Clinical trial NCT03319849: A Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2 - Protocol B
Official title: A Phase III Multicenter Randomized, Sham Controlled, Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Macular Telangiectasia Type 2
Keywords: Ciliary Neurotrophic Factor (CNTF)
Macular Telangiectasia (MacTel)
MacTel
Lead sponsor: Neurotech Pharmaceuticals
Sponsor class: INDUSTRY
Collaborators: name: The Lowy Medical Research Institute Limited
First posted: 2017-10-24
Enrollment: 119
Sex: ALL
Minimum age (years): 21
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: 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.
Primary purpose: TREATMENT

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.

Eligibility criteria: Key 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)

Key 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

Primary outcomes:
measure: The Rate of Change in the Area of EZ Area of Loss From Baseline Through Month 24
timeFrame: 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:
measure: Mean Change in Aggregate Retinal Sensitivity Loss and Aggregate Interpolated Retinal Sensitivity Loss by Microperimetry (mITT Population)
timeFrame: Baseline through 24 months.
measure: Monocular Reading Speed (mITT Population)
timeFrame: Baseline through 24 months.
Other outcomes:
Locations:
city: Beverly Hills
country: United States
facility: Retina Vitreous Assoc
state: California
city: Colorado Springs
country: United States
facility: Retina Consultants of Southern Colorado, P.C.
state: Colorado
city: Atlanta
country: United States
facility: Emory University Eye Center
state: Georgia
city: Chicago
country: United States
facility: Northwestern Medical Group
state: Illinois
city: Baltimore
country: United States
facility: Elman Retina Group, PA
state: Maryland
city: Boston
country: United States
facility: Massachusetts Eye and Ear Infirmary Ophthalmology Clinical Research Office
state: Massachusetts
city: Springfield
country: United States
facility: New England Retina Consultants
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan, Kellogg Eye Center
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Chesterfield
country: United States
facility: The Retina Institute
state: Missouri
city: Bloomfield
country: United States
facility: Retina Center of New Jersey - Envision Ocular, LLC
state: New Jersey
city: Rochester
country: United States
facility: Flaum Eye - University of Rochester
state: New York
city: Rochester
country: United States
facility: University of Rochester Strong Memorial Hospital
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Eye Institute
state: Ohio
city: Tulsa
country: United States
facility: Tulsa Retina Consultants
state: Oklahoma
city: Portland
country: United States
facility: Retina Northwest, PC
state: Oregon
city: Philadelphia
country: United States
facility: Scheie Eye Institute - University of Pennsylvania
state: Pennsylvania
city: West Columbia
country: United States
facility: Palmetto Retina Center
state: South Carolina
city: Knoxville
country: United States
facility: Southeastern Retina Associates, PC
state: Tennessee
city: Austin
country: United States
facility: Austin Clinical Research, LLC
state: Texas
city: Bellaire
country: United States
facility: Retina Consultants of Texas
state: Texas
city: Southlake
country: United States
facility: Retina Center of Texas
state: Texas
city: Sydney
country: Australia
facility: Save Sight Institute, Sydney
state: New South Wales
city: East Melbourne
country: Australia
facility: Centre for Eye Research Australia Macular Research Unit
state: Victoria
city: Freiburg
country: Germany
facility: Klinik at Freiberg, Germany
state: Baden-Wurttemberg Germany
city: Bonn
country: Germany
facility: University of Bonn
state: NRW
city: Muenster
country: Germany
facility: St. Franziskus, Muenster
state: NRW
Study officials:
affiliation: Save Sight/Sydney, Australia
name: Mark Gillies, MD
role: PRINCIPAL_INVESTIGATOR
References: