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

NCT ID: NCT03319849

Last Updated: 2024-09-24

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 119 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-01-22
• Study Completion Date: 2022-09-23

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.

Detailed Description

Phase 3, prospective, multicenter, masked, sham-controlled study with the overall study objective to evaluate the efficacy and safety of NT-501 for the treatment of MacTel. Secondary objective was to evaluate the safety of NT-501 in participants with MacTel. This was a multicenter study conducted at 20 study centers in the United States, Australia, Germany, and the United Kingdom.

Conditions

Macular Telangiectasia Type 2

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

NT-501

Test product

Group Type: EXPERIMENTAL

NT-501

Intervention Type: 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.

Sham

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

Group Type: SHAM_COMPARATOR

Sham

Intervention Type: PROCEDURE

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

Interventions

NT-501

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.

Intervention Type: COMBINATION_PRODUCT

Sham

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

Intervention Type: PROCEDURE

Eligibility Criteria

Inclusion Criteria

1. 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

2. 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

3. 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.

4. Participant must have steady fixation in the foveal or parafoveal area and sufficiently clear media for good quality photographs

5. Participant must be greater than 21 years of age or less than 80 years of age at screening

6. 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

7. Women of childbearing potential must agree to use highly effective contraception (Germany and France only)

Exclusion Criteria

1. Participant is medically unable to comply with study procedures or follow-up visits

2. Participant received intravitreal steroid therapy for non-neovascular MacTel within the last 3 months

3. 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

4. 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)

5. 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)

6. Participant has evidence of intraretinal neovascularization or subretinal neovascularization (SRNV), as evidenced by hemorrhage, hard exudate, subretinal fluid or intraretinal fluid in either eye

7. Participant has evidence of central serous chorio-retinopathy in either eye

8. Participant has evidence of pathologic myopia in either eye

9. Participant has significant corneal or media opacities in either eye

10. Participant has had a vitrectomy, penetrating keratoplasty, trabeculectomy, or trabeculoplasty

11. 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

12. Participant has undergone lens removal in the previous 3 months or YAG laser within 4 weeks

13. Participant was a participant in any other clinical trial of an intervention (drug or device) within the last 6 months

14. Participant is on chemotherapy

15. Participant is pregnant or breastfeeding

16. Participant has a history of malignancy that would compromise the 24-month study survival

17. Participant with a history of ocular herpes virus in either eye

18. Participant has, in the opinion of the investigator, any physical or mental condition that would increase the risk of participation in the study or may interfere with the study procedures, evaluations, and outcome assessments

19. Participant has evidence of intraretinal hyperreflectivity by OCT

• Minimum Eligible Age: 21 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The Lowy Medical Research Institute Limited

OTHER

Sponsor Role: collaborator

Neurotech Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Mark Gillies, MD

Role: PRINCIPAL_INVESTIGATOR

Save Sight/Sydney, Australia

Locations

Retina Vitreous Assoc

Beverly Hills, California, United States

Retina Consultants of Southern Colorado, P.C.

Colorado Springs, Colorado, United States

Emory University Eye Center

Atlanta, Georgia, United States

Northwestern Medical Group

Chicago, Illinois, United States

Elman Retina Group, PA

Baltimore, Maryland, United States

Massachusetts Eye and Ear Infirmary Ophthalmology Clinical Research Office

Boston, Massachusetts, United States

New England Retina Consultants

Springfield, Massachusetts, United States

University of Michigan, Kellogg Eye Center

Ann Arbor, Michigan, United States

Mayo Clinic

Rochester, Minnesota, United States

The Retina Institute

Chesterfield, Missouri, United States

Retina Center of New Jersey - Envision Ocular, LLC

Bloomfield, New Jersey, United States

Flaum Eye - University of Rochester

Rochester, New York, United States

University of Rochester Strong Memorial Hospital

Rochester, New York, United States

Cincinnati Eye Institute

Cincinnati, Ohio, United States

Tulsa Retina Consultants

Tulsa, Oklahoma, United States

Retina Northwest, PC

Portland, Oregon, United States

Scheie Eye Institute - University of Pennsylvania

Philadelphia, Pennsylvania, United States

Palmetto Retina Center

West Columbia, South Carolina, United States

Southeastern Retina Associates, PC

Knoxville, Tennessee, United States

Austin Clinical Research, LLC

Austin, Texas, United States

Retina Consultants of Texas

Bellaire, Texas, United States

Retina Center of Texas

Southlake, Texas, United States

Save Sight Institute, Sydney

Sydney, New South Wales, Australia

Centre for Eye Research Australia Macular Research Unit

East Melbourne, Victoria, Australia

Klinik at Freiberg, Germany

Freiburg im Breisgau, Baden-Wurttemberg Germany, Germany

University of Bonn

Bonn, North Rhine-Westphalia, Germany

St. Franziskus, Muenster

Münster, North Rhine-Westphalia, Germany

Countries

United States; Australia; Germany

References

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.

Reference Type: DERIVED

PMID: 40693847 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

NTMT-03-B

Identifier Type: -

Identifier Source: org_study_id