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Protocol Details

Phase II, Multicenter, Open-label Safety Study of Bilateral NT-501 in Participants with Macular Telangiectasia Type 2

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts




Sponsoring Institute

National Eye Institute (NEI)

Recruitment Detail

Type: No longer recruiting/follow-up only
Gender: Male & Female
Min Age: 18 Years
Max Age: N/A

Referral Letter Required


Population Exclusion(s)

Pregnant Women;
Adults who are or may become unable to consent;



Recruitment Keyword(s)



Macular Telangiectasia

Investigational Drug(s)


Investigational Device(s)



Drug: NT-501

Supporting Site

National Eye Institute


The NT-501 implant is a small capsule. It is placed inside the eye with a minor operation. In an earlier study, the eyes of participants with the degenerative vision disease Macular Telangiectasia Type 2 (MacTel) who got the implant seemed to do better as compared to those who did not. Researchers want to test the safety of placing the NT-501 in both eyes, by giving a second NT-501 to participants who already received one in the other eye in the earlier study.


To assess whether the NT-501 implant is well tolerated when the implants are placed in both eyes.


People with MacTel who already received NT-501 in one eye.


Participants will be screened with a medical history and physical exam. They will give blood samples. They may give a urine sample. They will have an eye exam and vision check. Their pupils will be measured and dilated. The lens, back of the eye, and retina will be examined. The pressure in their eyes will be checked. Photos will be taken of the implant site. The thickness of their retinas will be examined. Their MacTel will be confirmed by injecting dye into an arm vein and taking pictures of the back of their eye.

Participants will have implant surgery. Their pupils will be dilated. Their study eye will be numbed. Their vital signs may be monitored. They may receive fluids by vein. The implant will be held by a stitch in the eye. They will get a steroid injection. They will get eye drops to use at home.

Participants will have follow-up visits 1 day, 1 week, and 1, 3, and 6 months after surgery. They will repeat some screening tests.

Participation will last for 7 months.

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Study eligibility is to be assessed at Visit 1 (Screening). To participate in this study, the potential participant must meet all the following criteria:

General Inclusion Criteria:<TAB>

-Participants from the Phase 3 study must have completed the Month-24 visit

-Participant in the MacTel Phase 1/2 extension study or the Phase 3 study must exit these studies prior to entering the Bilateral Implant safety study (NTMT-02B)

-Participant must be willing and able to follow the study instructions and be willing and able to complete all required study procedures and visits

-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

-Females of childbearing potential must consent to and complete a pregnancy test during the screening visit

-A participant s refusal to allow the collection of blood samples for Ciliary neurotrophic factor (CNTF), Ab to CNTF, Nab to CNTF, Ab to NTC-201.6A cells, and Ab to DFHR will not exclude the participant from study participation

Ocular Inclusion Criteria

-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 their study eyes

-Participant must have steady fixation in the foveal or parafoveal area in the study eye and sufficiently clear media for good quality photographs

-Participant must have a NT-501 implant in one eye and have completed the Phase 1/2 extension study (NTMT-01/-2E) or the Month 24-visit of the Phase 3 study (NTMT-03)


Unless otherwise stated, eligibility assessments are to be performed at Visit 1 (Screening). The following criteria will exclude participant from entry into the study.

General Exclusion Criteria

-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)

-Participant is too ill to likely complete the entire study, based on the investigator s assessment

-Participant, in the opinion of the investigator, is not suitable for study participation

-Participant has any screening laboratory result (serum chemistry, hematology, urinalysis) that in the opinion of the investigator is not suitable for study participation

-History or current evidence of severe hypersensitivity to the NT-501 implant

-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 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

-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

-Participant has uncontrolled glaucoma; or ocular hypertension, i.e., IOP >= 25 mmHg in the study eye

-Participant has evidence of intraretinal or subretinal neovascularization or central serous chorio-retinopathy in the study eye

-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 non-proliferative or proliferative diabetic retinopathy, retinal detachment, macular hole, geographic atrophy, intraretinal or subretinal neovascularization, central serous chorio-retinopathy, pathological myopia

-Participant has a vitreous hemorrhage in the study eye at Visit 1 (Screening)

-Participant has the spherical equivalent of refractive error in the study eye demonstrating more than 8 diopters of myopia

-Participant has a history or evidence of penetrating ocular trauma in the study eye

-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

-Participant has uveitis, history of uveitis in either eye or history of ocular herpes virus in either eye

-Participant has undergone major surgery within the last 6 months (systemic or ocular in either eye) or participant who is likely to require major surgery within 6 months of Visit 1 (Screening)

-Participant has a 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)

-Participant has ocular hypotension in either eye (<6 mmHg) that in the opinion of the Investigator would interfere with the NT-501 implantation

-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 the administration of NT-501

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Not Provided

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Principal Investigator

Referral Contact

For more information:

Tiarnan D. Keenan, M.D.
National Eye Institute (NEI)
(301) 451-6330

Faith Chen
National Eye Institute (NEI)
National Institutes of Health
Building 10
Room 10D45
10 Center Drive
Bethesda, Maryland 20892
(301) 402-1369

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: TTY Users Dial 7-1-1

Clinical Trials Number:


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