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

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by Neurotech Pharmaceuticals
Sponsor:
Collaborators:
Lowy Medical Research Institute (LMRI)
The EMMES Corporation
Duke Reading Center
Information provided by (Responsible Party):
Neurotech Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01949324
First received: September 19, 2013
Last updated: April 8, 2014
Last verified: April 2014
  Purpose

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.


Condition Intervention Phase
Macular Telangiectasia Type 2
Biological: Ciliary neurotrophic factor (CNTF)
Procedure: Surgery
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Subject)
Primary Purpose: Treatment
Official Title: A Phase 2 Multicenter Randomized Clinical Trial of Ciliary Neurotrophic Factor (CNTF) for Macular Telangiectasia Type 2 (MacTel)

Resource links provided by NLM:


Further study details as provided by Neurotech Pharmaceuticals:

Primary Outcome Measures:
  • Ellipsoid zone (area of IS/OS loss) as measured by en face imagining by SDOCT in study eye(s) [ Time Frame: 24 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    Change in the ellipsoid zone from baseline to Month 12.

  • Retinal sensitivity (dB) as measured by microperimetry [ Time Frame: 12 and 24 months ] [ Designated as safety issue: No ]
    Change in retinal sensitivity (dB) as measured by microperimetry from baseline to Months 12 and 24.

  • Increase in ellipsoid zone [ Time Frame: 12 and 24 months ] [ Designated as safety issue: No ]
    Proportion of study eyes with a 35% or more increase from baseline in the ellipsoid zone at Months 12 and 24.

  • Visual Acuity [ Time Frame: 12 and 24 months ] [ Designated as safety issue: No ]
    Change in best corrected visual acuity (BCVA) from baseline to Months 12 and 24.

  • Visual Acuity [ Time Frame: 12 and 24 Months ] [ Designated as safety issue: No ]
    Proportion of study eyes with 15 or more letter loss from baseline in BCVA at Months 12 and 24.

  • Visual Acuity [ Time Frame: 12 and 24 Months ] [ Designated as safety issue: No ]
    Proportion of study eyes with 10 or more letter loss from baseline in BCVA at Months 12 and 24.

  • Reading Speed [ Time Frame: 12 and 24 Months ] [ Designated as safety issue: No ]
    Change in reading speed as measured by the IReST from baseline to Months 12 and 24.


Other Outcome Measures:
  • Cone density as measured by AOSLO [ Time Frame: 12 and 24 Months ] [ Designated as safety issue: No ]
    Change in cone density as measured by AOSLO from baseline to Months 12 and 24, in selected participants.

  • National Eye Institute Visual Functioning Questionnaire [ Time Frame: 12 and 24 months ] [ Designated as safety issue: No ]
    Change in NEI VFQ (overall and subscale) from baseline to Months 12 and 24.

  • Electroretinogram changes [ Time Frame: 6, 12 and 24 Months ] [ Designated as safety issue: Yes ]
    Electroretinogram (ERG) changes from baseline to Months 6, 12 and 24, in selected clinics/participants.


Estimated Enrollment: 68
Study Start Date: April 2014
Estimated Study Completion Date: September 2016
Estimated Primary Completion Date: June 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NT-501 Implant
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) Procedure: Surgery
Surgery to implant device for NT-501 arm and sham surgery for Sham arm
Sham Comparator: Sham procedure
Non-penetrating sham procedure to mimic implant procedure
Procedure: Surgery
Surgery to implant device for NT-501 arm and sham surgery for Sham arm

  Eligibility

Ages Eligible for Study:   21 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01949324

Contacts
Contact: Jennifer Trombley jtrombley@lmri.net

Locations
United States, California
Jules Stein Eye Institute Not yet recruiting
Los Angeles, California, United States, 90095
Principal Investigator: Steven Schwartz, MD         
United States, Florida
Bascom Palmer Not yet recruiting
Miami, Florida, United States, 33136
Principal Investigator: Philip Rosenfeld, MD, PhD         
United States, Maryland
National Eye Institute Not yet recruiting
Bethesda, Maryland, United States, 20892
Principal Investigator: Henry Wiley, MD         
United States, Michigan
University of Michigan, Kellogg Eye Center Recruiting
Ann Arbor, Michigan, United States, 48105
Principal Investigator: Grant Comer, MD         
United States, Wisconsin
University of Wisconsin Not yet recruiting
Madison, Wisconsin, United States, 53705
Principal Investigator: Barbara Blodi, MD         
Australia, New South Wales
Save Sight Institute Recruiting
Sydney, New South Wales, Australia
Principal Investigator: Mark Gillies, MD, PhD         
Australia
Centre for Eye Research Australia Recruiting
East Melbourne, Australia
Principal Investigator: Robyn Guymer, MD         
Lions Eye Institute Recruiting
Nedlands (Perth), Australia
Principal Investigator: Ian Constable, MD         
Sponsors and Collaborators
Neurotech Pharmaceuticals
Lowy Medical Research Institute (LMRI)
The EMMES Corporation
Duke Reading Center
  More Information

Additional Information:
No publications provided

Responsible Party: Neurotech Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01949324     History of Changes
Other Study ID Numbers: NTMT-02
Study First Received: September 19, 2013
Last Updated: April 8, 2014
Health Authority: United States: Food and Drug Administration
Australia: Therapeutic Goods Administration

Keywords provided by Neurotech Pharmaceuticals:
MacTel

Additional relevant MeSH terms:
Telangiectasis
Vascular Diseases
Cardiovascular Diseases

ClinicalTrials.gov processed this record on July 20, 2014