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

NCT03316300 | Completed Phase 3 Results DMC FDA Drug FDA Device

• 1 other identifier: NTMT-03-A
• Study Type: interventional
• Target: 120
• Locations: 4 countries
• Sites: 20

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 (MacTel)

Keywords

Ciliary Neurotrophic Factor (CNTF); Macular Telangiectasia Type 2; 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 subjects 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 Procedure

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 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 (20)

Scripps Clinic Medical Group

La Jolla, California, 92037, United States

Location

Jules Stein Eye Institute / David Geffen School of Medicine

Los Angeles, California, 90095, United States

Location

Byers Eye Institute at Stanford University

Palo Alto, California, 94303, United States

Location

Bay Area Retina Associates

Walnut Creek, California, 94598, United States

Location

Retina Consultants of Southern Colorado, P.C.

Colorado Springs, Colorado, 80909, United States

Location

Retina Associates of New Orleans

Metairie, Louisiana, 70006, United States

Location

National Institute of Health, NIH

Bethesda, Maryland, 20892, United States

Location

Cumberland Valley Retina Consultants

Hagerstown, Maryland, 21740, United States

Location

Associated Retinal Consultants, P.C.

Royal Oak, Michigan, 48073, United States

Location

Sierra Eye Associates

Reno, Nevada, 89502, United States

Location

Retina Associates of Cleveland, Inc.

Cleveland, Ohio, 44122, United States

Location

Tulsa Retina Consultants

Tulsa, Oklahoma, 74114, United States

Location

Retina Northwest, PC

Portland, Oregon, 97221, United States

Location

Southeastern Retina Associates, PC

Knoxville, Tennessee, 37922, United States

Location

Texas Retina Associates

Dallas, Texas, 75231, United States

Location

University of Utah John A. Moran Eye Center

Salt Lake City, Utah, 84132, United States

Location

Lions Eye Institute

Perth, Western Australia, 6009, Australia

Location

Lariboisiere Hospital

Paris, 75010, France

Location

Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust

Oxford, Oxfordshire, OX3 9DU, United Kingdom

Location

Moorfields Eye Hospital NHS Foundation Trust

London, EC1V 2PD, United Kingdom

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

Results Point of Contact

• Title: CMO
• Organization: Neurotech Pharmaceuticals, LLC

neurotech@neurotechusa.com

401-333-3880

Study Officials

• Paul S Bernstein, MD

  University of Utah - John A. Moran Eye Center

  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 20, 2017
• Study Start: November 24, 2017
• 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 (16); AU (1); FR (1); GB (2)