search
Back to results

A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa

Primary Purpose

Retinitis Pigmentosa

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
NT-501
NT-501
Sponsored by
Neurotech Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Retinitis Pigmentosa focused on measuring Retinitis Pigmentosa, eye disease, retinal disorder, CNTF

Eligibility Criteria

18 Years - 64 Years (Adult)All SexesDoes not accept healthy volunteers

Study inclusion / exclusion criteria:

Criteria for patients to qualify for the study include, but are not limited to:

  • Over 18 years of age, and less than 65 years of age
  • Diagnosis of retinitis pigmentosa, Usher Syndrome Type 2 or 3 or Choroideremia
  • Visual acuity no worse than 20/63
  • Experience with at least two full threshold Humphrey Visual Field 30-2 tests, one completed within the year prior to enrolling in this study

The following criteria will exclude patients from the study:

  • Pregnant or lactating females, or females planning to become pregnant during the study or not using an acceptable method of contraception.
  • Retinitis pigmentosa caused by a classic syndrome, including Usher Type I
  • Other eye diseases including advanced cataract.
  • Chronic systemic disease requiring continuous treatment with systemic steroids, immunosuppressive medications or insulin.

Sites / Locations

  • Retina-Vitreous Associates Medical Group
  • University of Califoria, Davis
  • University of California, San Francisco
  • Bascom Palmer Eye Insitute
  • Ophthalmic Consultants of Boston
  • Kellogg Eye Center
  • University of Minnesota
  • NY University Medical Center
  • Casey Eye Institue
  • The Hamilton Eye Institute
  • Retina Foundation of Southwest
  • University of Utah

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

1 NT-501 implant

2 NT-501 implant

Arm Description

High Dose

Low Dose

Outcomes

Primary Outcome Measures

Change in Humphrey visual field sensitivity from baseline to month 12.

Secondary Outcome Measures

Change in visual field sensitivity
Mean, median and distribution of change in Best Corrected Visual Acuity
Change in ERG
Change in OCT
Change in inflammation
Change in vision-related quality of life

Full Information

First Posted
March 9, 2007
Last Updated
November 15, 2016
Sponsor
Neurotech Pharmaceuticals
search

1. Study Identification

Unique Protocol Identification Number
NCT00447980
Brief Title
A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa
Official Title
A Phase II/III Study of Encapsulated Human NTC-201 Cell Implants Releasing Ciliary Neurotrophic Factor (CNTF) for Participants With Retinitis Pigmentosa Using Visual Field Sensitivity as the Primary Outcome
Study Type
Interventional

2. Study Status

Record Verification Date
November 2016
Overall Recruitment Status
Completed
Study Start Date
January 2007 (undefined)
Primary Completion Date
July 2010 (Actual)
Study Completion Date
July 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurotech Pharmaceuticals

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II & III, and Choroideremia. This research is being done because there are no effective therapies for people with these retinal degenerations. They are genetic disorders that affect one's ability to see at night, and later cause tunnel vision and loss of central vision. Retinal degenerations affect the retina, a light sensitive layer of cells in the back of the eye. Slowly over time, these cells die and cause permanent loss of vision. 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 in one eye, as well as a sham (or placebo) surgery in the other eye.
Detailed Description
This study will involve about 18 visits over 2½ years for specific tests of the participant's vision and health. These visits may include visual exams, blood draw for laboratory testing, brief medical history and exam, and occasionally a questionnaire (survey), in addition to the visit for the surgical procedures. The primary effectiveness outcome for this study will be a visual field score one year after the implant surgery. There will be about 12 centers participating in this study, and up to 60 people enrolled, across the US. Each participant joining the study who has completed initial screening will then be scheduled to have a brief surgical procedure performed on each eye, one of which will include a very small cell-filled implant. Follow-up visits for repeat assessments will be required regularly to determine if the implant being tested is safe and effective for use to treat retinitis pigmentosa (RP).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Retinitis Pigmentosa
Keywords
Retinitis Pigmentosa, eye disease, retinal disorder, CNTF

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantOutcomes Assessor
Allocation
Randomized
Enrollment
68 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1 NT-501 implant
Arm Type
Experimental
Arm Description
High Dose
Arm Title
2 NT-501 implant
Arm Type
Experimental
Arm Description
Low Dose
Intervention Type
Drug
Intervention Name(s)
NT-501
Other Intervention Name(s)
CNTF implant
Intervention Description
High Dose
Intervention Type
Drug
Intervention Name(s)
NT-501
Other Intervention Name(s)
CNTF implant
Intervention Description
Low Dose
Primary Outcome Measure Information:
Title
Change in Humphrey visual field sensitivity from baseline to month 12.
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Change in visual field sensitivity
Time Frame
24 months
Title
Mean, median and distribution of change in Best Corrected Visual Acuity
Time Frame
12 to 24 months
Title
Change in ERG
Time Frame
12 to 24 months
Title
Change in OCT
Time Frame
12 to 24 months
Title
Change in inflammation
Time Frame
12 to 24 months
Title
Change in vision-related quality of life
Time Frame
12 to 24 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
64 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Study inclusion / exclusion criteria: Criteria for patients to qualify for the study include, but are not limited to: Over 18 years of age, and less than 65 years of age Diagnosis of retinitis pigmentosa, Usher Syndrome Type 2 or 3 or Choroideremia Visual acuity no worse than 20/63 Experience with at least two full threshold Humphrey Visual Field 30-2 tests, one completed within the year prior to enrolling in this study The following criteria will exclude patients from the study: Pregnant or lactating females, or females planning to become pregnant during the study or not using an acceptable method of contraception. Retinitis pigmentosa caused by a classic syndrome, including Usher Type I Other eye diseases including advanced cataract. Chronic systemic disease requiring continuous treatment with systemic steroids, immunosuppressive medications or insulin.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Weng Tao, MD, PhD
Organizational Affiliation
Neurotech Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Retina-Vitreous Associates Medical Group
City
Beverly Hills
State/Province
California
ZIP/Postal Code
90211
Country
United States
Facility Name
University of Califoria, Davis
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
University of California, San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94143-0730
Country
United States
Facility Name
Bascom Palmer Eye Insitute
City
Miami
State/Province
Florida
ZIP/Postal Code
33101
Country
United States
Facility Name
Ophthalmic Consultants of Boston
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Kellogg Eye Center
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48105
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455-0501
Country
United States
Facility Name
NY University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Casey Eye Institue
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239-4197
Country
United States
Facility Name
The Hamilton Eye Institute
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38163
Country
United States
Facility Name
Retina Foundation of Southwest
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
23668681
Citation
Birch DG, Weleber RG, Duncan JL, Jaffe GJ, Tao W; Ciliary Neurotrophic Factor Retinitis Pigmentosa Study Groups. Randomized trial of ciliary neurotrophic factor delivered by encapsulated cell intraocular implants for retinitis pigmentosa. Am J Ophthalmol. 2013 Aug;156(2):283-292.e1. doi: 10.1016/j.ajo.2013.03.021. Epub 2013 May 10.
Results Reference
derived
PubMed Identifier
23049090
Citation
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.
Results Reference
derived
PubMed Identifier
21087953
Citation
Talcott KE, Ratnam K, Sundquist SM, Lucero AS, Lujan BJ, Tao W, Porco TC, Roorda A, Duncan JL. Longitudinal study of cone photoreceptors during retinal degeneration and in response to ciliary neurotrophic factor treatment. Invest Ophthalmol Vis Sci. 2011 Apr 6;52(5):2219-26. doi: 10.1167/iovs.10-6479.
Results Reference
derived

Learn more about this trial

A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa

We'll reach out to this number within 24 hrs