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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06971939




Registration number
NCT06971939
Ethics application status
Date submitted
29/04/2025
Date registered
14/05/2025

Titles & IDs
Public title
Phase 4 Study: Long-term Safety and Efficacy of NT-501 in MacTel Type 2, Including Sham Procedure Participants
Scientific title
A Phase 4, Multicenter, Open-label Study to Evaluate Long-term Safety and Efficacy of Revakinagene Taroretcel-Lwey (NT-501) Previously Implanted During a Phase 1, Phase 2, or Phase 3 Clinical Study and to Evaluate NT-501 Implanted in Participants Who Underwent the Sham Procedure in a Phase 3 Clinical Study of Macular Telangiectasia Type 2 (MacTel)
Secondary ID [1] 0 0
NTMT-04
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Macular Telangiectasia Type 2 (MacTel) 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
Other interventions - NT-501/revakinagene taroretcel-lwey

Other: Arm 1 (Phase 3 Long-term Follow-up) - For participants who received NT-501 (ENCELTO) in a previous Phase 3 study (NTMT-03-A or NTMT-03-B), or for participants who received NT-501 (ENCELTO) in both eyes (i.e. participated in study NTMT-02B) but received the first NT-501 (ENCELTO) implant in a Phase 3 study. No new treatment intervention will be administered. Arm 2 is focused on long term follow-up for participant who already has the ENCELTO implant at time of study screening and enrollment.

Other: Arm 2 (Phase 1 and Phase 2 Long-term Follow-up) - For participants who received NT-501 (ENCELTO) in a previous Phase 1 study (NTMT-01), Phase 2 study (NTMT-02), in the sub study of Phase 1 and Phase 2 extension study (NTMT-01/02E), or for participants who received NT-501 (ENCELTO) in both eyes (i.e. participated in study NTMT-02B) but received the first NT-501 (ENCELTO) implant in a Phase 1 or Phase 2 study. No new treatment intervention will be administered. Arm 2 is focused on long term follow-up for participant who already has the ENCELTO implant at time of study screening and enrollment.

Experimental: Arm 3 (Phase 3 Sham to Implant Crossover) - For participants who underwent Sham procedure in either Phase 3 study (NTMT-03-A or NTMT-03-B) and will receive NT-501 in this study.


Other interventions: NT-501/revakinagene taroretcel-lwey
NT-501 is surgically implanted into the vitreous cavity to continuously release recombinant human ciliary neurotrophic factor (CNTF).
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of ocular and nonocular adverse events (AEs) and serious adverse events (SAEs)
Timepoint [1] 0 0
Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants
Primary outcome [2] 0 0
Percentage of participants with a loss of 15 letters or more in best corrected visual acuity (BCVA) compared to presurgery baseline BCVA
Timepoint [2] 0 0
Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants
Secondary outcome [1] 0 0
Change from presurgery baseline in ellipsoid zone (EZ) area loss
Timepoint [1] 0 0
Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants
Secondary outcome [2] 0 0
Change from presurgery baseline in BCVA
Timepoint [2] 0 0
Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants
Secondary outcome [3] 0 0
Change from presurgery baseline in aggregate retinal sensitivity
Timepoint [3] 0 0
Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants
Secondary outcome [4] 0 0
Change from presurgery baseline in monocular reading speed
Timepoint [4] 0 0
Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants
Secondary outcome [5] 0 0
Change from presurgery baseline in the National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25)
Timepoint [5] 0 0
Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants

Eligibility
Key inclusion criteria
Inclusion Criteria

To be eligible to participate in this study, an individual participant must meet all the following criteria:

1. Male or female adult participants who previously completed an NT-501 MacTel clinical study and:

1. Arm 1: received NT-501 intraocular implant in either Phase 3 study (NTMT-03-A or NTMT-03-B)
2. Arm 2: received NT-501 intraocular implant in the Phase 1 study (NTMT-01), Phase 2 study (NTMT-02), or in the substudy of the Phase 1 and Phase 2 extension study (NTMT-01/02E)

Note: participants who received NT-501 in both eyes (ie, participated in study NTMT-02B) will be enrolled in either Arm 1 or Arm 2 based on the study in which the first NT 501 was implanted (ie, participants who received the first implant in a Phase 3 study will be enrolled in Arm 1 and those who received the first implant in the Phase 1 study or Phase 2 study will be enrolled in Arm 2).
3. Arm 3: underwent the sham procedure in either Phase 3 study (NTMT-03-A or NTMT-03-B)
2. Participants must have steady fixation in the foveal or parafoveal area and sufficiently clear media for good quality retinal imaging.
3. Participant or their legally authorized representative must be able to provide written informed consent to participate in the study, in accordance with the International Conference on Harmonisation (ICH) Good Clinical Practice (GCP) guidelines, and local regulations, before initiating any clinical study related procedures.
4. Arm 3: female participants of childbearing potential must agree to use highly effective contraception. Highly effective forms of contraception (generally a first-year typical use pregnancy risk of 1% or less) include the following: contraceptive implants; an intrauterine device; permanent contraceptive procedures (vasectomy, bilateral salpingectomy, bilateral tubal occlusion, or partial salpingectomy); abstinence. A combination of male condom with either cap, diaphragm, or sponge with spermicide (ie, double barrier methods) is also considered an acceptable, but not highly effective, method of birth control.

Note: Participants enrolled at investigator sites in France or Germany must agree to use 2 forms of contraception listed above during study participation.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria

To participate in this study, the potential participant must not meet any of the following criteria. (Note that the ocular exclusion criteria are relevant to the study eye[s], unless indicated for both eyes):

Exclusion Criteria Applicable to Arms 1, 2, and 3:

1. Participant is medically unable to comply with clinical study procedures or visits.
2. 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.

Exclusion Criteria Applicable to Arm 3 Only:
3. Participant has significant corneal or media opacities in the study eye to be implanted.
4. Participant has evidence of pathologic myopia in the study eye to be implanted.
5. Participant has any of the following lens opacities in the study eye to be implanted: 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. Note: the participant can be reconsidered for enrollment 90 days after undergoing cataract surgery.
6. Participant has undergone lens removal in the study eye to be implanted in the previous 3 months or YAG laser within 4 weeks of the screening/baseline visit.
7. Participant has evidence of ocular disease in the study eye to be implanted 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).
8. Received intravitreal steroid therapy in the study eye to be implanted within the last year.
9. Received intravitreal anti-vascular endothelial growth factor therapy in the study eye to be implanted within the last 6 months.
10. Evidence of active exudative intraretinal neovascularization or subretinal neovascularization in the study eye to be implanted, as evidenced by hemorrhage, hard exudate, subretinal fluid, or intraretinal fluid.
11. Evidence of central serous chorioretinopathy in the study eye to be implanted.
12. Participant underwent vitrectomy, penetrating keratoplasty, trabeculectomy, or trabeculoplasty in the study eye to be implanted.
13. A history of ocular herpes virus in the study eye to be implanted.
14. Participant is on chemotherapy.
15. Participant is pregnant or breastfeeding.
16. Participation in any other clinical study of an intervention (drug or device) within 6 months prior to the screening/baseline visit.
17. A history of malignancy that would compromise survival during the clinical study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 4
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
15/06/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
15/06/2030
Actual
Sample size
Target
285
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Cerulea - East Melbourne
Recruitment postcode(s) [1] 0 0
- East Melbourne
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
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Michigan
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
Missouri
Country [8] 0 0
United States of America
State/province [8] 0 0
Oregon
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Tennessee
Country [11] 0 0
United States of America
State/province [11] 0 0
Texas
Country [12] 0 0
United States of America
State/province [12] 0 0
Utah
Country [13] 0 0
France
State/province [13] 0 0
Paris
Country [14] 0 0
Germany
State/province [14] 0 0
Bonn
Country [15] 0 0
Germany
State/province [15] 0 0
Münster
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Neurotech Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Thomas Aaberg, Jr, MD
Address 0 0
Neurotech Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Patricia Davis
Address 0 0
Country 0 0
Phone 0 0
401-333-3880
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


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/NCT06971939