A Phase 2 Multicenter Randomized Clinical Trial of CNTF for MacTel

Phase 2

Completed

• Conditions: Macular Telangiectasia Type 2
• Interventions: Biological: Ciliary neurotrophic factor (CNTF); Procedure: Sham procedure; Device: NT-501 Implant; Procedure: NT-501 Implant procedure

• Registration Number: NCT01949324
• Lead Sponsor: Neurotech Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2013-09-19
• Study First Submitted QC: 2013-09-19
• Study First Posted: 2013-09-24
• Study Start: 2014-04
• Primary Completion: 2017-04-14
• Study Completion: 2017-05-22
• Disposition First Submitted: 2018-05-04
• Disposition First Submitted QC: 2018-05-04
• Disposition First Posted: 2018-05-08
• Status Verified: 2018-09
• Last Update Submitted: 2018-09-10
• Last Update Posted: 2018-09-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 67

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
NT-501 Implant procedure	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).
NT-501 Implant procedure	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).
NT-501 Implant procedure	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 were derived from the NTC-200 cell line by genetic modification so as to secrete recombinant human ciliary neurotrophic factor (CNTF).
Sham procedure	Sham procedure	Non-penetrating sham procedure to mimic implant procedure

Primary Outcome Measures

Name	Time	Method
Ellipsoid zone (area of IS/OS loss) as measured by en face imagining by SDOCT in study eye(s)	24 months	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

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

Trial Locations

Locations (11)

Jules Stein Eye Institute; 🇺🇸 Los Angeles, California, United States

Bascom Palmer; 🇺🇸 Miami, Florida, United States

Emory University; 🇺🇸 Atlanta, Georgia, United States

University of Michigan, Kellogg Eye Center; 🇺🇸 Ann Arbor, Michigan, United States

Retina Associates of Cleveland, Inc.; 🇺🇸 Beachwood, Ohio, United States

Save Sight Institute; 🇦🇺 Sydney, New South Wales, Australia

Centre for Eye Research Australia; 🇦🇺 East Melbourne, Australia

Lions Eye Institute; 🇦🇺 Nedlands, Australia

National Eye Institute; 🇺🇸 Bethesda, Maryland, United States

Massachusetts Eye and Ear Infirmary, Retina Service; 🇺🇸 Boston, Massachusetts, United States

University of Wisconsin; 🇺🇸 Madison, Wisconsin, United States