A Phase 2 Multicenter Randomized Clinical Trial of CNTF for MacTel
1 other identifier
interventional
67
2 countries
11
Brief Summary
This study is a phase 2, randomized, multi-center, single-masked study to evaluate the efficacy and safety of the NT-501 implants in participants with Mactel.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Apr 2014
Typical duration for phase_2
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 19, 2013
CompletedFirst Posted
Study publicly available on registry
September 24, 2013
CompletedStudy Start
First participant enrolled
April 1, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 14, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
May 22, 2017
CompletedSeptember 12, 2018
September 1, 2018
3 years
September 19, 2013
September 10, 2018
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Ellipsoid zone (area of IS/OS loss) as measured by en face imagining by SDOCT in study eye(s)
Change in the ellipsoid zone (area of IS/OS loss) from baseline to month 24 as measured by en face imaging by SDOCT in study eye(s)
24 months
Secondary Outcomes (7)
Ellipsoid zone
12 months
Retinal sensitivity (dB) as measured by microperimetry
12 and 24 months
Increase in ellipsoid zone
12 and 24 months
Visual Acuity
12 and 24 months
Visual Acuity
12 and 24 Months
- +2 more secondary outcomes
Other Outcomes (3)
Cone density as measured by AOSLO
12 and 24 Months
National Eye Institute Visual Functioning Questionnaire
12 and 24 months
Electroretinogram changes
6, 12 and 24 Months
Study Arms (2)
NT-501 Implant procedure
EXPERIMENTALThe investigational product is the NT-501 encapsulated cell system which consists of cells encapsulated within a semi-permeable polymer membrane and supportive matrices. NT-501 contains NTC-201 cells that were derived from the NTC-200 cell line by genetic modification so as to secrete recombinant human ciliary neurotrophic factor (CNTF).
Sham procedure
SHAM COMPARATORNon-penetrating sham procedure to mimic implant procedure
Interventions
Ciliary neurotrophic factor released from NT-501 encapsulated cell implant
Surgery to implant device for NT-501 encapsulated cell implant releasing human ciliary neurotrophic factor arm
Eligibility Criteria
You may qualify if:
- Participant must be offered sufficient opportunity to review and to understand the informed consent form, agree to the form's contents and sign the protocol's informed consent
- Participant must have at least one study eye with a positive diagnosis of MacTel Type 2
- Participant must have an IS/OS PR break in the study eye(s) and en face ellipsoid zone (area of IS/OS loss) as measured by SDOCT between 0.16 mm2 and 4.00 mm2
- If female, participant must be incapable of pregnancy
- If male, participant must agree to use an effective form of birth control during the study
You may not qualify if:
- Participant is unable to provide informed consent
- Participant is less than 21 years of age or greater than 80 years of age
- Participant is medically unable to comply with study procedures or follow-up visits
- Participant was a study subject in any other clinical trial of an intervention (drug or device) within the last 6 months
- Participant is pregnant or breastfeeding
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Neurotech Pharmaceuticalslead
- The Lowy Medical Research Institute Limitedcollaborator
- The Emmes Company, LLCcollaborator
Study Sites (11)
Jules Stein Eye Institute
Los Angeles, California, 90095, United States
Bascom Palmer
Miami, Florida, 33136, United States
Emory University
Atlanta, Georgia, 30322, United States
National Eye Institute
Bethesda, Maryland, 20892, United States
Massachusetts Eye and Ear Infirmary, Retina Service
Boston, Massachusetts, 02114, United States
University of Michigan, Kellogg Eye Center
Ann Arbor, Michigan, 48105, United States
Retina Associates of Cleveland, Inc.
Beachwood, Ohio, 44122, United States
University of Wisconsin
Madison, Wisconsin, 53705, United States
Save Sight Institute
Sydney, New South Wales, Australia
Centre for Eye Research Australia
East Melbourne, Australia
Lions Eye Institute
Nedlands, Australia
Related Publications (1)
Loo J, Cai CX, Choong J, Chew EY, Friedlander M, Jaffe GJ, Farsiu S. Deep learning-based classification and segmentation of retinal cavitations on optical coherence tomography images of macular telangiectasia type 2. Br J Ophthalmol. 2022 Mar;106(3):396-402. doi: 10.1136/bjophthalmol-2020-317131. Epub 2020 Nov 23.
PMID: 33229343DERIVED
MeSH Terms
Interventions
Intervention Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- SINGLE
- Who Masked
- PARTICIPANT
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 19, 2013
First Posted
September 24, 2013
Study Start
April 1, 2014
Primary Completion
April 14, 2017
Study Completion
May 22, 2017
Last Updated
September 12, 2018
Record last verified: 2018-09