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Protocol Details

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 NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts




Sponsoring Institute

National Eye Institute (NEI)

Recruitment Detail

Type: Completed Study; data analyses ongoing
Gender: Male & Female
Min Age: 21 Years
Max Age: 80 Years

Referral Letter Required


Population Exclusion(s)

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


Ciliary Neurotrophic Factor

Recruitment Keyword(s)



Macular Telangiectasia

Investigational Drug(s)


Investigational Device(s)



Procedure/Surgery: NT-501
Procedure/Surgery: Sham

Supporting Site

National Eye Institute


People with the eye disease Macular Telangiectasia Type 2 (MacTel) often lose vision. The molecule ciliary neurotrophic factor (CNTF) might keep some eye cells healthy. Previously, releasing CNTF from a small capsule in the eye helped people with MacTel. Researchers want to test the capsule again and compare results.


To test if releasing CNTF from a small capsule implanted in the eye helps people with MacTel.


Adults 21-80 years old with MacTel


Participants will be screened with:

-Medical history

-Physical exam

-Vital signs

-Eyes dilated, measured, photographed

-Eye tests with flashing lights, laser scan, dye injected in an arm

-Reading tests

-Vision questions

-Urine pregnancy tests

-Blood tests

Participants will be randomly assigned to Group A or B. Participants and testing staff will not know which group participants are in.

All participants will have surgery:

-Eyedrops will dilate and numb the eye

-Participants may have a numbing injection and medicine to make them sleepy.

-Group A: The capsule will be implanted and held with a permanent stitch on the white of the eye. The outside of the eye will be closed with stitches that get absorbed. An injection will reduce swelling.

-Group B: Doctors will make a small cut in the eye and place only the absorbable stitches. They will talk like they are doing the implant surgery. They may give a pretend injection.

-All participants will get eyedrops for infection and swelling and need a ride home. They will wear an eyepatch for several hours.

Participants will have 7 visits and 2 calls over 2 years.

Visits may include:

-Medical history

-Vital signs

-Eyes dilated, measured, photographed

-Eye tests with flashing lights, laser scan, dye injected in an arm

-Reading tests

-Vision questions

-Urine pregnancy tests

-Blood tests

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To participate in this study, the potential participant and at least 1 of their eyes must meet all of the following criteria:

1. Participant must have at least 1 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

2. Participant must have an IS/OS PR break and EZ (area of IS/OS loss) as measured by SD-OCT between 0.16 and 2.00 mm^2

3. Participant s BCVA is 54-letter score or better (20/80 or better) as measured by the ETDRS chart at screening

4. Participant must have steady fixation in the foveal or parafoveal area and sufficiently clear media for good quality photographs

5. Participant must be greater than 21 years of age or less than 80 years of age at screening

6. Participant must be able to provide written informed consent to participate in the study, in accordance with the ICH GCP guidelines, and local regulations, before initiating any study-related procedures


To participate in this study, the potential participant must not meet any of the following criteria. The ocular exclusion criteria are related to the study eye (unless indicated for either eye):

1. Participant is medically unable to comply with study procedures or follow-up visits

2. Participant received intravitreal steroid therapy for nonneovascular MacTel within the last 3 months

3. 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 therapy in the fellow eye at randomization

4. 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)

5. Participant has a chronic requirement (eg, greater than or equal to 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)

6. Participant has evidence of intraretinal neovascularization or SRNV, as evidenced by hemorrhage, hard exudate, subretinal fluid or intraretinal fluid in either eye

7. Participant has evidence of central serous chorioretinopathy in either eye

8. Participant has evidence of pathologic myopia in either eye

9. Participant has significant corneal or media opacities in either eye

10. Participant has had a vitrectomy, penetrating keratoplasty, trabeculectomy, or trabeculoplasty

11. 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

12. Participant has undergone lens removal in the previous 3 months or YAG laser within 4 weeks

13. Participant was a participant in any other clinical trial of an intervention (drug or device) within the last 6 months

14. Participant is on chemotherapy

15. Participant is pregnant or breastfeeding

16. Participant has a history of malignancy that would compromise the 24-month study survival

17. Participant with a history of ocular herpes virus in either eye

18. 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

19. Participant has evidence of intraretinal hyperreflectivity by OCT

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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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