Extension Study of NT-501 Ciliary Neurotrophic Factor (CNTF) Implant for Macular Telangiectasia (MacTel)

This study is a prospective, phase 2 extension study of participants previously enrolled in NTMT-01 and NTMT-02. This study is designed to evaluate long term safety and efficacy of the NT-501 implant in participants previously enrolled in the NTMT-01 and NTMT-02 protocols.

Study Overview

• Status: Completed
• Conditions: Macular Telangiectasia
• Intervention / Treatment: Biological: Ciliary neurotrophic factor (CNTF); Procedure: Surgery; Procedure: Surgery

• Study Type: Interventional
• Enrollment (Actual): 64
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia, 2000
      • Sydney Eye Hospital
  • Victoria
    • East Melbourne, Victoria, Australia, 3002
      • Centre for Eye Research Australia
  • Western Australia
    • Nedlands, Western Australia, Australia, 6009
      • Lions Eye Institute
• United States
  • California
    • Los Angeles, California, United States, 90095
      • Stein Eye Institute / David Geffen School of Medicine
  • Florida
    • Miami, Florida, United States, 33136
      • University of Miami-Miller School of Medicine, Bascom Palmer Eye Institute
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University School of Medicine, Dept of Ophthalmology, Emory University Eye Center
  • Maryland
    • Rockville, Maryland, United States, 20892
      • NIH Clinical Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts Eye and Ear Infirmary
  • Michigan
    • Ann Arbor, Michigan, United States, 48105
      • University of Michigan, Kellogg Eye Center
  • Ohio
    • Cleveland, Ohio, United States, 44122
      • Retina Associates of Cleveland, INC
  • Wisconsin
    • Madison, Wisconsin, United States, 53705
      • University of Wisconsin-Madison, Department of Ophthalmology and Visual Sciences

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 19 years to 78 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Previously enrolled in the NTMT-01 or NTMT-02 protocol and received the NT-501 implant and/or underwent a Sham procedure
• Participant must be offered sufficient opportunity to review and to understand the informed consent form, agree to the form's contents, and provide written informed consent.

Exclusion Criteria:

• There are no Exclusion Criteria

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1; Participants completed protocol NTMT-01. All participants received surgery to implant NT-501. All participants received ciliary neurotrophic factor (CNTF).	Biological: Ciliary neurotrophic factor (CNTF); The investigational product is the NT-501 encapsulated cell system, which consists of cells encapsulated within a semi-permeable polymer membrane and supportive matrices. NT-501 contains NTC-201 cells that secrete recombinant human CNTF, which were derived from genetically modified NTC-200 cells.; Procedure: Surgery; Surgery to implant device for NT-501; Procedure: Surgery; Sham surgery
Experimental: Cohort 2; Participants completed protocol NTMT-02. Participants received surgery to implant NT-501 or sham surgery to mimic implant procedure. Participants that received NT-501 implant were exposed to ciliary neurotrophic factor (CNTF).	Biological: Ciliary neurotrophic factor (CNTF); The investigational product is the NT-501 encapsulated cell system, which consists of cells encapsulated within a semi-permeable polymer membrane and supportive matrices. NT-501 contains NTC-201 cells that secrete recombinant human CNTF, which were derived from genetically modified NTC-200 cells.; Procedure: Surgery; Surgery to implant device for NT-501; Procedure: Surgery; Sham surgery

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Ellipsoid zone (area of IS/OS loss)	Change from baseline to 36, 48, 60, 72 months as measured by SD-OCT for Cohort 2	36, 48, 60, and 72 months
Ellipsoid zone (area of IS/OS loss)	Change from baseline to 72, 84, 96, and 108 months as measured by SD-OCT for Cohort 1	72, 84, 96, and 108 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Retinal sensitivity (dB)	Change from baseline to 36, 48, 60, and 72 months as measured by microperimetry for Cohort 2	36, 48, 60, and 72 months
Retinal sensitivity (dB)	Change from baseline to 72, 84, 96, and 108 months as measured by microperimetry for Cohort 1	72, 84, 96, and 108 months
Increase in ellipsoid zone (area of IS/OS loss)	Proportion of study eyes with a 35% or more increase from baseline at 36, 48, 60, and 72 months for Cohort 2	36, 48, 60, and 72 months
Increase in ellipsoid zone (area of IS/OS loss)	Proportion of study eyes with a 35% or more increase from baseline at 72, 84, 96, and 108 months for Cohort 1	72, 84, 96, and 108 months
Visual acuity	Change in best corrected visual acuity from baseline to 36, 48, 60, and 72 months for Cohort 2	36, 48, 60, and 72 months
Visual acuity	Change in best corrected visual acuity from baseline to 72, 84, 96, and 108 months for Cohort 1	72, 84, 96, and 108 months
Visual acuity	Proportion of study eyes with 15 or more letter loss in BCVA from baseline to 36, 48, 60, and 72 months for Cohort 2	36, 48, 60, and 72 months
Visual acuity	Proportion of study eyes with 15 or more letter loss in BCVA from baseline to 72, 84, 96, and 108 months for Cohort 1	72, 84, 96, and 108 months
Visual acuity	Proportion of study eyes with 10 or more letter loss in BCVA from baseline to 36, 48, 60, and 72 months for Cohort 2	36, 48, 60, and 72 months
Visual acuity	Proportion of study eyes with 10 or more letter loss in BCVA from baseline to 72, 84, 96, and 108 months for Cohort 1	72, 84, 96, and 108 months
Reading speed	Change from baseline to 36, 48, 60, and 72 months in reading speed as measured by IReST for Cohort 2	36, 48, 60, and 72 months
Reading speed	Change from baseline to 72, 84, 96, and 108 months in reading speed as measured by IReST for Cohort 1	72, 84, 96, and 108 months

Collaborators and Investigators

• Sponsor: Neurotech Pharmaceuticals
• Investigators: Study Chair: Thomas Hohman, PhD, Neurotech Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): May 12, 2017
• Primary Completion (Actual): May 11, 2021
• Study Completion (Actual): May 11, 2021

Study Registration Dates

• First Submitted: March 1, 2017
• First Submitted That Met QC Criteria: March 1, 2017
• First Posted (Actual): March 7, 2017

Study Record Updates

• Last Update Posted (Actual): March 17, 2022
• Last Update Submitted That Met QC Criteria: March 15, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Telangiectasis
• Other Study ID Numbers: NTMT-01/02E

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No