Safety Study to Evaluate Bilateral CNTF Implants in Subjects With MacTel Type 2

NCT04729972 | Completed Phase 2 Results FDA Drug FDA Device

• 1 other identifier: NTMT-02B
• Study Type: interventional
• Target: 33
• Locations: 2 countries
• Sites: 10

Brief Summary

This is a multi-center, open-label, 6-month study to evaluate the safety of bilateral CNTF implants in participants with Macular Telangiectasia Type 2.

Conditions

Macular Telangiectasia Type 2

Keywords

Macular Telangiectasia Type 2; CNTF; Mactel

Outcome Measures

Primary Outcomes (1)

• Frequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events

  Assess the incidence and severity of adverse events (AEs) following bilateral ocular implantation of NT-501

  6 months

Secondary Outcomes (1)

• Serum Levels of CNTF and Immunogenicity for NT-501

  6 months

Study Arms (1)

NT-501 in Fellow Eye

EXPERIMENTAL

NT-501 was implanted on Day 0 in the fellow eye of subjects who received a single NT-501 in a previous study; subjects were followed for 6 months post-implantation of NT-501 in this study.

Combination Product: NT-501 CNTF implant

Interventions

NT-501 CNTF implant COMBINATION_PRODUCT

Single implantation of CNTF-secreting NT-501 device into fellow (untreated) eye

NT-501 in Fellow Eye

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants from the NTMT-03 study must have completed the Month-24 visit
• Participant in the MacTel Phase 1/2 extension (NTMT-01/02E) study or the Phase 3 study must exit these studies prior to entering the Bilateral Implant safety study (NTMT-02B)
• Participant must be willing and able to follow the study instructions and be willing and able to complete all required study procedures and visits
• Participant must be willing and able to provide a signed Informed Consent, as well as written documentation in accordance with the relevant country and local privacy requirements, e.g., written data protection consent
• Females of childbearing potential must consent to a pregnancy test before entering the study
• A participant's refusal to allow the collection of blood samples for analysis of serum CNTF, serum Ab or Nab to CNTF, Ab to NTC-201.6A cells and Ab to DFHR levels in one eye will not exclude the participant from study participation
• Participant must have a positive diagnosis of MacTel type 2 with evidence of fluorescein leakage typical of MacTel and at least one of the other features that include hyperpigmentation that is outside of a 500-micron radius from the center of the fovea, retinal opacification, crystalline deposits, right-angle vessels, or inner/outer lamellar cavities in the study eye
• Participant must have steady fixation in the foveal or parafoveal area in the study eye and sufficiently clear media for good quality photographs

You may not qualify if:

• Females of childbearing potential (those who are not surgically sterilized or post- menopausal, i.e., absence of menstruation for 12 months or longer) may not participate in the study if any of the following conditions exists:
• Pregnant (positive pregnancy test at Visit 1 or intend to become pregnant during the study)
• Nursing (lactating)
• Do not agree to use adequate birth control methods for the duration of the study and until 90 days after the last administration of study drug (adequate birth control methods are: hormonal - oral, implantable, transdermal or injectable contraceptives; mechanical - spermicide in conjunction with a barrier such as condom or diaphragm, intrauterine device \[IUD\] or surgical sterilization of partner, or total sexual abstinence)
• Participant is too ill to likely complete the entire study, based on the investigator's assessment
• Participant, in the opinion of the investigator, is not suitable for study participation
• Participant with any screening laboratory finding (serum chemistry, hematology, urinalysis) that in the opinion of the investigator is not suitable for study participation
• Participant has a history or current evidence of severe hypersensitivity to the NT-501 implant
• Participant has a history or current evidence of a medical condition (systemic or ophthalmic disease, metabolic dysfunction, physical examination finding or clinical laboratory finding) that may in the opinion of the investigator preclude the safe administration of the NT-501 implant or adherence to the scheduled study visits, safe participation in the study or affect the results of the study (e.g., unstable or progressive cardiovascular, cerebral vascular, pulmonary, Parkinson's, liver or renal disease; depression, cancer, or dementia
• Participant has a history or evidence of the following surgeries/procedures in the study eye, as assessed at Visit 1, including:
• Submacular surgery
• Vitrectomy
• Retinal detachment
• Incisional glaucoma surgery
• Trabeculectomy or trabeculoplasty

Sponsors & Collaborators

• Neurotech Pharmaceuticals: lead
• The Lowy Medical Research Institute Limited: collaborator

Study Sites (10)

Jules Stein Eye Institute

Los Angeles, California, 90095, United States

Location

National Eye Institute

Washington D.C., District of Columbia, 20892, United States

Location

Bascom Palmer

Miami, Florida, 33136, United States

Location

Massachusetts Eye and Ear Infirmary, Retina Service

Boston, Massachusetts, 02114, United States

Location

University of Michigan, Kellogg Eye Center

Ann Arbor, Michigan, 48105, United States

Location

Retina Associates of Cleveland, Inc.

Beachwood, Ohio, 44122, United States

Location

Retina Consultants of Texas

Houston, Texas, 77030, United States

Location

Save Sight Institute

Sydney, New South Wales, Australia

Location

Centre for Eye Research Australia

East Melbourne, Victoria, 3002, Australia

Location

Lions Eye Institute

Nedlands, Western Australia, 6009, Australia

Location

Related Publications (1)

• Chew EY, Clemons TE, Jaffe GJ, Johnson CA, Farsiu S, Lad EM, Guymer R, Rosenfeld P, Hubschman JP, Constable I, Wiley H, Singerman LJ, Gillies M, Comer G, Blodi B, Eliott D, Yan J, Bird A, Friedlander M; Macular Telangiectasia Type 2-Phase 2 CNTF Research Group. Effect of Ciliary Neurotrophic Factor on Retinal Neurodegeneration in Patients with Macular Telangiectasia Type 2: A Randomized Clinical Trial. Ophthalmology. 2019 Apr;126(4):540-549. doi: 10.1016/j.ophtha.2018.09.041. Epub 2018 Oct 4.

  PMID: 30292541 RESULT

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 DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 25, 2021
• First Posted: January 29, 2021
• Study Start: July 21, 2021
• Primary Completion: December 8, 2022
• Study Completion: December 8, 2022
• Last Updated: March 26, 2025
• Results First Posted: March 26, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (7); AU (3)