Phase 4 Study: Long-term Safety and Efficacy of NT-501 in MacTel Type 2, Including Sham Procedure Participants

Conditions

• Macular Telangiectasia Type 2 (MacTel)

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• NT-501/revakinagene taroretcel-lwey — COMBINATION_PRODUCT
  NT-501 is surgically implanted into the vitreous cavity to continuously release recombinant human ciliary neurotrophic factor (CNTF).

Study Details

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. 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.

Key Dates

First listed

May 14, 2025

Start date

Nov 21, 2025

Status verified

Jul 2026

Primary completion

Jan 15, 2032

Completion

Jan 15, 2032

Study Design

Enrollment

285 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• 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 1 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.

Primary Outcome Measure

Incidence of ocular and nonocular adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: 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 ]

Central Contacts

• Patricia Davis
  401-333-3880
  [email protected]

Locations (23)

Find similar trials in La Jolla, CA

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