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A Phase 2 Multicenter Randomized Clinical Trial of CNTF for MacTel

Primary Purpose

Macular Telangiectasia Type 2

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Ciliary neurotrophic factor (CNTF)
Sham procedure
NT-501 Implant
NT-501 Implant procedure
Sponsored by
Neurotech Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Macular Telangiectasia Type 2 focused on measuring MacTel

Eligibility Criteria

21 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Jules Stein Eye Institute
  • Bascom Palmer
  • Emory University
  • National Eye Institute
  • Massachusetts Eye and Ear Infirmary, Retina Service
  • University of Michigan, Kellogg Eye Center
  • Retina Associates of Cleveland, Inc.
  • University of Wisconsin
  • Save Sight Institute
  • Centre for Eye Research Australia
  • Lions Eye Institute

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Sham Comparator

Arm Label

NT-501 Implant procedure

Sham procedure

Arm Description

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).

Non-penetrating sham procedure to mimic implant procedure

Outcomes

Primary Outcome Measures

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)

Secondary Outcome Measures

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

Full Information

First Posted
September 19, 2013
Last Updated
September 10, 2018
Sponsor
Neurotech Pharmaceuticals
Collaborators
The Lowy Medical Research Institute Limited, The Emmes Company, LLC
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1. Study Identification

Unique Protocol Identification Number
NCT01949324
Brief Title
A Phase 2 Multicenter Randomized Clinical Trial of CNTF for MacTel
Official Title
A Phase 2 Multicenter Randomized Clinical Trial of Ciliary Neurotrophic Factor (CNTF) for Macular Telangiectasia Type 2 (MacTel)
Study Type
Interventional

2. Study Status

Record Verification Date
September 2018
Overall Recruitment Status
Completed
Study Start Date
April 2014 (undefined)
Primary Completion Date
April 14, 2017 (Actual)
Study Completion Date
May 22, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurotech Pharmaceuticals
Collaborators
The Lowy Medical Research Institute Limited, The Emmes Company, LLC

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
Yes
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Macular Telangiectasia Type 2
Keywords
MacTel

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
Participant
Allocation
Randomized
Enrollment
67 (Actual)

8. Arms, Groups, and Interventions

Arm Title
NT-501 Implant procedure
Arm Type
Experimental
Arm Description
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).
Arm Title
Sham procedure
Arm Type
Sham Comparator
Arm Description
Non-penetrating sham procedure to mimic implant procedure
Intervention Type
Biological
Intervention Name(s)
Ciliary neurotrophic factor (CNTF)
Intervention Description
Ciliary neurotrophic factor released from NT-501 encapsulated cell implant
Intervention Type
Procedure
Intervention Name(s)
Sham procedure
Intervention Description
Sham surgery for Sham arm
Intervention Type
Device
Intervention Name(s)
NT-501 Implant
Intervention Description
NT-501 encapsulated cell implant
Intervention Type
Procedure
Intervention Name(s)
NT-501 Implant procedure
Intervention Description
Surgery to implant device for NT-501 encapsulated cell implant releasing human ciliary neurotrophic factor arm
Primary Outcome Measure Information:
Title
Ellipsoid zone (area of IS/OS loss) as measured by en face imagining by SDOCT in study eye(s)
Description
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)
Time Frame
24 months
Secondary Outcome Measure Information:
Title
Ellipsoid zone
Description
Change in the ellipsoid zone from baseline to Month 12.
Time Frame
12 months
Title
Retinal sensitivity (dB) as measured by microperimetry
Description
Change in retinal sensitivity (dB) as measured by microperimetry from baseline to Months 12 and 24.
Time Frame
12 and 24 months
Title
Increase in ellipsoid zone
Description
Proportion of study eyes with a 35% or more increase from baseline in the ellipsoid zone at Months 12 and 24.
Time Frame
12 and 24 months
Title
Visual Acuity
Description
Change in best corrected visual acuity (BCVA) from baseline to Months 12 and 24.
Time Frame
12 and 24 months
Title
Visual Acuity
Description
Proportion of study eyes with 15 or more letter loss from baseline in BCVA at Months 12 and 24.
Time Frame
12 and 24 Months
Title
Visual Acuity
Description
Proportion of study eyes with 10 or more letter loss from baseline in BCVA at Months 12 and 24.
Time Frame
12 and 24 Months
Title
Reading Speed
Description
Change in reading speed as measured by the IReST from baseline to Months 12 and 24.
Time Frame
12 and 24 Months
Other Pre-specified Outcome Measures:
Title
Cone density as measured by AOSLO
Description
Change in cone density as measured by AOSLO from baseline to Months 12 and 24, in selected participants.
Time Frame
12 and 24 Months
Title
National Eye Institute Visual Functioning Questionnaire
Description
Change in NEI VFQ (overall and subscale) from baseline to Months 12 and 24.
Time Frame
12 and 24 months
Title
Electroretinogram changes
Description
Electroretinogram (ERG) changes from baseline to Months 6, 12 and 24, in selected clinics/participants.
Time Frame
6, 12 and 24 Months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
21 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
Jules Stein Eye Institute
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Bascom Palmer
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
National Eye Institute
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Facility Name
Massachusetts Eye and Ear Infirmary, Retina Service
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
University of Michigan, Kellogg Eye Center
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48105
Country
United States
Facility Name
Retina Associates of Cleveland, Inc.
City
Beachwood
State/Province
Ohio
ZIP/Postal Code
44122
Country
United States
Facility Name
University of Wisconsin
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53705
Country
United States
Facility Name
Save Sight Institute
City
Sydney
State/Province
New South Wales
Country
Australia
Facility Name
Centre for Eye Research Australia
City
East Melbourne
Country
Australia
Facility Name
Lions Eye Institute
City
Nedlands
Country
Australia

12. IPD Sharing Statement

Citations:
PubMed Identifier
33229343
Citation
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.
Results Reference
derived

Learn more about this trial

A Phase 2 Multicenter Randomized Clinical Trial of CNTF for MacTel

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