CompletedNCT03316300Updated Sep 24, 2024Results posted

A Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2 - Protocol A

A Phase 3 interventional study of NT-501 and Sham Procedure in Macular Telangiectasia Type 2 (MacTel), sponsored by Neurotech Pharmaceuticals. Completed at 20 sites in 4 countries. Open to participants aged 21 Years to 80 Years. Per ClinicalTrials.gov, last updated 2024-09-24.

Sponsored by Neurotech Pharmaceuticals · Phase 3, Interventional, and Treatment

Phase: Phase 3
Study type: Interventional
Enrollment: 120
Allocation: Randomized
Ages: 21 Years to 80 Years
Sex: All

Study 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 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 studied

Macular Telangiectasia Type 2 (MacTel)

Browse trials for

Telangiectasis 69

Keywords

Ciliary Neurotrophic Factor (CNTF)
Macular Telangiectasia Type 2
MacTel

In context

Telangiectasis

69 studies on the registry are indexed under Telangiectasis; 12 are open to participants now.

This study's enrollment of 120 is above the median of 30 across 59 interventional studies indexed under Telangiectasis.

Browse Telangiectasis studies →

Lead sponsor

Neurotech Pharmaceuticals is the lead sponsor of 13 studies on the registry; 2 are open to participants now.

Of its 8 completed or terminated interventional studies of FDA-regulated products, 7 (88%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 21 Years to 80 Years
Sexes eligible: All
Accepts healthy volunteers: No

Eligibility criteria

Key Inclusion Criteria:

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 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 must agree to use highly effective (Germany and France only)

Key Exclusion Criteria:

Participant is medically unable to comply with study procedures or follow-up visits
Participant received intravitreal 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
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 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

Terms in these criteria, in plain language(5)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

Study design

Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: Double (Participant, Outcomes assessor)
Enrollment: 120 participants (actual)

Study arms

Experimental
NT-501
Test product
Combination Product: NT-501
Sham comparator
Sham
A sham surgical procedure was performed to mimic the implant procedure; there was no comparator product.
Procedure: Sham Procedure

Interventions

Combination productNT-501
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.
ProcedureSham Procedure
The sham surgery involved a superficial conjunctival incision performed under local anesthetic and closure with a single suture.

What researchers measure

Primary outcomes

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.
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.

Secondary outcomes

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)
Time frame: 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
Time frame: Baseline through 24 months.

Results

Posted Sep 24, 2024

Participant flow

Screening period for up to 30 days

Participant flow — Overall Study
Milestone	NT-501	Sham
Started	61	59
Treated (underwent surgery)	58	57
Completed	53	52
Not completed	8	7
Withdrew: Withdrawal by subject	3	2
Withdrew: Eligible and did not enroll in amendment	4	4
Withdrew: Adverse event	1	0
Withdrew: Covid-19	0	1

Outcome measures

PrimaryThe 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.

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.
Reported as:: Mean · mm^2

The Rate of Change in the Area of EZ Area of Loss From Baseline Through Month 24
mm^2	NT-501	Sham
The Rate of Change in the Area of EZ Area of Loss From Baseline Through Month 24	0.075 ± 0.0123	0.166 ± 0.0125

Statistical analysis

NT-501 vs Sham · Mixed Models Analysis · p = <0.0001 · Mean difference (final values): -0.091 · 95% CI -0.13 to -0.06

SecondaryMean 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)

Time frame:: Baseline through 24 months.
Reported as:: Mean · dB

Mean Change in Aggregate Retinal Sensitivity Loss and Aggregate Interpolated Retinal Sensitivity Loss by Microperimetry (mITT Population)
dB	Aggregate Retinal Sensitivity Loss (dB) - NT-501	Aggregate Retinal Sensitivity Loss (dB) - Sham	Aggregate Interpolated Retinal Sensitivity Loss (dB) - NT-501	Aggregate Interpolated Retinal Sensitivity Loss (dB) - Sham
Baseline Actual	63.86 ± 80.642	58.30 ± 62.272	42588.33 ± 64255.621	33684.07 ± 40566.334
Month 24 Actual	86.50 ± 96.170	100.15 ± 85.991	60330.58 ± 81883.302	62598.85 ± 57942.882
Month 24 Change from Baseline	25.27 ± 34.238	43.02 ± 41.763	19244.29 ± 24911.285	29749.64 ± 27021.695

SecondaryMonocular 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

Time frame:: Baseline through 24 months.
Reported as:: Mean · Reading Speed (words per minute)

Monocular Reading Speed (mITT Population)
Reading Speed (words per minute)	NT-501	Sham
Baseline Actual	92.09 ± 43.717	96.01 ± 54.010
Month 24 Actual	85.70 ± 50.512	84.38 ± 42.917
Month 24 Change from Baseline	-6.18 ± 29.188	-12.20 ± 42.188

Adverse events

Collected over The Adverse events reported are those that occurred through month 24.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
NT-501	0/58 (0%)	12/58 (20.7%)	54/58 (93.1%)
Sham	1/57 (1.8%)	7/57 (12.3%)	47/57 (82.5%)

Most frequent serious events

Showing 10 of 22

Most frequent serious events
Event	NT-501	Sham
Suture Related ComplicationsInjury, poisoning and procedural complications	2/58	0/57
Soft Tissue InjuryInjury, poisoning and procedural complications	0/58	1/57
Cardiac failure congestiveCardiac disorders	0/58	1/57
Coronary Artery DiseaseCardiac disorders	0/58	1/57
Myocardial InfarcationCardiac disorders	0/58	1/57
OsteoarthritisMusculoskeletal and connective tissue disorders	0/58	1/57
AppendicitisInfections and infestations	0/58	1/57
SepsisInfections and infestations	0/58	1/57
Basal cell carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps)	1/58	0/57
Prostate CancerNeoplasms benign, malignant and unspecified (incl cysts and polyps)	1/58	0/57

Most frequent other events

Showing 10 of 197

Most frequent other events
Event	NT-501	Sham
Conjunctival haemmorhageEye disorders	18/58	12/57
Delayed dark adaptationEye disorders	12/58	1/57
MiosisEye disorders	10/58	0/57
Eye pruritusEye disorders	9/58	3/57
Dry eyeEye disorders	8/58	6/57
Eye painEye disorders	8/58	5/57
Ocular discomfortEye disorders	8/58	1/57
Foreign body sensation in eyesEye disorders	6/58	7/57
Vision blurredEye disorders	7/58	3/57
Vitreous floatersEye disorders	7/58	0/57

Baseline characteristics

The modified intent-to-treat population comprised all randomized subjects who underwent surgery (NT-501 implantation or sham surgery) whether or not the subject adhered to the study protocol.

Age, Categorical
Age, Categorical(Participants)	NT-501	Sham	Total
<=18 years	0	0	0
Between 18 and 65 years	37	37	74
>=65 years	21	20	41

Age, Continuous
Age, Continuous(years)	NT-501	Sham	Total
Mean	61.1 ± 8.01	60.2 ± 8.39	60.7 ± 8.17

Sex: Female, Male
Sex: Female, Male(Participants)	NT-501	Sham	Total
Female	39	40	79
Male	19	17	36

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	NT-501	Sham	Total
Hispanic or Latino	1	5	6
Not Hispanic or Latino	57	52	109
Unknown or Not Reported	0	0	0

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	NT-501	Sham	Total
American Indian or Alaska Native	0	1	1
Asian	2	3	5
Native Hawaiian or Other Pacific Islander	0	0	0
Black or African American	1	2	3
White	50	48	98
More than one race	0	0	0
Unknown or Not Reported	5	3	8

Baseline Ocular Characteristics (mITT) - Ocular Visual Acuity
Baseline Ocular Characteristics (mITT) - Ocular Visual Acuity(Best-corrected visual acuity (letters))	NT-501	Sham	Total
Mean	70.8 ± 9.11	73.3 ± 8.64	72.0 ± 8.93

Baseline Ocular Characteristics (mITT) - Area of EZ loss (mm^2)
Baseline Ocular Characteristics (mITT) - Area of EZ loss (mm^2)(Area of EZ loss (mm^2))	NT-501	Sham	Total
Mean	0.513 ± 0.4773	0.487 ± 0.3576	0.501 ± 0.4206

Baseline Ocular Characteristics (mITT) - Monocular Reading Speed
Baseline Ocular Characteristics (mITT) - Monocular Reading Speed(words per minute)	NT-501	Sham	Total
Mean	92.09 ± 43.717	96.01 ± 54.010	94.03 ± 48.907

3 further baseline measures are reported on the registry.

Study locations

20 sites

Scripps Clinic Medical Group
La Jolla, California 92037, United States
Jules Stein Eye Institute / David Geffen School of Medicine
Los Angeles, California 90095, United States
Byers Eye Institute at Stanford University
Palo Alto, California 94303, United States
Bay Area Retina Associates
Walnut Creek, California 94598, United States
Retina Consultants of Southern Colorado, P.C.
Colorado Springs, Colorado 80909, United States
Retina Associates of New Orleans
Metairie, Louisiana 70006, United States
National Institute of Health, NIH
Bethesda, Maryland 20892, United States
Cumberland Valley Retina Consultants
Hagerstown, Maryland 21740, United States
Associated Retinal Consultants, P.C.
Royal Oak, Michigan 48073, United States
Sierra Eye Associates
Reno, Nevada 89502, United States
Retina Associates of Cleveland, Inc.
Cleveland, Ohio 44122, United States
Tulsa Retina Consultants
Tulsa, Oklahoma 74114, United States
Retina Northwest, PC
Portland, Oregon 97221, United States
Southeastern Retina Associates, PC
Knoxville, Tennessee 37922, United States
Texas Retina Associates
Dallas, Texas 75231, United States
University of Utah John A. Moran Eye Center
Salt Lake City, Utah 84132, United States
Lions Eye Institute
Perth, Western Australia 6009, Australia
Lariboisiere Hospital
Paris, 75010, France
Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire OX3 9DU, United Kingdom
Moorfields Eye Hospital NHS Foundation Trust
London, EC1V 2PD, United Kingdom

References and documents

Study documents

Study protocol ↗ · Aug 31, 2021
Statistical analysis plan ↗ · May 20, 2021

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

Timeline

Tracking since Sep 25, 2026

ElapsedEstimatedEstimated, not yet reached

Ran from Nov 2017 to primary completion in Aug 2022, 57 months.

Results were first posted in Sep 2024, 2 years after primary completion.

How these figures are worked out, and which studies are left out of them.

Registered: Oct 3, 2017
First posted: Oct 20, 2017
StartActual: Nov 24, 2017
Primary completionActual: Aug 31, 2022
CompletionActual: Sep 23, 2022
Results first posted: Sep 24, 2024
Last update: Sep 24, 2024

What these dates mean

Start.: When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.: When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.: When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.: Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.

No changes since tracking began. The registry record was last updated on Sep 24, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT03316300
Lead sponsor: Neurotech Pharmaceuticals
Collaborators: The Lowy Medical Research Institute Limited
Responsible party: Sponsor
First posted: Oct 20, 2017
Start date: Nov 24, 2017
Primary completion: Aug 31, 2022
Completion: Sep 23, 2022
Results posted: Sep 24, 2024
Last update: Sep 24, 2024

Study contacts

Paul S Bernstein, MD

principal investigator · University of Utah - John A. Moran Eye Center

Oversight

Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: Yes

View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Aug 2024. You cannot join it, but the record below documents what was studied.

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