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

Phase 3

Completed

• Conditions: Macular Telangiectasia Type 2 (MacTel)
• Interventions: Combination Product: NT-501; Procedure: Sham Procedure

• Registration Number: NCT03316300
• Lead Sponsor: Neurotech Pharmaceuticals

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, France, and the United Kingdom.

Study Timeline

• Study First Submitted: 2017-10-03
• Study First Submitted QC: 2017-10-19
• Study First Posted: 2017-10-20
• Study Start: 2017-11-24
• Primary Completion: 2022-08-31
• Study Completion: 2022-09-23
• Status Verified: 2022-10
• Last Update Submitted: 2023-02-03
• Last Update Posted: 2023-02-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 115

Inclusion Criteria

• Participant must have at least one study eye with a positive diagnosis of MacTel Type 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 (SDOCT) between 0.16 mm^2 and 2.00 mm^2
• Participant's best corrected visual acuity is 54 letter score or better (20/80 or better) as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart

Key

Exclusion Criteria

• 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
• 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
• Participant was a study participant in any other clinical trial of an intervention (drug or device) within the last 6 months
• Participant is pregnant or breastfeeding
• Participant has a chronic requirement (eg ≥ 4 weeks at a time) for ocular medications

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
NT-501	NT-501	Test product
Sham	Sham Procedure	A sham surgical procedure was performed to mimic the implant procedure; there was no comparator product.

Primary Outcome Measures

Name	Time	Method
The Rate of Change in the Area of EZ Area of Loss From Baseline Through Month 24	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.	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.

Secondary Outcome Measures

Name	Time	Method
Mean Change in Aggregate Retinal Sensitivity Loss and Aggregate Interpolated Retinal Sensitivity Loss by Microperimetry (mITT Population)	Baseline through 24 months.	Change from baseline in retinal sensitivity loss as measured by as measured by Macular Integrity Assessment (MAIA)
Monocular Reading Speed (mITT Population)	Baseline through 24 months.	Change from baseline through Month 24 for Monocular reading speed assessed using International Reading Speed Texts (IReST) cards developed by the IReST Study Group 21

Trial Locations

Locations (20)

Associated Retinal Consultants, P.C.; 🇺🇸 Royal Oak, Michigan, United States

Scripps Clinic Medical Group; 🇺🇸 La Jolla, California, United States

Byers Eye Institute at Stanford University; 🇺🇸 Palo Alto, California, United States

National Institute of Health, NIH; 🇺🇸 Bethesda, Maryland, United States

Retina Northwest, PC; 🇺🇸 Portland, Oregon, United States

Lariboisiere Hospital; 🇫🇷 Paris, France

Moorfields Eye Hospital NHS Foundation Trust; 🇬🇧 London, United Kingdom

Jules Stein Eye Institute / David Geffen School of Medicine; 🇺🇸 Los Angeles, California, United States

Bay Area Retina Associates; 🇺🇸 Walnut Creek, California, United States

Retina Consultants of Southern Colorado, P.C.; 🇺🇸 Colorado Springs, Colorado, United States

Retina Associates of New Orleans; 🇺🇸 Metairie, Louisiana, United States

Cumberland Valley Retina Consultants; 🇺🇸 Hagerstown, Maryland, United States

Sierra Eye Associates; 🇺🇸 Reno, Nevada, United States

Retina Associates of Cleveland, Inc.; 🇺🇸 Cleveland, Ohio, United States

Tulsa Retina Consultants; 🇺🇸 Tulsa, Oklahoma, United States

Southeastern Retina Associates, PC; 🇺🇸 Knoxville, Tennessee, United States

Texas Retina Associates; 🇺🇸 Dallas, Texas, United States

University of Utah John A. Moran Eye Center; 🇺🇸 Salt Lake City, Utah, United States

Lions Eye Institute; 🇦🇺 Perth, Western Australia, Australia

Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust; 🇬🇧 Oxford, Oxfordshire, United Kingdom