A Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2 - Protocol A

A Phase III Multicenter Randomized, Sham Controlled, Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2

This study is a phase 3, randomized, multi-center study to evaluate the efficacy and safety of the NT-501 implants in participants with macular telangiectasia type 2.

Study Overview

• Status: Completed
• Conditions: Macular Telangiectasia Type 2
• Intervention / Treatment: Combination product: NT-501; Procedure: Sham

• Study Type: Interventional
• Enrollment (Actual): 115
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • Western Australia
    • Perth, Western Australia, Australia, 6009
      • Lions Eye Institute
• France
  • Paris, France, 75010
    • Cochin Hospital
• United Kingdom
  • London, United Kingdom, EC1V 2PD
    • Moorfields Eye Hospital
  • Oxfordshire
    • Oxford, Oxfordshire, United Kingdom, OX3 9DU
      • Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust
• United States
  • California
    • La Jolla, California, United States, 92037
      • Scripps Clinic Carmel Valley Ambulatory Surgery Center
    • Los Angeles, California, United States, 90041
      • Global Research Foundation
    • Los Angeles, California, United States, 90095
      • Jules Stein Eye Institute / David Geffen School of Medicine
    • Palo Alto, California, United States, 94303
      • Byers Eye Institute at Stanford
    • Walnut Creek, California, United States, 94598
      • Bay Area Retina Associates
  • Colorado
    • Colorado Springs, Colorado, United States, 80909
      • Retina Consultants of Southern Colorado, P.C.
  • Florida
    • Winter Haven, Florida, United States, 33880
      • Center for Retina and Macular Disease
  • Louisiana
    • Metairie, Louisiana, United States, 70006
      • Retina Associates of New Orleans
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • National Institute Of Health
    • Hagerstown, Maryland, United States, 21740
      • Cumberland Valley Retina Consultants
  • Michigan
    • Royal Oak, Michigan, United States, 48073
      • Associated Retinal Consultants, P.C.
  • Nevada
    • Reno, Nevada, United States, 89502
      • Sierra Eye Associates
  • Ohio
    • Cleveland, Ohio, United States, 44122
      • Retina Associates of Cleveland, Inc.
  • Oklahoma
    • Tulsa, Oklahoma, United States, 74114
      • Tulsa Retina Consultants
  • Tennessee
    • Germantown, Tennessee, United States, 38138
      • Charles Retina Institute
    • Knoxville, Tennessee, United States, 37909
      • Southeastern Retina Associates, PC
  • Texas
    • Dallas, Texas, United States, 75231
      • Texas Retina Associates
  • Utah
    • Salt Lake City, Utah, United States, 84132
      • University of Utah John A. Moran Eye Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 21 years to 80 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Key Inclusion Criteria:

• Participant must have at least one study eye with a positive diagnosis of MacTel Type 2
• Participant must have an Inner Segment - Outer Segment Junction Line (IS/OS) Photo Receptor (PR) break in the study eye(s) and en face EZ (area of IS/OS loss) as measured by spectral-domain optical coherence tomography (SDOCT) between 0.16 mm^2 and 2.00 mm^2
• Participant's best corrected visual acuity is 54 letter score or better (20/80 or better) as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart

Key Exclusion Criteria:

• Participant received intravitreal steroid therapy for non-neovascular MacTel within the last 3 months
• Participant has ever received intravitreal anti-vascular endothelial growth factor (VEGF) therapy in the study eye or has, within the past 3 months, received intravitreal anti-VEGF in the fellow eye
• Participant has evidence of ocular disease other than MacTel that, in the judgment of the examining physician, may confound the diagnosis, procedures or outcome of the study
• Participant was a study participant in any other clinical trial of an intervention (drug or device) within the last 6 months
• Participant is pregnant or breastfeeding
• Participant has a chronic requirement (eg ≥ 4 weeks at a time) for ocular medications

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: DOUBLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
SHAM_COMPARATOR: Sham	Procedure: Sham; Non-penetrating sham procedure to mimic implant procedure.
EXPERIMENTAL: NT-501	Combination product: NT-501; Surgery to receive one NT-501 device implant.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of Change in Ellipsoid Zone (EZ) Area Loss	Rate of change in EZ area loss as measured by en face imaging by Spectral-Domain Optical Coherence Tomography (SD-OCT) in the study eye.	Baseline through 24 months.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean Change in Aggregate Sensitivity of Microperimetry	NT-501 compared to sham relative to mean change in aggregate sensitivity of microperimetry within the EZ line break area.	Baseline through 24 months.
National Eye Institute-Visual Function Questionnaire (NEI-VFQ)	NT-501 compared to sham relative to mean change in the NEI-VFQ near activities subscale score.	Baseline through 24 months.
Mean Change in Reading Speed	NT-501 compared to sham relative to mean change in reading speed assessed using International Reading Speed Texts (IReST) cards.	Baseline through 24 months.

Collaborators and Investigators

• Sponsor: Neurotech Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (ACTUAL): November 24, 2017
• Primary Completion (ACTUAL): August 31, 2022
• Study Completion (ACTUAL): September 23, 2022

Study Registration Dates

• First Submitted: October 3, 2017
• First Submitted That Met QC Criteria: October 19, 2017
• First Posted (ACTUAL): October 20, 2017

Study Record Updates

• Last Update Posted (ACTUAL): February 8, 2023
• Last Update Submitted That Met QC Criteria: February 3, 2023
• Last Verified: October 1, 2022

More Information

Terms related to this study

• Keywords: Ciliary Neurotrophic Factor (CNTF); Macular Telangiectasia (MacTel)
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Telangiectasis
• Other Study ID Numbers: NTMT-03-A

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: Yes