A Study of MTP-131 Topical Ophthalmic Solution in Subjects With Diabetic Macular Edema and Non-Exudative Intermediate Age-related Macular Degeneration (SPIOC-101)

Stealth BioTherapeutics

Status and phase: Completed; Phase 2; Phase 1

Conditions: Diabetic Macular Edema; Age-related Macular Degeneration

Treatments: Drug: MTP-131

Study type: Interventional

Funder types: Industry

Identifiers

NCT02314299

SPIOC-101

Details and patient eligibility

About

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.

Enrollment: 20 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

20 participants in 2 patient groups

Low Dose Regimen

Experimental group

Description:

MTP-131 (Low Dose) sterile topical ophthalmic solution twice a day into the study eye

Treatment:

Drug: MTP-131

High Dose Regimen

Experimental group

Description:

MTP-131 (High Dose) sterile topical ophthalmic solution twice a day into the study eye

Treatment:

Drug: MTP-131