A Phase 3 interventional study of NT-501 and Sham in Macular Telangiectasia Type 2, sponsored by Neurotech Pharmaceuticals. Completed at 27 sites in 3 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
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.
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, Germany, and the United Kingdom.
69 studies on the registry are indexed under Telangiectasis; 12 are open to participants now.
This study's enrollment of 119 is above the median of 30 across 59 interventional studies indexed under Telangiectasis.
Browse Telangiectasis studies →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.
Key Inclusion Criteria:
Key Exclusion Criteria:
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.
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.
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.
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.
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.
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.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Test product
Combination Product: NT-501
A sham surgical procedure was performed to mimic the implant procedure; there was no comparator product.
Procedure: Sham
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.
The sham surgery involved a superficial conjunctival incision performed under local anesthetic and closure with a single suture.
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 participants 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.
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.
Screening period for up to 30 days
| Milestone | NT-501 | Sham |
|---|---|---|
| Started | 60 | 59 |
| Treated (underwent surgery) | 59 | 54 |
| Completed | 57 | 49 |
| Not completed | 3 | 10 |
| Withdrew: Adverse event | 1 | 0 |
| Withdrew: Physician decision | 0 | 1 |
| Withdrew: Withdrawal by subject | 0 | 1 |
| Withdrew: Covid-19 | 0 | 1 |
| Withdrew: Other | 1 | 2 |
| Withdrew: Eligible and did not enroll in amendment | 1 | 3 |
| Withdrew: Lost to follow-up | 0 | 2 |
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 participants with MacTel.
| mm^2 | NT-501 | Sham |
|---|---|---|
| The Rate of Change in the Area of EZ Area of Loss From Baseline Through Month 24 | 0.111 ± 0.0142 | 0.160 ± 0.0149 |
Change from baseline in retinal sensitivity loss as measured by as measured by Macular Integrity Assessment (MAIA)
| 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 | 55.54 ± 56.050 | 51.84 ± 57.380 | 34864.17 ± 35305.260 | 32445.03 ± 38667.790 |
| Month 24 Actual | 97.77 ± 78.620 | 93.02 ± 70.414 | 61076.49 ± 52619.379 | 58360.26 ± 50535.070 |
| Month 24 Change from Baseline | 40.02 ± 51.278 | 41.97 ± 41.111 | 24859.88 ± 31165.862 | 26309.32 ± 25374.040 |
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
| Reading Speed (words per minute) | NT-501 | Sham |
|---|---|---|
| Baseline Actual | 96.49 ± 47.314 | 94.09 ± 42.806 |
| Month 24 Actual | 92.18 ± 45.090 | 77.33 ± 43.899 |
| Month 24 Change from Baseline | -5.46 ± 29.648 | -18.88 ± 33.705 |
Collected over During the Clinical Trial. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| NT-501 | 1/59 (1.7%) | 11/59 (18.6%) | 56/59 (94.9%) |
| Sham | 0/54 (0%) | 10/54 (18.5%) | 48/54 (88.9%) |
| Event | NT-501 | Sham |
|---|---|---|
| Suture related complicationInjury, poisoning and procedural complications | 3/59 | 0/54 |
| Transient ischaemic attackNervous system disorders | 2/59 | 0/54 |
| Chronic obstructive pulmonary diseaseRespiratory, thoracic and mediastinal disorders | 1/59 | 1/54 |
| CellulitisInfections and infestations | 1/59 | 1/54 |
| Breast cancerNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/59 | 1/54 |
| Colon cancerNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/59 | 1/54 |
| Invasive ductal breast carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/59 | 1/54 |
| Tendon ruptureInjury, poisoning and procedural complications | 0/59 | 1/54 |
| Atrial fibrillationCardiac disorders | 0/59 | 1/54 |
| PalpitationsCardiac disorders | 0/59 | 1/54 |
| Event | NT-501 | Sham |
|---|---|---|
| Conjunctival haemorrhageEye disorders | 19/59 | 10/54 |
| Delayed dark adaptationEye disorders | 15/59 | 2/54 |
| Suture related complicationInjury, poisoning and procedural complications | 14/59 | 2/54 |
| Foreign body sensation in eyesEye disorders | 14/59 | 8/54 |
| Conjunctival hyperaemiaEye disorders | 12/59 | 7/54 |
| Eye painEye disorders | 12/59 | 6/54 |
| MiosisEye disorders | 8/59 | 0/54 |
| HeadacheNervous system disorders | 7/59 | 6/54 |
| Conjunctival oedemaEye disorders | 4/59 | 6/54 |
| Vision blurredEye disorders | 6/59 | 3/54 |
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(Participants) | NT-501 | Sham | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 42 | 36 | 78 |
| >=65 years | 17 | 18 | 35 |
| Age, Continuous(years) | NT-501 | Sham | Total |
|---|---|---|---|
| Mean | 58.5 ± 7.61 | 58.7 ± 8.87 | 58.6 ± 8.20 |
| Sex: Female, Male(Participants) | NT-501 | Sham | Total |
|---|---|---|---|
| Female | 46 | 36 | 82 |
| Male | 13 | 18 | 31 |
| Ethnicity (NIH/OMB)(Participants) | NT-501 | Sham | Total |
|---|---|---|---|
| Hispanic or Latino | 4 | 4 | 8 |
| Not Hispanic or Latino | 55 | 49 | 104 |
| Unknown or Not Reported | 0 | 1 | 1 |
| Race (NIH/OMB)(Participants) | NT-501 | Sham | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 3 | 1 | 4 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 55 | 47 | 102 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 1 | 6 | 7 |
| Baseline Ocular Characteristics (mITT) - Ocular Visual Acuity(Best-corrected visual acuity (letters)) | NT-501 | Sham | Total |
|---|---|---|---|
| Mean | 74.4 ± 7.76 | 73.6 ± 9.23 | 74.0 ± 8.47 |
| Baseline Ocular Characteristics (mITT) - Area of EZ loss (mm^2)(Area of EZ loss (mm^2)) | NT-501 | Sham | Total |
|---|---|---|---|
| Mean | 0.518 ± 0.3120 | 0.476 ± 0.2934 | 0.498 ± 0.3027 |
| Baseline Ocular Characteristics (mITT) - Monocular Reading Speed(words per minute) | NT-501 | Sham | Total |
|---|---|---|---|
| Mean | 96.49 ± 47.314 | 94.09 ± 42.806 | 95.36 ± 45.051 |
3 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
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Studies of the drug tested here: Ciliary Neurotrophic Factor
Neurotech Pharmaceuticals