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

NCT03071965 | Completed Phase 2 Results FDA Drug

• 1 other identifier: NTMT-01/02E
• Study Type: interventional
• Target: 70
• Locations: 2 countries
• Sites: 11

Brief Summary

This was a prospective, multicenter, single-masked, sham-controlled extension study designed to provide long-term safety and efficacy follow-up data for subjects with MacTel who had NT-501 implanted intraocularly and/or underwent sham surgery in the respective precursor study (NTMT-01 or NTMT-02). A substudy was conducted in which subjects enrolled in study (NTMT-02), who had 1 study-eligible eye that underwent sham surgery, were offered the option to have NT-501 implanted in the same study eye. Of the 19 subjects who had 1 study-eligible eye and underwent sham surgery in the Cohort 2 precursor study (NTMT-02), 16 subjects elected to have NT-501 implanted in the same study eye during the substudy

Conditions

MacTel (Macular Telangiectasia) Type 2

Outcome Measures

Primary Outcomes (2)

• Ellipsoid Zone (Area of IS/OS Loss) - Cohort 1

  Change from baseline to 72, 84, 96 and 108 months post-surgery (based on participants' original implant date during precursor study) by SD-OCT for Cohort 1

  72, 84, 96, and 108 months (based on participants' original implant date during precursor study)

• Ellipsoid Zone (Area of IS/OS Loss) - Cohort 2

  Change in EZ from baseline to 36, 48, 60 and 72 Months post-surgery (based on participants' original implant date during precursor study). Cohort 2 sham surgery arm includes 16 participants (16 eyes) that received an NT-501 following their Month 36 follow-up

  Baseline to 36, 48, 60 and 72 Months post-surgery (based on participants' original implant date during precursor study)

Secondary Outcomes (4)

• Change in Retinal Sensitivity (dB) From Baseline - Cohort 2 Only

  Baseline to 72 Months (based on participants' original implant date)

• Change in BCVA From Baseline for Cohort 1

  Baseline to 108 Months (based on participants' original implant date)

• Change in BCVA From Baseline for Cohort 2

  Baseline to 72 Months (based on participants' original implant date)

• Change in Reading Speed From Baseline - Cohort 2 Only

  Baseline to 72 Months (based on participants' original implant date)

Study Arms (2)

Cohort 1

EXPERIMENTAL

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); Procedure: Surgery

Cohort 2

EXPERIMENTAL

Participants completed protocol NTMT-02. Participants for cohort 2 arm were either: * NT-501 group: included subjects who each had 1 study-eligible eye that had NT-501 implanted and a fellow eye that did not undergo any study intervention. * Sham group: included subjects who each had 1 study-eligible eye that underwent sham surgery and a fellow eye that did not undergo any study intervention. * Sham+NT-501 group: included subjects who had both eyes that were study-eligible, and had NT-501 implanted in 1 eye and underwent sham surgery in the contralateral eye.

Biological: Ciliary neurotrophic factor (CNTF); Procedure: Surgery

Interventions

Ciliary neurotrophic factor (CNTF) BIOLOGICAL

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.

Cohort 1; Cohort 2

Surgery PROCEDURE

Surgery to implant device for NT-501

Cohort 1; Cohort 2

Eligibility Criteria

• Age: 21 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

Sponsors & Collaborators

• Neurotech Pharmaceuticals: lead

Study Sites (11)

Stein Eye Institute / David Geffen School of Medicine

Los Angeles, California, 90095, United States

Location

University of Miami-Miller School of Medicine, Bascom Palmer Eye Institute

Miami, Florida, 33136, United States

Location

Emory University School of Medicine, Dept of Ophthalmology, Emory University Eye Center

Atlanta, Georgia, 30322, United States

Location

NIH Clinical Center

Rockville, Maryland, 20892, United States

Location

Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, 02114, United States

Location

University of Michigan, Kellogg Eye Center

Ann Arbor, Michigan, 48105, United States

Location

Retina Associates of Cleveland, Inc

Cleveland, Ohio, 44122, United States

Location

University of Wisconsin-Madison, Department of Ophthalmology and Visual Sciences

Madison, Wisconsin, 53705, United States

Location

Sydney Eye Hospital

Sydney, New South Wales, 2000, Australia

Location

Centre for Eye Research Australia

East Melbourne, Victoria, 3002, Australia

Location

Lions Eye Institute

Nedlands, Western Australia, 6009, Australia

Location

MeSH Terms

Interventions

Ciliary Neurotrophic Factor; Surgical Procedures, Operative

Intervention Hierarchy (Ancestors)

Nerve Growth Factors; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Nerve Tissue Proteins; Biological Factors

Results Point of Contact

• Title: CMO
• Organization: Neurotech Pharmaceuticals, LLC

neurotech@neurotechusa.com

401-333-3880

Study Officials

• Thomas Aaberg, Jr, MD

  Neurotech Pharmaceuticals, LLC

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 1, 2017
• First Posted: March 7, 2017
• Study Start: May 12, 2017
• Primary Completion: May 11, 2021
• Study Completion: May 11, 2021
• Last Updated: May 9, 2025
• Results First Posted: May 9, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

AU (3); US (8)