Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01949324




Registration number
NCT01949324
Ethics application status
Date submitted
19/09/2013
Date registered
24/09/2013
Date last updated
12/09/2018

Titles & IDs
Public title
A Phase 2 Multicenter Randomized Clinical Trial of CNTF for MacTel
Scientific title
A Phase 2 Multicenter Randomized Clinical Trial of Ciliary Neurotrophic Factor (CNTF) for Macular Telangiectasia Type 2 (MacTel)
Secondary ID [1] 0 0
NTMT-02
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Macular Telangiectasia Type 2 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Ciliary neurotrophic factor (CNTF)
Treatment: Surgery - Sham procedure
Treatment: Devices - NT-501 Implant
Treatment: Surgery - NT-501 Implant procedure

Experimental: NT-501 Implant procedure - The 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 comparator: Sham procedure - Non-penetrating sham procedure to mimic implant procedure


Treatment: Other: Ciliary neurotrophic factor (CNTF)
Ciliary neurotrophic factor released from NT-501 encapsulated cell implant

Treatment: Surgery: Sham procedure
Sham surgery for Sham arm

Treatment: Devices: NT-501 Implant
NT-501 encapsulated cell implant

Treatment: Surgery: NT-501 Implant procedure
Surgery to implant device for NT-501 encapsulated cell implant releasing human ciliary neurotrophic factor arm
Intervention code [1] 0 0
Treatment: Other
Intervention code [2] 0 0
Treatment: Surgery
Intervention code [3] 0 0
Treatment: Devices
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Ellipsoid zone (area of IS/OS loss) as measured by en face imagining by SDOCT in study eye(s)
Timepoint [1] 0 0
24 months
Secondary outcome [1] 0 0
Ellipsoid zone
Timepoint [1] 0 0
12 months
Secondary outcome [2] 0 0
Retinal sensitivity (dB) as measured by microperimetry
Timepoint [2] 0 0
12 and 24 months
Secondary outcome [3] 0 0
Increase in ellipsoid zone
Timepoint [3] 0 0
12 and 24 months
Secondary outcome [4] 0 0
Visual Acuity
Timepoint [4] 0 0
12 and 24 months
Secondary outcome [5] 0 0
Visual Acuity
Timepoint [5] 0 0
12 and 24 Months
Secondary outcome [6] 0 0
Visual Acuity
Timepoint [6] 0 0
12 and 24 Months
Secondary outcome [7] 0 0
Reading Speed
Timepoint [7] 0 0
12 and 24 Months

Eligibility
Key inclusion criteria
* 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
Minimum age
21 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/04/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
22/05/2017
Sample size
Target
Accrual to date
Final
67
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Save Sight Institute - Sydney
Recruitment hospital [2] 0 0
Centre for Eye Research Australia - East Melbourne
Recruitment hospital [3] 0 0
Lions Eye Institute - Nedlands
Recruitment postcode(s) [1] 0 0
- Sydney
Recruitment postcode(s) [2] 0 0
- East Melbourne
Recruitment postcode(s) [3] 0 0
- Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Wisconsin

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Neurotech Pharmaceuticals
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
The Lowy Medical Research Institute Limited
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Commercial sector/industry
Name [2] 0 0
The Emmes Company, LLC
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT01949324