A Study of an Encapsulated Cell Technology (ECT) Implant for Patients With Atrophic Macular Degeneration

NCT00447954 | Completed Phase 2 Results DMC FDA Drug FDA Device

• 1 other identifier: CNTF 2
• Study Type: interventional
• Target: 53
• Locations: 1 country
• Sites: 8

Brief Summary

The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in participants with atrophic macular degeneration. This research is being done because there are no effective therapies for people with atrophic macular degeneration. Age-related macular degeneration (AMD) is a condition that affects the macula, the central part of the retina that we use for seeing details. There are two types of AMD, one is the wet type in which new blood vessels grow, also known as choroidal neovascularization (CNV), but the other is the dry type in which the healthy cells die, and that is the target of this study. This is called atrophic macular degeneration. The implant is a small capsule that contains human retinal pigment epithelium cells. These cells have been given the ability to make CNTF and release it through the capsule membrane into the surrounding fluid. In this study, two different CNTF dose levels will be used: a high dose and a low dose, as well as a sham surgery (or placebo) group.

Conditions

Nonexudative Age-Related Macular Degeneration, Unspecified Eye, Intermediate Dry Stage

Keywords

eye disease; retinal disorder; CNTF; AMD; Atrophic AMD

Outcome Measures

Primary Outcomes (1)

• BCVA Response Defined as an Increased in 10 Letters at 1 Year Post-implant

  Response is defined as a improvement from baseline at Month 12 in monocular best visual acuity (BCVA) in the study eye of at least 10 letters as assessed by standard Early Treatment Diabetic Retinopathy Study (ETDRS) chart.

  1 year post-implant

Secondary Outcomes (1)

• The Change in BCVA Over the 18-month Follow-up Period

  From initial implant 18 months post-implant

Study Arms (3)

1 - High Dose

EXPERIMENTAL

Participant had surgically implanted high dose (NT-501-6A.02) CNTF-secreting NT-501 device which produced \~20 ng/device/day.

Combination Product: High Dose NT-501 implant

2 - Low Dose

EXPERIMENTAL

Participant had surgically implanted high dose (NT-501-6A.02) CNTF-secreting NT-501 device which produced \~5 ng/device/day.

Combination Product: Low Dose NT-501 implant

3 - Sham

SHAM COMPARATOR

Sham surgery procedure in which no device was implanted

Other: Sham

Interventions

High Dose NT-501 implant COMBINATION_PRODUCT

High Dose NT-501

1 - High Dose

Low Dose NT-501 implant COMBINATION_PRODUCT

Low Dose NT-501

2 - Low Dose

Sham OTHER

Sham Procedure

3 - Sham

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• To participate in this study, the participant had to understand and sign the protocol's informed consent (if the participant's vision was impaired to the point where it was not possible to read the informed consent document, the informed consent document was read in its entirety to the participant).
• Women of childbearing potential (women with last menses \<1 year prior to screening) had to agree to use an effective form of birth control from study onset until they completed the 18 month study visit.
• Participant had to be medically able to undergo ophthalmic surgery for NT-501 implant.
• Best-corrected visual acuity in the study eye between 20/50 and 20/200 (68-34 letter score) as measured using EVA.
• Presence in the study and/or fellow eye of geographic atrophy (GA) compatible with category 3 or 4 age-related macular degeneration (AMD) as defined by AREDS (AREDS, 2001). Also, the GA in the study eye had to be associated with vision loss as assessed by a vision test. GA was defined as one or more well-defined, usually more or less circular patches of partial or complete depigmentation of the RPE, typically with exposure of underlying choroidal blood vessels. Even if much of the RPE appeared to be preserved and large choroidal vessels were not visible, a roundish patch of RPE partial depigmentation might still be classified as early GA. A patch had to be at least 175 microns in area.
• Participants had steady fixation in the study eye in the foveal or parafoveal area with media clear enough for good quality photographs.

You may not qualify if:

• Participant less than 50 years of age (to minimize geographic atrophy from causes other than AMD).
• Participant medically unable to comply with study procedures or follow-up visits.
• Participant had evidence of ocular disease other than AMD that might confound the outcome of the study (e.g., diabetic retinopathy, uveitis, etc.).
• Participant had chronic requirement (e.g., \> or = 4 weeks at a time) for ocular medications or had disease(s), that in the judgment of the examining physician, were vision threatening or might affect the primary outcome (artificial tears were permitted).
• Participant had evidence of classic or occult choroidal neovascularization in either eye, which might include serous RPE detachment, stippling on a fluorescein angiogram, macular edema, evidence of hemorrhage and lipid, and disciform scar.
• Participant had a requirement for acyclovir and/or related products during study duration. To be eligible for this study, the participant had to discontinue use of these products prior to enrollment and could not continue with the products until after they had completed the study.
• Participant had evidence of central serous chorio-retinopathy (CSR) in either eye.
• Participant had evidence of pathologic myopia in either eye.
• Participant had evidence of pseudovitelliform macular degeneration (a dominantly inherited disease characterized by a round or oval yellow subretinal macular deposit) in either eye.
• Participant was receiving systemic steroids or other immunosuppressive medications.
• Participant with evidence of vitreo-retinal traction maculopathy in either eye.
• Participant had a history of laser, photodynamic therapy (PDT), intravitreal injection of antivascular endothelial growth factor (VEGF) agent, or any previous treatment for AMD other than AREDS or equivalent supplement formulation.
• Prior history of vitrectomy, penetrating keratoplasty, trabeculectomy or trabeculoplasty.
• Participant had any of the following lens opacities: cortical opacity \> standard 3, posterior subcapsular opacity \> standard 3, or a nuclear opacity \> standard 3 as measured on the AREDS clinical lens grading system.
• Participant had undergone lens removal in the last 3 months.

Sponsors & Collaborators

• Neurotech Pharmaceuticals: lead

Study Sites (8)

Retina-Vitreous Associates Medical Group

Beverly Hills, California, 90211, United States

Location

Retina Group of Florida

Hollywood, Florida, 33021-6746, United States

Location

Bascom Palmer Eye Institute

Miami, Florida, 33101, United States

Location

Ophthalmic Consultants of Boston

Boston, Massachusetts, 02114, United States

Location

Beaumont Eye Institute

Royal Oak, Michigan, 48073-6710, United States

Location

Retina Foundation of Southwest

Dallas, Texas, 75231, United States

Location

Vitreoretinal Consultants

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

Related Publications (1)

• Kauper K, McGovern C, Sherman S, Heatherton P, Rapoza R, Stabila P, Dean B, Lee A, Borges S, Bouchard B, Tao W. Two-year intraocular delivery of ciliary neurotrophic factor by encapsulated cell technology implants in patients with chronic retinal degenerative diseases. Invest Ophthalmol Vis Sci. 2012 Nov 1;53(12):7484-91. doi: 10.1167/iovs.12-9970.

  PMID: 23049090 DERIVED

MeSH Terms

Conditions

Eye Diseases; Retinal Diseases

Interventions

salicylhydroxamic acid

Results Point of Contact

• Title: CMO
• Organization: Neurotech Pharmaceuticals, LLC

neurotech@neurotechusa.com

401-333-3880

Study Officials

• Weng Tao, M.D., PhD

  Neurotech Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 9, 2007
• First Posted: March 15, 2007
• Study Start: January 5, 2007
• Primary Completion: May 11, 2009
• Study Completion: May 11, 2009
• Last Updated: March 28, 2025
• Results First Posted: March 28, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)