Macular Telangiectasia Type 2 (MacTel)
Conditions
Keywords
Ciliary Neurotrophic Factor (CNTF), Macular Telangiectasia Type 2 (MacTel)
Brief summary
The purpose of this global, multicenter, open-label, Phase 4 clinical extension study is to evaluate the long-term safety and efficacy of revakinagene taroretcel-lwey (Encelto™; hereinafter referred to as NT-501), in participants with macular telangiectasia type 2 (MacTel) who previously received the intraocular implant in a Phase 1, Phase 2, or Phase 3 clinical study and have retained at least 1 NT-501 at study entry. In addition, this study will evaluate the safety and efficacy of NT501 in participants who previously underwent the sham procedure in a Phase 3 MacTel clinical study and elect to have NT-501 implanted intraocularly in this Phase 4 study.
Detailed description
This extension study consists of 3 arms: * Arm 1 (Phase 3 Treated Participants' Long-term Follow-up): Arm 1 is to assess the long-term safety and efficacy of NT-501 in participants who received NT-501 in a previous MacTel Phase 3 clinical study (NTMT-03-A or NTMT-03-B) and have retained at least 1 NT-501at study entry. * Arm 2 (Phase 1 and Phase 2 Long-term Follow-up): Similar to Arm 1, Arm 2 is to assess the long-term safety of NT-501 in participants who received NT-501 in a previous MacTel Phase 1 or Phase 2 clinical study (NTMT-01, NTMT-02, or NTMT-01/02E) and have retained at least 1 NT-501 at study entry. * Arm 3 (Phase 3 Sham to Implant Crossover): Arm 3 will evaluate the safety and efficacy of NT-501 in participants who previously underwent the sham procedure in a Phase 3 MacTel clinical study (NTMT-03-A or NTMT-03-B) and elect to have NT-501 implanted intraocularly in this extension study. Arms 1 and 2 are noninterventional and observational studies. For participants who previously received a single NT-501 implant, the implanted eye is designated the study eye. For participants who received an NT-501 in both eyes (ie, the first implant received in NTMT-01, NTMT-02, NTMT-01/02E, NTMT-03A, or NTMT-03B, and the second implant received in NTMT-02B), both eyes will be designated as study eyes. Only participants in Arm 3 will receive active intervention during this Phase 4 study. A participant can receive NT-501 in either eye that meets eligibility criteria according to investigator assessment, and in accordance with participant choice. However, if only 1 eye is eligible for implantation based on all inclusion/exclusion criteria, then that eye will be selected for NT-501 implantation. The eye selected for NT-501 implantation will be designated as the study eye. Regardless of study eye designation, both eyes of each participant in all 3 study arms will be evaluated during the study.
Interventions
COMBINATION_PRODUCTNT-501/revakinagene taroretcel-lwey
NT-501 is surgically implanted into the vitreous cavity to continuously release recombinant human ciliary neurotrophic factor (CNTF).
Sponsors
Neurotech Pharmaceuticals
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Arms 1 and 2: For participants who previously received a single NT-501, the implanted eye is designated the study eye. For participants who received an NT-501 implant in both eyes (ie. the first implant received in NTMT-01, NTMT-02, NTMT 01/02E, NTMT-03-A, or NTMT-03-B, and the second implant received in NTMT-02B), both eyes will be designated as study eyes (the eye in which NT-501 was originally implanted will be study eye 1 and the other implanted eye will be study eye 2). Arm 3: The implanted eye will be designated as the study eye in Arm 3. Note, if NT-501 in not implanted in the Sham eye (ie. the eye that did not undergo intervention during the parent Phase 3 clinical study), then the sham eye will also be considered a study eye for long-term follow-up. Regardless of study eye designation, both eyes of each participant in all 3 study arms will be evaluated during the study.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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) and have retained at least 1 NT-501 at study entry. 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) and have retained at least 1 NT-501 at study entry. 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. 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. 3. 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.
Exclusion criteria
To participate in this study, the potential participant must not meet any of the following criteria. (Note that the ocular
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of ocular and nonocular adverse events (AEs) and serious adverse events (SAEs) | 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 | Incidence of ocular and nonocular adverse events (AEs) and serious adverse events (SAEs) |
| Percentage of participants with a loss of 15 letters or more in best corrected visual acuity (BCVA) compared to presurgery baseline BCVA | 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 | Percentage of participants with a loss of 15 letters or more in best corrected visual acuity (BCVA) compared to presurgery baseline BCVA using the Early Treatment Diabetic Retinopathy Study (ETDRS) distance chart |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from presurgery baseline in ellipsoid zone (EZ) area loss | 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 | Change from presurgery baseline in ellipsoid zone (EZ) area loss as measured by en face spectral domain optical coherence tomography (SD-OCT) for both eyes |
| Change from presurgery baseline in BCVA | 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 | Change from presurgery baseline in BCVA using the ETDRS distance chart for both eyes |
| Change from presurgery baseline in aggregate retinal sensitivity | 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 | Change from presurgery baseline in aggregate retinal sensitivity as measured by microperimetry within the EZ line break area for both eyes |
| Change from presurgery baseline in monocular reading speed | 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 | Change from presurgery baseline in monocular reading speed for both eyes |
| Change from presurgery baseline in the National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25) | 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 | Change from presurgery baseline in NEI-VFQ-25 near activities subscale score. All items are scored so that a high score represents better functioning. Each item is then converted to a 0 to 100 scale so that the lowest and highest possible scores are set at 0 and 100 points, respectively. |
Countries
Australia, United States
Contacts
CONTACTPatricia Davis
STUDY_CHAIRThomas Aaberg, Jr, MD
Neurotech Pharmaceuticals
Outcome results
None listed