MacTel (Macular Telangiectasia) Type 2Phase 2NCT03071965
Extension Study of NT-501 Ciliary Neurotrophic Factor (CNTF) Implant for Macular Telangiectasia (MacTel)
A Phase 2 study testing Ciliary neurotrophic factor (CNTF) and Surgery in people with MacTel (Macular Telangiectasia) Type 2, run by Neurotech Pharmaceuticals at 11 sites in 2 countries.
Trial summary
This was a prospective, multicenter, single-masked, sham-controlled extension study designed to provide long-term safety and efficacy follow-up data for subjects with MacTel who had NT-501 implanted intraocularly and/or underwent sham surgery in the respective precursor study (NTMT-01 or NTMT-02). Read more about this study
- Who is it for?
- Ages 21 to 80 years
- Conditions the study is for
- MacTel (Macular Telangiectasia) Type 2
- What is being tested?
- Ciliary neurotrophic factor (CNTF) and Surgery
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 11 sitesin 2 countries
- Study dates
- 12 May 2017to 11 May 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Previously enrolled in the NTMT-01 or NTMT-02 protocol and received the NT-501 implant and/or underwent a Sham procedure
- Participant must be offered sufficient opportunity to review and to understand the informed consent form, agree to the form's contents, and provide written informed consent.
Who cannot join
- There are no Exclusion Criteria
Trial locations and contacts
11 sites in 2 countries.
11 sites in 2 countries
United States8 sites
University of Michigan, Kellogg Eye Center
Ann Arbor, Michigan, 48105
Emory University School of Medicine, Dept of Ophthalmology, Emory University Eye Center
Atlanta, Georgia, 30322
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114
Retina Associates of Cleveland, Inc
Cleveland, Ohio, 44122
Stein Eye Institute / David Geffen School of Medicine
Los Angeles, California, 90095
University of Wisconsin-Madison, Department of Ophthalmology and Visual Sciences
Madison, Wisconsin, 53705
University of Miami-Miller School of Medicine, Bascom Palmer Eye Institute
Miami, Florida, 33136
NIH Clinical Center
Rockville, Maryland, 20892
Australia3 sites
Centre for Eye Research Australia
East Melbourne, Victoria, 3002
Lions Eye Institute
Nedlands, Western Australia, 6009
Sydney Eye Hospital
Sydney, New South Wales, 2000
About this study
This was a prospective, multicenter, single-masked, sham-controlled extension study designed to provide long-term safety and efficacy follow-up data for subjects with MacTel who had NT-501 implanted intraocularly and/or underwent sham surgery in the respective precursor study (NTMT-01 or NTMT-02).
A substudy was conducted in which subjects enrolled in study (NTMT-02), who had 1 study-eligible eye that underwent sham surgery, were offered the option to have NT-501 implanted in the same study eye. Of the 19 subjects who had 1 study-eligible eye and underwent sham surgery in the Cohort 2 precursor study (NTMT-02), 16 subjects elected to have NT-501 implanted in the same study eye during the substudy
- Conditions
- MacTel (Macular Telangiectasia) Type 2
- Being tested
- Ciliary neurotrophic factor (CNTF), Surgery
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ciliary neurotrophic factor (CNTF)Biological
The investigational product is the NT-501 encapsulated cell system, which consists of cells encapsulated within a semi-permeable polymer membrane and supportive matrices. NT-501 contains NTC-201 cells that secrete recombinant human CNTF, which were derived from genetically modified NTC-200 cells.
SurgeryProcedure or surgery
Surgery to implant device for NT-501
SurgeryProcedure or surgery
Sham surgery
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Participants completed protocol NTMT-01. All participants received surgery to implant NT-501. All participants received ciliary neurotrophic factor (CNTF). Receives: Biological: Ciliary neurotrophic factor (CNTF); Procedure: Surgery |
| Cohort 2 | Experimental | Participants completed protocol NTMT-02. Participants for cohort 2 arm were either:
* NT-501 group: included subjects who each had 1 study-eligible eye that had NT-501 implanted and a fellow eye that did not undergo any study intervention.
* Sham group: included subjects who each had 1 study-eligible eye that underwent sham surgery and a fellow eye that did not undergo any study intervention.
* Sham+NT-501 group: included subjects who had both eyes that were study-eligible, and had NT-501 implanted in 1 eye and underwent sham surgery in the contralateral eye. Receives: Biological: Ciliary neurotrophic factor (CNTF); Procedure: Surgery |
What is being measured
The primary outcome is the main question the study is designed to answer.
Ellipsoid Zone (Area of IS/OS Loss) - Cohort 1
Change from baseline to 72, 84, 96 and 108 months post-surgery (based on participants' original implant date during precursor study) by SD-OCT for Cohort 1
Measured: 72, 84, 96, and 108 months (based on participants' original implant date during precursor study)
Ellipsoid Zone (Area of IS/OS Loss) - Cohort 2
Change in EZ from baseline to 36, 48, 60 and 72 Months post-surgery (based on participants' original implant date during precursor study). Cohort 2 sham surgery arm includes 16 participants (16 eyes) that received an NT-501 following their Month 36 follow-up
Measured: Baseline to 36, 48, 60 and 72 Months post-surgery (based on participants' original implant date during precursor study)
Secondary outcomes (4)
Change in Retinal Sensitivity (dB) From Baseline - Cohort 2 Only
Baseline to 72 Months - analyses also presented for additional times: 36, 48, and 60 Months post-surgery (based on participants' original implant date during precursor study) . Cohort 2 sham surgery arm includes 16 participants (16 eyes) that received an NT-501 following their Month 36 follow-up in this study.
Measured: Baseline to 72 Months (based on participants' original implant date)
Change in BCVA From Baseline for Cohort 1
Baseline to 108 Months - analyses also presented for additional times: 84, 96 and 108 Months (based on participants' original implant date)
Measured: Baseline to 108 Months (based on participants' original implant date)
Change in BCVA From Baseline for Cohort 2
Baseline to 72 Months - analyses also presented for additional times: 36, 48, and 60 Months (based on participants' original implant date during precursor study) . Cohort 2 sham surgery arm includes 16 participants (16 eyes) that received an NT-501 following their Month 36 follow-up
Measured: Baseline to 72 Months (based on participants' original implant date)
Change in Reading Speed From Baseline - Cohort 2 Only
Baseline to 72 Months - analyses also presented for additional times: 36, 48, and 60 Months (based on participants' original implant date during precursor study) . Cohort 2 sham surgery arm includes 16 participants (16 eyes) that received an NT-501 following their Month 36 follow-up. Note: there were
Measured: Baseline to 72 Months (based on participants' original implant date)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2017
- Study start12 May 2017
- Primary completion11 May 2021
- Study completion11 May 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for MacTel (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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 70 participants (actual)
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, LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03071965
- Sponsor's protocol ID
- NTMT-01/02E
- Status last verified
- May 2025
- First submitted
- 1 Mar 2017
- Last updated
- 9 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Extension Study of NT-501 Ciliary Neurotrophic Factor (CNTF) Implant for Macular Telangiectasia (MacTel)
- ClinicalTrials.gov ID
- NCT03071965
- Acronym / sponsor ID
- NTMT-01/02E
- Conditions
- MacTel (Macular Telangiectasia) Type 2
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- Neurotech PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 12 May 2017 (actual)
- Primary completion
- 11 May 2021 (actual)
- Study completion
- 11 May 2021 (actual)
- First posted
- 7 Mar 2017 (actual)
- Results
- Posted 9 May 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was a prospective, multicenter, single-masked, sham-controlled extension study designed to provide long-term safety and efficacy follow-up data for subjects with MacTel who had NT-501 implanted intraocularly and/or underwent sham surgery in the respective precursor study (NTMT-01 or NTMT-02).
A substudy was conducted in which subjects enrolled in study (NTMT-02), who had 1 study-eligible eye that underwent sham surgery, were offered the option to have NT-501 implanted in the same study eye. Of the 19 subjects who had 1 study-eligible eye and underwent sham surgery in the Cohort 2 precursor study (NTMT-02), 16 subjects elected to have NT-501 implanted in the same study eye during the substudy
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: Ciliary neurotrophic factor (CNTF), Procedure: SurgeryParticipants completed protocol NTMT-01. All participants received surgery to implant NT-501. All participants received ciliary neurotrophic factor (CNTF). |
| Cohort 2 | Experimental | Biological: Ciliary neurotrophic factor (CNTF), Procedure: SurgeryParticipants completed protocol NTMT-02. Participants for cohort 2 arm were either: * NT-501 group: included subjects who each had 1 study-eligible eye that had NT-501 implanted and a fellow eye that did not undergo any study intervention. * Sham group: included subjects who each had 1 study-eligible eye that underwent sham surgery and a fellow eye that did not undergo any study intervention. * Sham+NT-501 group: included subjects who had both eyes that were study-eligible, and had NT-501 implanted in 1 eye and underwent sham surgery in the contralateral eye. |
Who may qualify
- Ages
- 21 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Previously enrolled in the NTMT-01 or NTMT-02 protocol and received the NT-501 implant and/or underwent a Sham procedure
- Participant must be offered sufficient opportunity to review and to understand the informed consent form, agree to the form's contents, and provide written informed consent.
Key exclusion criteria (1 listed)
- There are no Exclusion Criteria
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 |
|---|---|---|
| Ciliary neurotrophic factor (CNTF)The investigational product is the NT-501 encapsulated cell system, which consists of cells encapsulated within a semi-permeable polymer membrane and supportive matrices. NT-501 contains NTC-201 cells that secrete recombinant human CNTF, which were derived from genetically modified NTC-200 cells. | Biological | Cohort 1, Cohort 2 |
| SurgerySurgery to implant device for NT-501 | Procedure or surgery | Cohort 1, Cohort 2 |
| SurgerySham surgery | Procedure or surgery | Cohort 2 |
Primary outcome
Ellipsoid Zone (Area of IS/OS Loss) - Cohort 1
Time frame: 72, 84, 96, and 108 months (based on participants' original implant date during precursor study)
Change from baseline to 72, 84, 96 and 108 months post-surgery (based on participants' original implant date during precursor study) by SD-OCT for Cohort 1
Ellipsoid Zone (Area of IS/OS Loss) - Cohort 2
Time frame: Baseline to 36, 48, 60 and 72 Months post-surgery (based on participants' original implant date during precursor study)
Change in EZ from baseline to 36, 48, 60 and 72 Months post-surgery (based on participants' original implant date during precursor study). Cohort 2 sham surgery arm includes 16 participants (16 eyes) that received an NT-501 following their Month 36 follow-up
4 secondary outcomes
Change in Retinal Sensitivity (dB) From Baseline - Cohort 2 Only
Time frame: Baseline to 72 Months (based on participants' original implant date)
Change in BCVA From Baseline for Cohort 1
Time frame: Baseline to 108 Months (based on participants' original implant date)
Change in BCVA From Baseline for Cohort 2
Time frame: Baseline to 72 Months (based on participants' original implant date)
Change in Reading Speed From Baseline - Cohort 2 Only
Time frame: Baseline to 72 Months (based on participants' original implant date)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for MacTel (Macular Telangiectasia) Type 2
RecruitingPhase 3
A Study to Determine the Safety and Efficacy of NT-501 With M-CHFM
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. This study is not recruiting at the moment.
Thomas Aaberg, Jr, MD study chair
Neurotech Pharmaceuticals, LLC
11 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ciliary neurotrophic factor (CNTF) and Surgery in people with MacTel (Macular Telangiectasia) Type 2, run by Neurotech Pharmaceuticals at 11 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 2 inclusion and 1 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: finished, as of the sponsor's last update on 9 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Ellipsoid Zone (Area of IS/OS Loss) - Cohort 1 (time frame: 72, 84, 96, and 108 months (based on participants' original implant date during precursor study)). 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 9 May 2025.