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

Start date

Nov 21, 2025

Status verified

May 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 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.

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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  PHASE3 · Not Yet Recruiting · The Lowy Medical Research Institute Limited · La Jolla, California