A Phase 2 Multicenter Randomized Clinical Trial of CNTF for MacTel

A Phase 2 Multicenter Randomized Clinical Trial of Ciliary Neurotrophic Factor (CNTF) for Macular Telangiectasia Type 2 (MacTel)

This study is a phase 2, randomized, multi-center, single-masked study to evaluate the efficacy and safety of the NT-501 implants in participants with Mactel.

Study Overview

• Status: Completed
• Conditions: Macular Telangiectasia Type 2
• Intervention / Treatment: Biological: Ciliary neurotrophic factor (CNTF); Device: NT-501 Implant; Procedure: NT-501 Implant procedure; Procedure: Sham procedure

• Study Type: Interventional
• Enrollment (Actual): 67
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • East Melbourne, Australia
    • Centre for Eye Research Australia
  • Nedlands, Australia
    • Lions Eye Institute
  • New South Wales
    • Sydney, New South Wales, Australia
      • Save Sight Institute
• United States
  • California
    • Los Angeles, California, United States, 90095
      • Jules Stein Eye Institute
  • Florida
    • Miami, Florida, United States, 33136
      • Bascom Palmer
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • National Eye Institute
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts Eye and Ear Infirmary, Retina Service
  • Michigan
    • Ann Arbor, Michigan, United States, 48105
      • University of Michigan, Kellogg Eye Center
  • Ohio
    • Beachwood, Ohio, United States, 44122
      • Retina Associates of Cleveland, Inc.
  • Wisconsin
    • Madison, Wisconsin, United States, 53705
      • University of Wisconsin

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:

• Participant must be offered sufficient opportunity to review and to understand the informed consent form, agree to the form's contents and sign the protocol's informed consent
• Participant must have at least one study eye with a positive diagnosis of MacTel Type 2
• Participant must have an IS/OS PR break in the study eye(s) and en face ellipsoid zone (area of IS/OS loss) as measured by SDOCT between 0.16 mm2 and 4.00 mm2
• If female, participant must be incapable of pregnancy
• If male, participant must agree to use an effective form of birth control during the study

Exclusion Criteria:

• Participant is unable to provide informed consent
• Participant is less than 21 years of age or greater than 80 years of age
• Participant is medically unable to comply with study procedures or follow-up visits
• Participant was a study subject in any other clinical trial of an intervention (drug or device) within the last 6 months
• Participant is pregnant or breastfeeding

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NT-501 Implant procedure; 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 were derived from the NTC-200 cell line by genetic modification so as to secrete recombinant human ciliary neurotrophic factor (CNTF).	Biological: Ciliary neurotrophic factor (CNTF); Ciliary neurotrophic factor released from NT-501 encapsulated cell implant; Device: NT-501 Implant; NT-501 encapsulated cell implant; Procedure: NT-501 Implant procedure; Surgery to implant device for NT-501 encapsulated cell implant releasing human ciliary neurotrophic factor arm
Sham Comparator: Sham procedure; Non-penetrating sham procedure to mimic implant procedure	Procedure: Sham procedure; Sham surgery for Sham arm

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Ellipsoid zone (area of IS/OS loss) as measured by en face imagining by SDOCT in study eye(s)	Change in the ellipsoid zone (area of IS/OS loss) from baseline to month 24 as measured by en face imaging by SDOCT in study eye(s)	24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Ellipsoid zone	Change in the ellipsoid zone from baseline to Month 12.	12 months
Retinal sensitivity (dB) as measured by microperimetry	Change in retinal sensitivity (dB) as measured by microperimetry from baseline to Months 12 and 24.	12 and 24 months
Increase in ellipsoid zone	Proportion of study eyes with a 35% or more increase from baseline in the ellipsoid zone at Months 12 and 24.	12 and 24 months
Visual Acuity	Change in best corrected visual acuity (BCVA) from baseline to Months 12 and 24.	12 and 24 months
Visual Acuity	Proportion of study eyes with 15 or more letter loss from baseline in BCVA at Months 12 and 24.	12 and 24 Months
Visual Acuity	Proportion of study eyes with 10 or more letter loss from baseline in BCVA at Months 12 and 24.	12 and 24 Months
Reading Speed	Change in reading speed as measured by the IReST from baseline to Months 12 and 24.	12 and 24 Months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cone density as measured by AOSLO	Change in cone density as measured by AOSLO from baseline to Months 12 and 24, in selected participants.	12 and 24 Months
National Eye Institute Visual Functioning Questionnaire	Change in NEI VFQ (overall and subscale) from baseline to Months 12 and 24.	12 and 24 months
Electroretinogram changes	Electroretinogram (ERG) changes from baseline to Months 6, 12 and 24, in selected clinics/participants.	6, 12 and 24 Months

Collaborators and Investigators

• Sponsor: Neurotech Pharmaceuticals
• Collaborators: The Emmes Company, LLC; The Lowy Medical Research Institute Limited

Publications and helpful links

General Publications

• Loo J, Cai CX, Choong J, Chew EY, Friedlander M, Jaffe GJ, Farsiu S. Deep learning-based classification and segmentation of retinal cavitations on optical coherence tomography images of macular telangiectasia type 2. Br J Ophthalmol. 2022 Mar;106(3):396-402. doi: 10.1136/bjophthalmol-2020-317131. Epub 2020 Nov 23.

Study record dates

Study Major Dates

• Study Start: April 1, 2014
• Primary Completion (Actual): April 14, 2017
• Study Completion (Actual): May 22, 2017

Study Registration Dates

• First Submitted: September 19, 2013
• First Submitted That Met QC Criteria: September 19, 2013
• First Posted (Estimate): September 24, 2013

Study Record Updates

• Last Update Posted (Actual): September 12, 2018
• Last Update Submitted That Met QC Criteria: September 10, 2018
• Last Verified: September 1, 2018

More Information

Terms related to this study

• Keywords: MacTel
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Telangiectasis
• Other Study ID Numbers: NTMT-02

Drug and device information, study documents

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