A Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2 - Protocol B

NCT03319849 | Completed Phase 3 Results DMC FDA Drug FDA Device

• 1 other identifier: NTMT-03-B
• Study Type: interventional
• Target: 119
• Locations: 3 countries
• Sites: 27

Brief Summary

This study is a phase 3, randomized, multi-center study to evaluate the efficacy and safety of the NT-501 implants in participants with macular telangiectasia type 2.

Conditions

Macular Telangiectasia Type 2

Keywords

Ciliary Neurotrophic Factor (CNTF); Macular Telangiectasia (MacTel); MacTel

Outcome Measures

Primary Outcomes (1)

• The Rate of Change in the Area of EZ Area of Loss From Baseline Through Month 24

  The rate of change in the area of EZ loss (IS/OS; macular photoreceptor loss) from baseline through month 24, as assessed using SD-OCT in the study eye of participants with MacTel.

  End point timeframe is through Month 24. Baseline, Month 6, 12, 16, 20 and 24. Month 6 was collected but not included in the primary analyses.

Secondary Outcomes (2)

• Mean Change in Aggregate Retinal Sensitivity Loss and Aggregate Interpolated Retinal Sensitivity Loss by Microperimetry (mITT Population)

  Baseline through 24 months.

• Monocular Reading Speed (mITT Population)

  Baseline through 24 months.

Study Arms (2)

NT-501

EXPERIMENTAL

Test product

Combination Product: NT-501

Sham

SHAM COMPARATOR

A sham surgical procedure was performed to mimic the implant procedure; there was no comparator product.

Procedure: Sham

Interventions

NT-501 COMBINATION_PRODUCT

Each NT-501 implant consisted of hCNTF-secreting NTC-201-6A.02 cells encapsulated within supportive matrices and surrounded by a semipermeable polymer membrane. The NTC-201-6A cells continuously secrete CNTF from the NT-501 implant into the vitreous cavity. Implanted by a qualified Health Care Professional.

NT-501

Sham PROCEDURE

The sham surgery involved a superficial conjunctival incision performed under local anesthetic and closure with a single suture.

Sham

Eligibility Criteria

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

You may qualify if:

• Participant must have at least one study eye with a positive diagnosis of MacTel 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
• Participant must have an Inner Segment - Outer Segment Junction Line (IS/OS) Photo Receptor (PR) break in the study eye(s) and en face EZ (area of IS/OS loss) as measured by spectral-domain optical coherence tomography (SD-OCT) between 0.16 mm\^2 and 2.00 mm\^2
• Participant's best corrected visual acuity (BCVA) is a 54-letter score or better (20/80 or better) as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart at screening.
• Participant must have steady fixation in the foveal or parafoveal area and sufficiently clear media for good quality photographs
• Participant must be greater than 21 years of age or less than 80 years of age at screening
• Participant must be able to provide written informed consent to participate in the study, in accordance with the International Conference on Harmonisation Good Clinical Practices guidelines, and local regulations, before initiating any study-related procedures
• Women of childbearing potential must agree to use highly effective contraception (Germany and France only)

You may not qualify if:

• Participant is medically unable to comply with study procedures or follow-up visits
• Participant received intravitreal steroid therapy for non-neovascular MacTel within the last 3 months
• Participant has ever received intravitreal anti-vascular endothelial growth factor (VEGF) therapy in the study eye OR has, within the past 3 months, received intravitreal anti-VEGF in the fellow eye at randomization
• Participant has evidence of ocular disease other than MacTel that, in the judgment of the examining physician, may confound the diagnosis, procedures or outcome of the study (eg, glaucoma, severe nonproliferative or proliferative diabetic retinopathy, uveitis)
• Participant has a chronic requirement (eg, ≥ 4 weeks at a time) for ocular medications and/or has a diagnosed disease that, in the judgment of the examining physician, may be vision threatening or may affect the primary outcome (artificial tears are permitted)
• Participant has evidence of intraretinal neovascularization or subretinal neovascularization (SRNV), as evidenced by hemorrhage, hard exudate, subretinal fluid or intraretinal fluid in either eye
• Participant has evidence of central serous chorio-retinopathy in either eye
• Participant has evidence of pathologic myopia in either eye
• Participant has significant corneal or media opacities in either eye
• Participant has had a vitrectomy, penetrating keratoplasty, trabeculectomy, or trabeculoplasty
• Participant has any of the following lens opacities: cortical opacity \> standard 3, posterior subcapsular opacity \> standard 2, or a nuclear opacity \> standard 3 as measured on the Age-Related Eye Disease Study (AREDS) clinical lens grading system
• Participant has undergone lens removal in the previous 3 months or YAG laser within 4 weeks
• Participant was a participant in any other clinical trial of an intervention (drug or device) within the last 6 months
• Participant is on chemotherapy
• Participant is pregnant or breastfeeding

Sponsors & Collaborators

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

Study Sites (27)

Retina Vitreous Assoc

Beverly Hills, California, 90211, United States

Location

Retina Consultants of Southern Colorado, P.C.

Colorado Springs, Colorado, 80909, United States

Location

Emory University Eye Center

Atlanta, Georgia, 30322, United States

Location

Northwestern Medical Group

Chicago, Illinois, 60611, United States

Location

Elman Retina Group, PA

Baltimore, Maryland, 21237, United States

Location

Massachusetts Eye and Ear Infirmary Ophthalmology Clinical Research Office

Boston, Massachusetts, 02114, United States

Location

New England Retina Consultants

Springfield, Massachusetts, 01107, United States

Location

University of Michigan, Kellogg Eye Center

Ann Arbor, Michigan, 48105, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

The Retina Institute

Chesterfield, Missouri, 63017, United States

Location

Retina Center of New Jersey - Envision Ocular, LLC

Bloomfield, New Jersey, 07003, United States

Location

Flaum Eye - University of Rochester

Rochester, New York, 14642, United States

Location

University of Rochester Strong Memorial Hospital

Rochester, New York, 14642, United States

Location

Cincinnati Eye Institute

Cincinnati, Ohio, 45242, United States

Location

Tulsa Retina Consultants

Tulsa, Oklahoma, 74114, United States

Location

Retina Northwest, PC

Portland, Oregon, 97221, United States

Location

Scheie Eye Institute - University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Palmetto Retina Center

West Columbia, South Carolina, 29169, United States

Location

Southeastern Retina Associates, PC

Knoxville, Tennessee, 37922, United States

Location

Austin Clinical Research, LLC

Austin, Texas, 78705, United States

Location

Retina Consultants of Texas

Bellaire, Texas, 77401, United States

Location

Retina Center of Texas

Southlake, Texas, 76092, United States

Location

Save Sight Institute, Sydney

Sydney, New South Wales, 2000, Australia

Location

Centre for Eye Research Australia Macular Research Unit

East Melbourne, Victoria, 3002, Australia

Location

Klinik at Freiberg, Germany

Freiburg im Breisgau, Baden-Wurttemberg Germany, 79106, Germany

Location

University of Bonn

Bonn, North Rhine-Westphalia, 53127, Germany

Location

St. Franziskus, Muenster

Münster, North Rhine-Westphalia, 48145, Germany

Location

Related Publications (1)

• Chew EY, Gillies M, Jaffe GJ, Gaudric A, Egan C, Constable I, Clemons T, Aaberg T, Manning DC, Hohman TC, Bird A, Friedlander M; MacTel CNTF NTMT-03 Research investigators. Cell-Based Ciliary Neurotrophic Factor Therapy for Macular Telangiectasia Type 2. NEJM Evid. 2025 Aug;4(8):EVIDoa2400481. doi: 10.1056/EVIDoa2400481. Epub 2025 Jul 22.

  PMID: 40693847 DERIVED

MeSH Terms

Interventions

salicylhydroxamic acid

Results Point of Contact

• Title: CMO
• Organization: Neurotech Pharmaceuticals, LLC

neurotech@neurotechusa.com

401-333-3880

Study Officials

• Mark Gillies, MD

  Save Sight/Sydney, Australia

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Masking Details: The subjects and all personnel at the image reading center remained masked to the treatment assignment throughout the study. In addition, the refractionist, VA examiner, and photographers/imagers were masked to treatment assignment (NT-501 implantation or sham procedure) at all follow-up visits. The ophthalmologist, surgeon, and clinic coordinator were instructed not to discuss the assigned treatment with the subject.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: After confirming eligibility in conjunction with the reading center, the study eye of each subject was randomized (1:1) to either have NT-501 implanted or to undergo the sham procedure.

Study Record Dates

• First Submitted: October 3, 2017
• First Posted: October 24, 2017
• Study Start: January 22, 2018
• Primary Completion: August 31, 2022
• Study Completion: September 23, 2022
• Last Updated: September 24, 2024
• Results First Posted: September 24, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (22); DE (3); AU (2)