Macular Telangiectasia Type 2 (MacTel)Phase 3NCT03316300
A Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2 - Protocol A
A Phase 3 study testing NT-501 in people with Macular Telangiectasia Type 2 (MacTel), run by Neurotech Pharmaceuticals at 20 sites in 4 countries.
Trial summary
This study is a phase 3, randomized, multi-center study to evaluate the efficacy and safety of the NT-501 implants in participants with macular telangiectasia type 2. Read more about this study
- Who is it for?
- Ages 21 to 80 years
- Conditions the study is for
- Macular Telangiectasia Type 2 (MacTel)
- What is being tested?
- NT-501
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 120 participants
- Where
- 20 sitesin 4 countries
- Study dates
- 24 Nov 2017to 23 Sep 2022
- Phase
- Phase 3
- 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
- Participant must have at least one study eye with 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
- Participant must have an Inner Segment - Outer Segment Junction Line (IS/OS) Photo Receptor (PR) break in the study eye(s) and en face EZ (area of IS/OS loss) as measured by spectral-domain optical coherence tomography (SD-OCT) between 0.16 mm^2 and 2.00 mm^2
- Participant's best corrected visual acuity (BCVA) is a 54-letter score or better (20/80 or better) as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart at screening.
- Participant must have steady fixation in the foveal or parafoveal area and sufficiently clear media for good quality photographs
- Participant must be greater than 21 years of age or less than 80 years of age at screening
- Participant 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
- Women of childbearing potential must agree to use highly effective contraception (Germany and France only)
Who cannot join
- Participant is medically unable to comply with study procedures or follow-up visits
- Participant received intravitreal steroid therapy for non-neovascular MacTel within the last 3 months
- Participant 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 in the fellow eye at randomization
- Participant 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 (eg, glaucoma, severe nonproliferative or proliferative diabetic retinopathy, uveitis)
- Participant has a chronic requirement (eg, ≥ 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)
- Participant has evidence of intraretinal neovascularization or subretinal neovascularization (SRNV), as evidenced by hemorrhage, hard exudate, subretinal fluid or intraretinal fluid in either eye
Show all 19Show fewer
- Participant has evidence of central serous chorio-retinopathy in either eye
- Participant has evidence of pathologic myopia in either eye
- Participant has significant corneal or media opacities in either eye
- Participant has had a vitrectomy, penetrating keratoplasty, trabeculectomy, or trabeculoplasty
- Participant 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
- Participant has undergone lens removal in the previous 3 months or YAG laser within 4 weeks
- Participant was a participant in any other clinical trial of an intervention (drug or device) within the last 6 months
- Participant is on chemotherapy
- Participant is pregnant or breastfeeding
- Participant has a history of malignancy that would compromise the 24-month study survival
- Participant with a history of ocular herpes virus in either eye
- Participant 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
- Participant has evidence of intraretinal hyperreflectivity by OCT
Trial locations and contacts
20 sites in 4 countries.
20 sites in 4 countries
United States16 sites
National Institute of Health, NIH
Bethesda, Maryland, 20892
Retina Associates of Cleveland, Inc.
Cleveland, Ohio, 44122
Retina Consultants of Southern Colorado, P.C.
Colorado Springs, Colorado, 80909
Texas Retina Associates
Dallas, Texas, 75231
Cumberland Valley Retina Consultants
Hagerstown, Maryland, 21740
Southeastern Retina Associates, PC
Knoxville, Tennessee, 37922
Scripps Clinic Medical Group
La Jolla, California, 92037
Jules Stein Eye Institute / David Geffen School of Medicine
Los Angeles, California, 90095
Retina Associates of New Orleans
Metairie, Louisiana, 70006
Byers Eye Institute at Stanford University
Palo Alto, California, 94303
Retina Northwest, PC
Portland, Oregon, 97221
Sierra Eye Associates
Reno, Nevada, 89502
Associated Retinal Consultants, P.C.
Royal Oak, Michigan, 48073
University of Utah John A. Moran Eye Center
Salt Lake City, Utah, 84132
Tulsa Retina Consultants
Tulsa, Oklahoma, 74114
Bay Area Retina Associates
Walnut Creek, California, 94598
United Kingdom2 sites
Moorfields Eye Hospital NHS Foundation Trust
London, EC1V 2PD
Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire, OX3 9DU
About this study
Official title: A Phase III Multicenter Randomized, Sham Controlled, Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2
This study is a phase 3, randomized, multi-center study to evaluate the efficacy and safety of the NT-501 implants in participants with macular telangiectasia type 2.
Read the detailed description
Phase 3, prospective, multicenter, masked, sham-controlled study with the overall study objective to evaluate the efficacy and safety of NT-501 for the treatment of MacTel. Secondary objective was to evaluate the safety of NT-501 in participants with MacTel. This was a multicenter study conducted at 20 study centers in the United States, Australia, France, and the United Kingdom.
- Conditions
- Macular Telangiectasia Type 2 (MacTel)
- Being tested
- NT-501, Sham Procedure
- Keywords
- Ciliary Neurotrophic Factor (CNTF), Macular Telangiectasia Type 2, MacTel
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NT-501Combination product
Each NT-501 implant consisted of hCNTF-secreting NTC-201-6A.02 cells encapsulated within supportive matrices and surrounded by a semipermeable polymer membrane.
The NTC-201-6A cells continuously secrete CNTF from the NT-501 implant into the vitreous cavity. Implanted by a qualified Health Care Professional.
Sham ProcedureProcedure or surgery
The sham surgery involved a superficial conjunctival incision performed under local anesthetic and closure with a single suture.
| Study group | Role | What they receive |
|---|---|---|
| NT-501 | Experimental | Test product Receives: Combination Product: NT-501 |
| Sham | Sham comparator | A sham surgical procedure was performed to mimic the implant procedure; there was no comparator product. Receives: Procedure: Sham Procedure |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Rate of Change in the Area of EZ Area of Loss From Baseline Through Month 24
The rate of change in the area of EZ loss (IS/OS; macular photoreceptor loss) from baseline through month 24, as assessed using SD-OCT in the study eye of subjects with MacTel.
Measured: End point timeframe is through Month 24. Baseline, Month 6, 12, 16, 20 and 24. Month 6 was collected but not included in the primary analyses.
Secondary outcomes (2)
Mean Change in Aggregate Retinal Sensitivity Loss and Aggregate Interpolated Retinal Sensitivity Loss by Microperimetry (mITT Population)
Change from baseline in retinal sensitivity loss as measured by as measured by Macular Integrity Assessment (MAIA)
Measured: Baseline through 24 months.
Monocular Reading Speed (mITT Population)
Change from baseline through Month 24 for Monocular reading speed assessed using International Reading Speed Texts (IReST) cards developed by the IReST Study Group 21
Measured: Baseline through 24 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2017
- Study start24 Nov 2017
- Primary completion31 Aug 2022
- Study completion23 Sep 2022
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Macular Telangiectasia Type 2 (MacTel)Studies 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
- After confirming eligibility in conjunction with the reading center, the study eye of each subject was randomized (1:1) to either have NT-501 implanted or to undergo the sham procedure.
- Masking
- Double blind
- Who is masked
- Participant, Outcomes assessor
- Enrolment
- 120 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurotech Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The Lowy Medical Research Institute Limited
- Responsible party
- Sponsor
- Investigators
- Paul S Bernstein, MD, Principal investigator, University of Utah - John A. Moran Eye Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03316300
- Sponsor's protocol ID
- NTMT-03-A
- Status last verified
- Aug 2024
- First submitted
- 3 Oct 2017
- Last updated
- 24 Sep 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Publications
Chew EY, Gillies M, Jaffe GJ, Gaudric A, Egan C, Constable I, Clemons T, Aaberg T, Manning DC, Hohman TC, Bird A, Friedlander M; MacTel CNTF NTMT-03 Research investigators. Cell-Based Ciliary Neurotrophic Factor Therapy for Macular Telangiectasia Type 2. NEJM Evid. 2025 Aug;4(8):EVIDoa2400481. doi: 10.1056/EVIDoa2400481. Epub 2025 Jul 22.
PubMed 40693847
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III Multicenter Randomized, Sham Controlled, Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2
- ClinicalTrials.gov ID
- NCT03316300
- Acronym / sponsor ID
- NTMT-03-A
- Conditions
- Macular Telangiectasia Type 2 (MacTel)
- Keywords
- Ciliary Neurotrophic Factor (CNTF), Macular Telangiectasia Type 2, MacTel
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 120 participants (actual)
- Lead sponsor
- Neurotech PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- The Lowy Medical Research Institute Limited
- Study start
- 24 Nov 2017 (actual)
- Primary completion
- 31 Aug 2022 (actual)
- Study completion
- 23 Sep 2022 (actual)
- First posted
- 20 Oct 2017 (actual)
- Results
- Posted 24 Sep 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is a phase 3, randomized, multi-center study to evaluate the efficacy and safety of the NT-501 implants in participants with macular telangiectasia type 2.
Detailed description
Phase 3, prospective, multicenter, masked, sham-controlled study with the overall study objective to evaluate the efficacy and safety of NT-501 for the treatment of MacTel. Secondary objective was to evaluate the safety of NT-501 in participants with MacTel. This was a multicenter study conducted at 20 study centers in the United States, Australia, France, and the United Kingdom.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, outcomes assessor)
- Number of arms
- 2
- Model description
- After confirming eligibility in conjunction with the reading center, the study eye of each subject was randomized (1:1) to either have NT-501 implanted or to undergo the sham procedure.
| Arm | Type | Receives |
|---|---|---|
| NT-501 | Experimental | Combination Product: NT-501Test product |
| Sham | Sham comparator | Procedure: Sham ProcedureA sham surgical procedure was performed to mimic the implant procedure; there was no comparator product. |
Who may qualify
- Ages
- 21 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Participant must have at least one study eye with 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
- Participant must have an Inner Segment - Outer Segment Junction Line (IS/OS) Photo Receptor (PR) break in the study eye(s) and en face EZ (area of IS/OS loss) as measured by spectral-domain optical coherence tomography (SD-OCT) between 0.16 mm^2 and 2.00 mm^2
- Participant's best corrected visual acuity (BCVA) is a 54-letter score or better (20/80 or better) as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart at screening.
- Participant must have steady fixation in the foveal or parafoveal area and sufficiently clear media for good quality photographs
- Participant must be greater than 21 years of age or less than 80 years of age at screening
Key exclusion criteria (19 listed)
- Participant is medically unable to comply with study procedures or follow-up visits
- Participant received intravitreal steroid therapy for non-neovascular MacTel within the last 3 months
- Participant 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 in the fellow eye at randomization
- Participant 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 (eg, glaucoma, severe nonproliferative or proliferative diabetic retinopathy, uveitis)
- Participant has a chronic requirement (eg, ≥ 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)
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 |
|---|---|---|
| NT-501Each NT-501 implant consisted of hCNTF-secreting NTC-201-6A.02 cells encapsulated within supportive matrices and surrounded by a semipermeable polymer membrane. The NTC-201-6A cells continuously secrete CNTF from the NT-501 implant into the vitreous cavity. Implanted by a qualified Health Care Professional. | Combination product | NT-501 |
| Sham ProcedureThe sham surgery involved a superficial conjunctival incision performed under local anesthetic and closure with a single suture. | Procedure or surgery | Sham |
Primary outcome
The Rate of Change in the Area of EZ Area of Loss From Baseline Through Month 24
Time frame: End point timeframe is through Month 24. Baseline, Month 6, 12, 16, 20 and 24. Month 6 was collected but not included in the primary analyses.
The rate of change in the area of EZ loss (IS/OS; macular photoreceptor loss) from baseline through month 24, as assessed using SD-OCT in the study eye of subjects with MacTel.
2 secondary outcomes
Mean Change in Aggregate Retinal Sensitivity Loss and Aggregate Interpolated Retinal Sensitivity Loss by Microperimetry (mITT Population)
Time frame: Baseline through 24 months.
Monocular Reading Speed (mITT Population)
Time frame: Baseline through 24 months.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40693847Chew EY, Gillies M, Jaffe GJ, Gaudric A, Egan C, Constable I, Clemons T, Aaberg T, Manning DC, Hohman TC, Bird A, Friedlander M; MacTel CNTF NTMT-03 Research investigators. Cell-Based Ciliary Neurotrophic Factor Therapy for Macular Telangiectasia Type 2. NEJM Evid. 2025 Aug;4(8):EVIDoa2400481. doi: 10.1056/EVIDoa2400481. Epub 2025 Jul 22.
- Individual participant dataSharing planned: No
Related trials for Macular Telangiectasia Type 2 (MacTel)
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Phase 4 Study: Long-term Safety and Efficacy of NT-501 in MacTel Type 2, Including Sham Procedure Participants
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Not yet recruitingPhase 3
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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.
Paul S Bernstein, MD principal investigator
University of Utah - John A. Moran Eye Center
20 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NT-501 in people with Macular Telangiectasia Type 2 (MacTel), run by Neurotech Pharmaceuticals at 20 sites in 4 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 19 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 24 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States, United Kingdom, Australia, France. 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 24 Sep 2024). 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?
The Rate of Change in the Area of EZ Area of Loss From Baseline Through Month 24 (time frame: End point timeframe is through Month 24. Baseline, Month 6, 12, 16, 20 and 24. Month 6 was collected but not included in the primary analyses.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03316300, which also lists 1 linked publication.
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 24 Sep 2024.