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

• ClinicalTrials.gov Identifier: NCT02314299
• Recruitment Status: Completed
• First Posted: December 11, 2014
• Last Update Posted: October 26, 2015
• Sponsor: Stealth BioTherapeutics Inc.
• Information provided by (Responsible Party): Stealth BioTherapeutics Inc.

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Diabetic Macular Edema; Age-related Macular Degeneration	Drug: MTP-131	Phase 1; Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 20 participants
• Allocation: Non-Randomized
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase 1/2, Open-Label, Dose-Escalation, Single Center, Clinical Study of MTP-131 (Ocuvia™) Topical Ophthalmic Solution in Subjects With Diabetic Macular Edema and Non-Exudative Intermediate Age-related Macular Degeneration.
• Study Start Date: November 2014
• Actual Primary Completion Date: May 2015
• Actual Study Completion Date: June 2015

Arms and Interventions

Arm	Intervention/treatment
Experimental: Low Dose Regimen; MTP-131 (Low Dose) sterile topical ophthalmic solution twice a day into the study eye	Drug: MTP-131
Experimental: High Dose Regimen; MTP-131 (High Dose) sterile topical ophthalmic solution twice a day into the study eye	Drug: MTP-131

Outcome Measures

Primary Outcome Measures :

1. Incidence and severity of systemic and ocular adverse events [ Time Frame: From Baseline to Day 28 ]
2. Change from Baseline in vital sign measurements [ Time Frame: From Baseline to Day 28 ]
3. Change from Baseline in best corrected visual acuity (BCVA) as measured with the early treatment diabetic retinopathy study (ETDRS) scale [ Time Frame: From Baseline to Day 28 ]
4. Change from Baseline in slit lamp examinations [ Time Frame: From Baseline to Day 28 ]
5. Change from Baseline in intraocular pressure [ Time Frame: From Baseline to Day 28 ]
6. Change from Baseline in dilated fundoscopic examinations [ Time Frame: From Baseline to Day 28 ]
7. Change from Baseline in central subfield thickness [ Time Frame: From Baseline to Day 28 ]

Secondary Outcome Measures :

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

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 80 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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

Contacts and Locations

Locations

United States, Massachusetts

Boston, Massachusetts, United States

Sponsors and Collaborators

Stealth BioTherapeutics Inc.

More Information

• Responsible Party: Stealth BioTherapeutics Inc.
• ClinicalTrials.gov Identifier: NCT02314299
• Other Study ID Numbers: SPIOC-101
• First Posted: December 11, 2014
• Last Update Posted: October 26, 2015
• Last Verified: October 2015

Keywords provided by Stealth BioTherapeutics Inc.:

Macular edema; Macular degeneration; MTP-131; Ocuvia™

Additional relevant MeSH terms:

Macular Degeneration; Macular Edema; Edema; Retinal Degeneration; Retinal Diseases; Eye Diseases