NCT03319849CompletedPhase 3
A Phase III Multicenter Randomized, Sham Controlled, Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2
Neurotech Pharmaceuticals27 sites in 3 countries119 target enrollmentStarted: January 22, 2018Last updated:
Conditions
Interventions
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Enrollment
- 119
- Locations
- 27
- Primary Endpoint
- The Rate of Change in the Area of EZ Area of Loss From Baseline Through Month 24
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Outcomes Assessor)
Masking Description
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.
Eligibility Criteria
- Ages
- 21 Years to 80 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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)
Exclusion Criteria
- •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
- •Participant has a history of malignancy that would compromise the 24-month study survival
- •Participant with a history of ocular herpes virus in either eye
- •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
- •Participant has evidence of intraretinal hyperreflectivity by OCT
Arms & Interventions
Sham
Sham Comparator
A sham surgical procedure was performed to mimic the implant procedure; there was no comparator product.
Intervention: Sham (Procedure)
NT-501
Experimental
Test product
Intervention: NT-501 (Combination Product)
Outcomes
Primary Outcomes
The Rate of Change in the Area of EZ Area of Loss From Baseline Through Month 24
Time Frame: 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 participants with MacTel.
Secondary Outcomes
- 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.)
Investigators
Study Sites (27)
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