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CompletedNCT04729972Updated Mar 26, 2025Results posted

Safety Study to Evaluate Bilateral CNTF Implants in Subjects With MacTel Type 2

A Phase 2 interventional study of NT-501 CNTF implant in Macular Telangiectasia Type 2, sponsored by Neurotech Pharmaceuticals. Completed at 10 sites in 2 countries. Open to participants aged 18 Years to 85 Years. Per ClinicalTrials.gov, last updated 2025-03-26.

Sponsored by Neurotech Pharmaceuticals · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
33
Allocation
Not applicable
Ages
18 Years to 85 Years
Sex
All
01

Study summary

This is a multi-center, open-label, 6-month study to evaluate the safety of bilateral CNTF implants in participants with Macular Telangiectasia Type 2.

Read the detailed description

This was a multicenter, open-label study designed to evaluate the safety of bilateral NT-501 in subjects with MacTel. All subjects who received NT-501 in a single eye prior to or in the Phase 1/2 extension study (NTMT-01/02E) or in 1 of the 2 Phase 3 studies (NTMT-03-A or NTMT-03-B), and met all other eligibility criteria, qualified for participation in the current study; enrolled subjects underwent intraocular implantation of NT-501 in the fellow eye and were followed for 6 months. Note that, throughout this report, all references to "study eye" mean the eye that received NT-501 in the current study (ie, not the eye that was treated in the previous study).

02

Conditions studied

  • Macular Telangiectasia Type 2

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Keywords

  • Macular Telangiectasia Type 2
  • CNTF
  • Mactel
03

Who can participate

Ages eligible
18 Years to 85 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

General Inclusion Criteria

  1. Participants from the NTMT-03 study must have completed the Month-24 visit
  2. Participant in the MacTel Phase 1/2 extension (NTMT-01/02E) study or the Phase 3 study must exit these studies prior to entering the Bilateral Implant safety study (NTMT-02B)
  3. Participant must be willing and able to follow the study instructions and be willing and able to complete all required study procedures and visits
  4. Participant must be willing and able to provide a signed Informed Consent, as well as written documentation in accordance with the relevant country and local privacy requirements, e.g., written data protection consent
  5. Females of childbearing potential must consent to a pregnancy test before entering the study
  6. A participant's refusal to allow the collection of blood samples for analysis of serum CNTF, serum Ab or Nab to CNTF, Ab to NTC-201.6A cells and Ab to DFHR levels in one eye will not exclude the participant from study participation

Ocular Inclusion Criteria

  1. Participant must have a positive diagnosis of MacTel type 2 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 in the study eye
  2. Participant must have steady fixation in the foveal or parafoveal area in the study eye and sufficiently clear media for good quality photographs

General Exclusion Criteria

  1. Females of childbearing potential (those who are not surgically sterilized or post- menopausal, i.e., absence of menstruation for 12 months or longer) may not participate in the study if any of the following conditions exists:

    • Pregnant (positive pregnancy test at Visit 1 or intend to become pregnant during the study)
    • Nursing (lactating)
    • Do not agree to use adequate birth control methods for the duration of the study and until 90 days after the last administration of study drug (adequate birth control methods are: hormonal - oral, implantable, transdermal or injectable contraceptives; mechanical - spermicide in conjunction with a barrier such as condom or diaphragm, intrauterine device [IUD] or surgical sterilization of partner, or total sexual abstinence)
  2. Participant is too ill to likely complete the entire study, based on the investigator's assessment
  3. Participant, in the opinion of the investigator, is not suitable for study participation
  4. Participant with any screening laboratory finding (serum chemistry, hematology, urinalysis) that in the opinion of the investigator is not suitable for study participation
  5. Participant has a history or current evidence of severe hypersensitivity to the NT-501 implant
  6. Participant has a history or current evidence of a medical condition (systemic or ophthalmic disease, metabolic dysfunction, physical examination finding or clinical laboratory finding) that may in the opinion of the investigator preclude the safe administration of the NT-501 implant or adherence to the scheduled study visits, safe participation in the study or affect the results of the study (e.g., unstable or progressive cardiovascular, cerebral vascular, pulmonary, Parkinson's, liver or renal disease; depression, cancer, or dementia

Ocular Exclusion Criteria

  1. Participant has a history or evidence of the following surgeries/procedures in the study eye, as assessed at Visit 1, including:

    • Submacular surgery
    • Vitrectomy
    • Retinal detachment
    • Incisional glaucoma surgery
    • Trabeculectomy or trabeculoplasty
    • Cataract surgery or laser-assisted in situ keratomileusis (LASIK) performed in the previous 6 months
  2. Participant has uncontrolled glaucoma; or ocular hypertension, i.e., IOP ≥ 25 mmHg in the study eye
  3. Participant has evidence of intraretinal or subretinal neovascularization or central serous chorioretinopathy in the study eye
  4. Participant has evidence of ocular disorder(s) in the study eye of a severity that could confound the interpretation of study results, compromise visual acuity or require medical or surgical intervention during the study period, including retinal vascular occlusion, severe nonproliferative or proliferative diabetic retinopathy, retinal detachment, macular hole, geographic atrophy, intraretinal or subretinal neovascularization, central serous chorio-retinopathy, pathological myopia
  5. Participant has a vitreous hemorrhage in the study eye at Visit 1 (Screening)
  6. Participant has a spherical equivalent of the refractive error in the study eye demonstrating more than 8 diopters of myopia
  7. Participant has a history or evidence of penetrating ocular trauma in the study eye
  8. Participant has an anticipated need for cataract extraction in the study eye within 6 months of Visit 1 (Screening) such as 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
  9. Participant has uveitis, history of uveitis in either eye or history of ocular herpes virus in either eye
  10. Participant has undergone major surgery within the last 6 months (systemic or ocular in either eye) or who are likely to require major surgery within 6 months of Visit 1 (Screening)
  11. Participant has periocular or ocular/intraocular infection or inflammation in either eye (such as infectious conjunctivitis, keratitis, scleritis, endophthalmitis) within 3 months prior to Visit 1 (Screening)
  12. Participant has ocular hypotension in either eye (\<6 mmHg) that in the opinion of the Investigator would interfere with the NT-501 implant insertion
  13. Participant has a history of scleritis, scleral thinning, periocular, ocular, or intraocular infection or inflammation, cicatrizing conjunctival diseases any other ocular conditions in the study eye that could interfere with or complicate the surgery associated with NT-501implant insertion
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
33 participants (actual)

Study arms

  • Experimental
    NT-501 in Fellow Eye

    NT-501 was implanted on Day 0 in the fellow eye of subjects who received a single NT-501 in a previous study; subjects were followed for 6 months post-implantation of NT-501 in this study.

    Combination Product: NT-501 CNTF implant

Interventions

  • Combination productNT-501 CNTF implant

    Single implantation of CNTF-secreting NT-501 device into fellow (untreated) eye

05

What researchers measure

Primary outcomes

  1. Frequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events

    Assess the incidence and severity of adverse events (AEs) following bilateral ocular implantation of NT-501

    Time frame: 6 months

Secondary outcomes

  1. Serum Levels of CNTF and Immunogenicity for NT-501

    Incidence of positive serum levels of CNTF and immunogenicity for NT-501 with quantification of antibodies (Ab) to CNTF, neutralizing antibodies (NAb) to CNTF, Ab to NTC-201-6A cells, and Ab to mouse dihydrofolate reductase (mDHFR) at any time.

    Time frame: 6 months

06

Results

Posted Mar 26, 2025

Participant flow

Of the 33 enrolled subjects, 32 (97.0%) received NT-501; the exception was a subject whose surgery was canceled due to an event of arterial fibrillation that occurred prior to surgery and resulted in subject discontinuation. No subject had NT-501 removed from either eye during the study, and all 32 subjects who received NT-501 in this study completed the study.

Participant flow — Overall Study
MilestoneNT-501 in Fellow Eye
Started33
Nt-501 implanted in study eye32
Completed32
Not completed1
Withdrew: Adverse event1

Outcome measures

PrimaryFrequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events

Assess the incidence and severity of adverse events (AEs) following bilateral ocular implantation of NT-501

Time frame:
6 months
Reported as:
Count of participants · Participants
Frequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events
ParticipantsNT-501 in Fellow Eye
At least 1 TEAE26
At least 1 ocular TEAE in study eye26
At least 1 ocular TEAE in fellow eye6
At least 1 nonocular TEAE13
At least 1 treatment related ocular TEAE26
At least 1 treatment related nonocular TEAE0
At least 1 SAE2
NT-501 removal from either eye0
SecondarySerum Levels of CNTF and Immunogenicity for NT-501

Incidence of positive serum levels of CNTF and immunogenicity for NT-501 with quantification of antibodies (Ab) to CNTF, neutralizing antibodies (NAb) to CNTF, Ab to NTC-201-6A cells, and Ab to mouse dihydrofolate reductase (mDHFR) at any time.

Time frame:
6 months
Reported as:
Count of participants · Participants
Serum Levels of CNTF and Immunogenicity for NT-501
ParticipantsNT-501 in Fellow Eye
CNTF Positive0
Ab Positive to CNTF1
Neutralizing Ab to CNTF0
Ab to NTC0
ab to DHRF0

Adverse events

Collected over Through 6 months post implant. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
NT-501 in Fellow Eye0/32 (0%)2/32 (6.3%)26/32 (81.3%)
Most frequent serious events
Most frequent serious events
EventNT-501 in Fellow Eye
Device ExtrusionSurgical and medical procedures1/32
Feces discoloredGastrointestinal disorders1/32
Most frequent other events
Showing 10 of 32
Most frequent other events
EventNT-501 in Fellow Eye
Eye painEye disorders9/32
Ocular discomfortEye disorders9/32
Conjunctival haemorrhageEye disorders8/32
Eye irritationEye disorders4/32
Dry eyeEye disorders2/32
Anterior chamber cellEye disorders1/32
Anterior chamber flareEye disorders1/32
CataractEye disorders1/32
Cataract subcapsularEye disorders1/32
Conjunctival hyperaemiaEye disorders1/32

Baseline characteristics

Baseline demographics and characteristics are based on total number of participants who completed NT-501 implant in study eye

Age, Customized
Age, Customized(years)NT-501 in Fellow Eye
Age (Years)63.0 ± 6.55
Age, Customized
Age, Customized(Participants)NT-501 in Fellow Eye
50 to < 60 years9
60 to < 70 years17
> or = to 70 years6
Sex: Female, Male
Sex: Female, Male(Participants)NT-501 in Fellow Eye
Female24
Male8
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)NT-501 in Fellow Eye
Hispanic or Latino2
Not Hispanic or Latino30
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)NT-501 in Fellow Eye
American Indian or Alaska Native0
Asian2
Native Hawaiian or Other Pacific Islander0
Black or African American1
White28
More than one race0
Unknown or Not Reported1
BCVA (letters) for Study Eye
BCVA (letters) for Study Eye(letters correctly read)NT-501 in Fellow Eye
Study Eye68.7 ± 11.01
Fellow Eye70.1 ± 9.58
07

Study locations

10 sites
  • Jules Stein Eye Institute
    Los Angeles, California 90095, United States
  • National Eye Institute
    Washington, District of Columbia 20892, United States
  • Bascom Palmer
    Miami, Florida 33136, United States
  • Massachusetts Eye and Ear Infirmary, Retina Service
    Boston, Massachusetts 02114, United States
  • University of Michigan, Kellogg Eye Center
    Ann Arbor, Michigan 48105, United States
  • Retina Associates of Cleveland, Inc.
    Beachwood, Ohio 44122, United States
  • Retina Consultants of Texas
    Houston, Texas 77030, United States
  • Save Sight Institute
    Sydney, New South Wales, Australia
  • Centre for Eye Research Australia
    East Melbourne, Victoria 3002, Australia
  • Lions Eye Institute
    Nedlands, Western Australia 6009, Australia
08

References and documents

Publications

  • Chew EY, Clemons TE, Jaffe GJ, Johnson CA, Farsiu S, Lad EM, Guymer R, Rosenfeld P, Hubschman JP, Constable I, Wiley H, Singerman LJ, Gillies M, Comer G, Blodi B, Eliott D, Yan J, Bird A, Friedlander M; Macular Telangiectasia Type 2-Phase 2 CNTF Research Group. Effect of Ciliary Neurotrophic Factor on Retinal Neurodegeneration in Patients with Macular Telangiectasia Type 2: A Randomized Clinical Trial. Ophthalmology. 2019 Apr;126(4):540-549. doi: 10.1016/j.ophtha.2018.09.041. Epub 2018 Oct 4. PubMed 30292541 ↗

Study documents

  • Study protocol · Jan 21, 2022
  • Statistical analysis plan · Jul 14, 2022

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

09

Registry details

Key details

Study ID
NCT04729972
Lead sponsor
Neurotech Pharmaceuticals
Collaborators
The Lowy Medical Research Institute Limited
Responsible party
Sponsor
First posted
Jan 29, 2021
Start date
Jul 21, 2021
Primary completion
Dec 8, 2022
Completion
Dec 8, 2022
Results posted
Mar 26, 2025
Last update
Mar 26, 2025

Study contacts

Thomas Aaberg, Jr, MD
study director · Neurotech Pharmaceuticals, LLC

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
Yes
View the source record on ClinicalTrials.gov ↗

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