations	From Baseline to Day 28
Change from Baseline in central subfield thickness	From Baseline to Day 28

Secondary Outcome Measures

Name	Time	Method
Change in central subfield thickness	From Baseline to Day 28	Diabetic Macular Edema (DME) subjects only
Change from Baseline in widefield angiography	From Baseline to Day 28	Diabetic Macular Edema (DME) subjects only
Change from Baseline in fundus autofluorescence pattern including the occurrence of and aggregate size of areas of macular atrophy	From Baseline to Day 28	Age-Related Macular Degeneration (AMD) subjects only
Change from Baseline in best corrected visual acuity (BCVA) as measured with the early treatment diabetic retinopathy study (ETDRS) scale	From baseline to Day 28	All subjects