Macular Telangiectasia Type 2Phase 3NCT06397131
A Study to Determine the Safety and Efficacy of NT-501 With M-CHFM
A Phase 3 study testing M-CHFM and CHFM in people with Macular Telangiectasia Type 2, run by Neurotech Pharmaceuticals at 6 sites in 2 countries.
Trial summary
The overall purpose of this global, multicenter, two cohort, central image evaluator masked study is to characterize the clinical safety and efficacy of NT-501 manufactured using the Medica - Clinical Hollow Fiber Membrane (M-CHFM) for the treatment of MacTel and to evaluate whether its safety profile and efficacy are… Read more about this study
- Who is it for?
- Ages 21 to 80 years
- Conditions the study is for
- Macular Telangiectasia Type 2
- What is being tested?
- M-CHFM and CHFM
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 6 Mar 2025to 24 Jul 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- To participate in this study, the potential subject and at least 1 of their eyes must meet all of the following criteria:
- Subjects must have a positive diagnosis of MacTel with evidence of fluorescein leakage typical of MacTel and at least one of the other features that include hyperpigmentation that is outside of a 500-micron radius from the center of the fovea, retinal opacification, crystalline deposits, right-angle vessels, or inner/outer lamellar cavities seen clinically or on SD-OCT.
- Subject must have a break in the ellipsoid zone (EZ) (area of IS/OS loss) as measured by SD-OCT between 0.16 and 2.00 millimeters squared.
- Subject's best-corrected visual acuity (BCVA) is 54-letter score or better (20/80 or better) as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart at screening.
- Subject must have steady fixation in the foveal or parafoveal area and sufficiently clear media for good quality retinal imaging.
- Subject must be greater than or equal to 21 years of age and less than 80 years of age at screening.
- Subject 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 Good Clinical Practices guidelines, and local regulations, before initiating any study-related procedures.
Who cannot join
- To participate in this study, the potential subject must not meet any of the following criteria. The ocular exclusion criteria are related to the study eye (unless indicated for either eye):
- Subject is medically unable to comply with study procedures or follow-up visits.
- Subject received intravitreal steroid therapy for non-neovascular MacTel within the last 3 months.
- Subject has ever received intravitreal anti-vascular endothelial growth factor (VEGF) therapy in the study eye OR has, within the past 3 months, received intravitreal anti-VEGF therapy in the fellow eye at randomization.
- Subject has evidence of ocular disease other than MacTel that, in the judgment of the examining physician, may confound the diagnosis, procedures, or outcome of the study (e.g., uncontrolled glaucoma, severe non-proliferative or proliferative diabetic retinopathy, uveitis).
- Subject has a chronic requirement (e.g., ≥ 4 weeks at a time) for ocular medications and/or has a diagnosed disease that, in the judgment of the examining physician, may be vision threatening or may affect the primary outcome (artificial tears are permitted). The exceptions to this exclusion criteria include artificial tears or one glaucoma medication for early or intermediate primary open-angle glaucoma.
Show all 22Show fewer
- Subject has evidence of intraretinal neovascularization or subretinal neovascularization (SRNV), as evidenced by hemorrhage, hard exudate, subretinal fluid or intraretinal fluid in either eye.
- Subject has evidence of intraretinal hyperreflectivity by OCT.
- Subject has evidence of central serous chorio-retinopathy in either eye.
- Subject has evidence of pathologic myopia in either eye.
- Subject has significant corneal or media opacities in either eye.
- Subject has had a vitrectomy, penetrating keratoplasty, trabeculectomy, or trabeculoplasty.
- Subject has any of the following lens opacities: 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.
- Subject has undergone lens removal in the previous 3 months or YAG laser within 4 weeks.
- Previous participation in any Neurotech MacTel clinical trial unless the participant: (1) previously consented and screened for a MacTel study but was a screen failure solely due to insufficient lesion size; or (2) has completed participation in the prior Neurotech MacTel study, is not actively enrolled in another Neurotech MacTel study, and has an eligible study eye that was not the study eye in the previous trial.
- Subject was a participant in any other ocular related or ocular impacted clinical trial of an intervention (drug or device) within the last 6 months.
- Subject is on chemotherapy.
- Subject is pregnant or breastfeeding.
- Subject has a history of malignancy that would compromise the 12-month study survival.
- Subject with a history of ocular herpes virus in either eye.
- Subject 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.
- Subject has evidence of subfoveal RPE hyperplasia.
Trial locations and contacts
6 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
6 sites in 2 countries
United States3 sites
- Active, not recruiting
Retina Consultants of Texas
Bellaire, Texas, 77401
- Active, not recruiting
Foundation for Vision Research
Grand Rapids, Michigan, 49546
- Active, not recruiting
Bay Area Retina Associates, A Medical Group
Walnut Creek, California, 94598
Australia3 sites, 3 recruiting
- Not yet recruiting
Cerulea
East Melbourne
Thuy Chau, tchau@ceruleaclinicaltrials.org.au
Penelope Allen, MD
- Recruiting
Lion's Eye Institute
Perth
Diana Bowman, diana.bowman@lei.org.au
Ian Constable, MD
- Not yet recruiting
Sydney Eye Hospital
Sydney
Emily Luu, emily.luu@sydney.edu.au
Elisa Cornish, MD
About this study
Official title: A Multicenter Study to Determine the Safety and Efficacy of NT-501 Utilizing the Medica Membrane in Macular Telangiectasia Type 2 (MacTel), Including a Randomized Comparative Expansion Cohort
The overall purpose of this global, multicenter, two cohort, central image evaluator masked study is to characterize the clinical safety and efficacy of NT-501 manufactured using the Medica - Clinical Hollow Fiber Membrane (M-CHFM) for the treatment of MacTel and to evaluate whether its safety profile and efficacy are comparable to those of NT-501 manufactured using the Clinical Hollow Fiber Membrane (CHFM).
Read the detailed description
This Phase 3 study consists of two cohorts:
- Cohort 1 (Extension): Includes participants previously enrolled under the original protocol who received NT-501 (M-CHFM) and will continue follow-up. Participants will undergo evaluations of safety and efficacy from pre-surgery through month 24 or in the event of NT-501 explantation for any reason.
- Cohort 2 (Expansion): Includes newly enrolled participants randomized 1:1 to receive either NT-501 (M-CHFM) or NT-501 (CHFM). Participants will undergo evaluations of safety and efficacy from pre-surgery through month 12 or in the event of NT-501 explantation for any reason.
- Conditions
- Macular Telangiectasia Type 2
- Being tested
- M-CHFM, CHFM
- Keywords
- Ciliary Neurotrophic Factor, Macular Telangiectasia (MacTel) Type 2
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
M-CHFMCombination product
Single implantation of NT-501 manufactured using the Medica - Clinical Hollow Fiber Membrane (M-CHFM)
CHFMCombination product
Also called ENCELTO
Single implantation of NT-501 manufactured using the Clinical Hollow Fiber Membrane (CHFM)
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 (Extension) - M-CHFM | Experimental | Previously enrolled Cohort 1 participants received M-CHFM and will undergo evaluations of safety and efficacy from pre-surgery through month 24 or in the event of NT-501 explantation for any reason. Receives: Combination Product: M-CHFM |
| Cohort 2 (Expansion) - M-CHFM | Experimental | Participants are randomized 1:1 to receive M-CHFM. Participants will undergo evaluations of safety and efficacy from pre-surgery through month 12 or in the event of NT-501 explantation for any reason. Receives: Combination Product: M-CHFM |
| Cohort 2 (Expansion) - CHFM | Active comparator | Participants are randomized 1:1 to receive CHFM (US commercially available ENCELTO). Participants will undergo evaluations of safety and efficacy from pre-surgery through month 12 or in the event of NT-501 explantation for any reason. Receives: Combination Product: CHFM |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cohort 1 (Extension) - Rate of change in EZ area loss as measured by en face imaging by Spectral-Domain Optical Coherence Tomography
Rate of change in EZ area loss as measured by en face imaging by Spectral-Domain Optical Coherence Tomography (SD-OCT) in the study eye of NT-501 in participants who received NT-501 with M-CHFM
Measured: Baseline to 24 months
Cohort 2 (Expansion) - Rate of change in EZ area loss as measured by en face imaging by Spectral-Domain Optical Coherence Tomography
Rate of change in EZ area loss as measured by en face imaging by Spectral-Domain Optical Coherence Tomography (SD-OCT) in the study eye of NT-501 in participants who received NT-501 with M-CHFM as compared to NT-501 with CHFM
Measured: Baseline to 12 months
Secondary outcomes (4)
Cohort 1 (Extension) - Incidence of ocular and non-ocular adverse events
Incidence of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs)
Measured: Baseline to 24 months
Cohort 2 (Expansion) - Incidence of ocular and non-ocular adverse events
Incidence of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs)
Measured: Baseline to 12 months
Cohort 1 (Extension) - Percentage of participants with a loss of 15 letters or more in best-corrected visual acuity
Percentage of participants with a loss of 15 letters or more in best-corrected visual acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) distance chart
Measured: Baseline to 24 months
Cohort 2 (Expansion) - Percentage of participants with a loss of 15 letters or more in best-corrected visual acuity
Percentage of participants with a loss of 15 letters or more in best-corrected visual acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) distance chart
Measured: Baseline to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 May 2024
- Study start6 Mar 2025
- Primary completion1 May 2028 (estimated)
- Study completion24 Jul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Macular Telangiectasia Type 2Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Cohort 1 is a non-randomized, single-group cohort in which all participants received M-CHFM. Cohort 2 is a 1:1 randomized, parallel-group comparison of CHFM and M-CHFM; each participant receives one implant type.
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Neurotech Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Thomas Aaberg, Jr., MD, Study chair, Neurotech Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06397131
- Sponsor's protocol ID
- NTMT-03C
- Status last verified
- Nov 2026
- First submitted
- 29 Apr 2024
- Last updated
- 5 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Study to Determine the Safety and Efficacy of NT-501 Utilizing the Medica Membrane in Macular Telangiectasia Type 2 (MacTel), Including a Randomized Comparative Expansion Cohort
- ClinicalTrials.gov ID
- NCT06397131
- Acronym / sponsor ID
- NTMT-03C
- Conditions
- Macular Telangiectasia Type 2
- Keywords
- Ciliary Neurotrophic Factor, Macular Telangiectasia (MacTel) Type 2
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Neurotech PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 6 Mar 2025 (actual)
- Primary completion
- 1 May 2028 (estimated)
- Study completion
- 24 Jul 2028 (estimated)
- First posted
- 2 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The overall purpose of this global, multicenter, two cohort, central image evaluator masked study is to characterize the clinical safety and efficacy of NT-501 manufactured using the Medica - Clinical Hollow Fiber Membrane (M-CHFM) for the treatment of MacTel and to evaluate whether its safety profile and efficacy are comparable to those of NT-501 manufactured using the Clinical Hollow Fiber Membrane (CHFM).
Detailed description
This Phase 3 study consists of two cohorts:
- Cohort 1 (Extension): Includes participants previously enrolled under the original protocol who received NT-501 (M-CHFM) and will continue follow-up. Participants will undergo evaluations of safety and efficacy from pre-surgery through month 24 or in the event of NT-501 explantation for any reason.
- Cohort 2 (Expansion): Includes newly enrolled participants randomized 1:1 to receive either NT-501 (M-CHFM) or NT-501 (CHFM). Participants will undergo evaluations of safety and efficacy from pre-surgery through month 12 or in the event of NT-501 explantation for any reason.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 3
- Model description
- Cohort 1 is a non-randomized, single-group cohort in which all participants received M-CHFM. Cohort 2 is a 1:1 randomized, parallel-group comparison of CHFM and M-CHFM; each participant receives one implant type.
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (Extension) - M-CHFM | Experimental | Combination Product: M-CHFMPreviously enrolled Cohort 1 participants received M-CHFM and will undergo evaluations of safety and efficacy from pre-surgery through month 24 or in the event of NT-501 explantation for any reason. |
| Cohort 2 (Expansion) - M-CHFM | Experimental | Combination Product: M-CHFMParticipants are randomized 1:1 to receive M-CHFM. Participants will undergo evaluations of safety and efficacy from pre-surgery through month 12 or in the event of NT-501 explantation for any reason. |
| Cohort 2 (Expansion) - CHFM | Active comparator | Combination Product: CHFMParticipants are randomized 1:1 to receive CHFM (US commercially available ENCELTO). Participants will undergo evaluations of safety and efficacy from pre-surgery through month 12 or in the event of NT-501 explantation for any reason. |
Who may qualify
- Ages
- 21 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- To participate in this study, the potential subject and at least 1 of their eyes must meet all of the following criteria:
- Subjects must have a positive diagnosis of MacTel with evidence of fluorescein leakage typical of MacTel and at least one of the other features that include hyperpigmentation that is outside of a 500-micron radius from the center of the fovea, retinal opacification, crystalline deposits, right-angle vessels, or inner/outer lamellar cavities seen clinically or on SD-OCT.
- Subject must have a break in the ellipsoid zone (EZ) (area of IS/OS loss) as measured by SD-OCT between 0.16 and 2.00 millimeters squared.
- Subject's best-corrected visual acuity (BCVA) is 54-letter score or better (20/80 or better) as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart at screening.
- Subject must have steady fixation in the foveal or parafoveal area and sufficiently clear media for good quality retinal imaging.
Key exclusion criteria (22 listed)
- To participate in this study, the potential subject must not meet any of the following criteria. The ocular exclusion criteria are related to the study eye (unless indicated for either eye):
- Subject is medically unable to comply with study procedures or follow-up visits.
- Subject received intravitreal steroid therapy for non-neovascular MacTel within the last 3 months.
- Subject has ever received intravitreal anti-vascular endothelial growth factor (VEGF) therapy in the study eye OR has, within the past 3 months, received intravitreal anti-VEGF therapy in the fellow eye at randomization.
- Subject has evidence of ocular disease other than MacTel that, in the judgment of the examining physician, may confound the diagnosis, procedures, or outcome of the study (e.g., uncontrolled glaucoma, severe non-proliferative or proliferative diabetic retinopathy, uveitis).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| M-CHFMSingle implantation of NT-501 manufactured using the Medica - Clinical Hollow Fiber Membrane (M-CHFM) | Combination product | Cohort 1 (Extension) - M-CHFM, Cohort 2 (Expansion) - M-CHFM |
| CHFMAlso: ENCELTOSingle implantation of NT-501 manufactured using the Clinical Hollow Fiber Membrane (CHFM) | Combination product | Cohort 2 (Expansion) - CHFM |
Primary outcome
Cohort 1 (Extension) - Rate of change in EZ area loss as measured by en face imaging by Spectral-Domain Optical Coherence Tomography
Time frame: Baseline to 24 months
Rate of change in EZ area loss as measured by en face imaging by Spectral-Domain Optical Coherence Tomography (SD-OCT) in the study eye of NT-501 in participants who received NT-501 with M-CHFM
Cohort 2 (Expansion) - Rate of change in EZ area loss as measured by en face imaging by Spectral-Domain Optical Coherence Tomography
Time frame: Baseline to 12 months
Rate of change in EZ area loss as measured by en face imaging by Spectral-Domain Optical Coherence Tomography (SD-OCT) in the study eye of NT-501 in participants who received NT-501 with M-CHFM as compared to NT-501 with CHFM
4 secondary outcomes
Cohort 1 (Extension) - Incidence of ocular and non-ocular adverse events
Time frame: Baseline to 24 months
Cohort 2 (Expansion) - Incidence of ocular and non-ocular adverse events
Time frame: Baseline to 12 months
Cohort 1 (Extension) - Percentage of participants with a loss of 15 letters or more in best-corrected visual acuity
Time frame: Baseline to 24 months
Cohort 2 (Expansion) - Percentage of participants with a loss of 15 letters or more in best-corrected visual acuity
Time frame: Baseline to 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- Individual participant dataSharing planned: No
Related trials for Macular Telangiectasia Type 2
RecruitingPhase 4
Phase 4 Study: Long-term Safety and Efficacy of NT-501 in MacTel Type 2, Including Sham Procedure Participants
Macular Telangiectasia Type 2 (MacTel)
Not yet recruitingPhase 3
Study of Serine Supplementation to Protect Vision in MacTel
Macular Telangiectasia Type 2 (MacTel)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Thomas Aaberg, Jr., MD study chair
Neurotech Pharmaceuticals
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing M-CHFM and CHFM in people with Macular Telangiectasia Type 2, run by Neurotech Pharmaceuticals at 6 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Patricia Davis (401-333-3880, p.davis@neurotechusa.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cohort 1 (Extension) - Rate of change in EZ area loss as measured by en face imaging by Spectral-Domain Optical Coherence Tomography (time frame: Baseline to 24 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2026.