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CNTF Implants for CNGB3 Achromatopsia (CNTF-CNGB3-1)

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ClinicalTrials.gov Identifier: NCT01648452
Recruitment Status : Completed
First Posted : July 24, 2012
Results First Posted : February 25, 2014
Last Update Posted : November 21, 2016
Sponsor:
Information provided by (Responsible Party):
Paul A. Sieving, M.D., National Institutes of Health Clinical Center (CC)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Eye Disease
Achromatopsia
Intervention Biological: NT-501 CNTF-releasing implant
Enrollment 5
Recruitment Details  
Pre-assignment Details  
Arm/Group Title NT-501 CNTF-releasing Implant
Hide Arm/Group Description Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Period Title: Overall Study
Started 5
Completed 5 [1]
Not Completed 0
[1]
One participant was explanted due to an adverse event. Remainder kept implant at study completion.
Arm/Group Title NT-501 CNTF-releasing Implant
Hide Arm/Group Description Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Overall Number of Baseline Participants 5
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 5 participants
35.6  (11.37)
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
<=18 years
0
   0.0%
Between 18 and 65 years
5
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
Female
2
  40.0%
Male
3
  60.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 5 participants
5
1.Primary Outcome
Title Number of Adverse Events at Six Months Post-Implantation
Hide Description The primary outcome is the total number of adverse events reported within six months post-implantation.
Time Frame Day 1, Week 1, Week 4, Week 12, Week 24 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title NT-501 CNTF-releasing Implant
Hide Arm/Group Description:
Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: Adverse Events
10
2.Primary Outcome
Title Number of Severe Adverse Events at Six Months Post-Implantation
Hide Description The number of severe adverse events reported within six months post-implantation.
Time Frame Day 1, Week 1, Week 4, Week 12, Week 24 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title NT-501 CNTF-releasing Implant
Hide Arm/Group Description:
Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: Adverse Events
1
3.Primary Outcome
Title Number of Ocular Adverse Events at Six Months Post-Implantation
Hide Description The number of eye-related adverse events reported within six months post-implantation.
Time Frame Day 1, Week 1, Week 4, Week 12, Week 24 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title NT-501 CNTF-releasing Implant
Hide Arm/Group Description:
Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: Adverse Events
9
4.Primary Outcome
Title Number of Non-Ocular Adverse Events at Six Months Post-Implantation
Hide Description The number of non eye-related adverse events reported within six months post-implantation.
Time Frame Day 1, Week 1, Week 4, Week 12, Week 24 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title NT-501 CNTF-releasing Implant
Hide Arm/Group Description:
Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: Adverse Events
1
5.Secondary Outcome
Title Number of Adverse Events at All Time Points Post-Implantation
Hide Description The total number of adverse events reported from Day 1 post-implantation through study completion at Year 3.
Time Frame Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title NT-501 CNTF-releasing Implant
Hide Arm/Group Description:
Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: Adverse Events
15
6.Secondary Outcome
Title Number of Severe Adverse Events at All Time Points Post-Implantation
Hide Description

The total number of severe adverse events reported from Day 1 post-implantation through study completion at Year 3.

Although there were two serious adverse events (SAEs) reported during the study, only one event's severity was classified as "severe."

Time Frame Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title NT-501 CNTF-releasing Implant
Hide Arm/Group Description:
Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: Adverse Events
1
7.Secondary Outcome
Title Number of Ocular Adverse Events at All Time Points Post-Implantation
Hide Description The total number of eye-related adverse events reported from Day 1 post-implantation through study completion at Year 3.
Time Frame Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title NT-501 CNTF-releasing Implant
Hide Arm/Group Description:
Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: Adverse Events
11
8.Secondary Outcome
Title Number of Non-Ocular Adverse Events at All Time Points Post-Implantation
Hide Description The total number of non eye-related adverse events reported from Day 1 post-implantation through study completion at Year 3.
Time Frame Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title NT-501 CNTF-releasing Implant
Hide Arm/Group Description:
Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: Adverse Events
4
9.Secondary Outcome
Title Number of Participants Who Experienced an Improvement in Visual Acuity of Greater Than 0.3 logMAR (Logarithm of the Minimum Angle of Resolution) Post-Implantation in the Study Eye.
Hide Description

Improvement of visual acuity was assessed on both the study and control eyes. Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20. .

The LogMAR scale [expressed as the (decadic) logarithm of the minimum angle of resolution (range from +1.00 to -0.30)] converts the geometric sequence of a traditional chart to a linear scale. It measures VA loss; positive values indicate vision loss, whereas negative values denote normal or better VA. A lower LogMAR value indicates better VA. For example, a visual acuity of 20/20 corresponds to a logMAR value of zero (0), and a visual acuity of 20/100 corresponds to a LogMAR value of 0.7.

Time Frame Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title NT-501 CNTF-releasing Implant
Hide Arm/Group Description:
Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: participants
0
10.Secondary Outcome
Title Number of Participants Who Experienced an Improvement in Visual Acuity of Greater Than 0.3 logMAR (Logarithm of the Minimum Angle of Resolution) Post-Implantation in the Untreated Control Eye.
Hide Description

Improvement of visual acuity was assessed on both the study and control eyes. Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20. .

The LogMAR scale [expressed as the (decadic) logarithm of the minimum angle of resolution (range from +1.00 to -0.30)] converts the geometric sequence of a traditional chart to a linear scale. It measures VA loss; positive values indicate vision loss, whereas negative values denote normal or better VA. A lower LogMAR value indicates better VA. For example, a visual acuity of 20/20 corresponds to a logMAR value of zero (0), and a visual acuity of 20/100 corresponds to a LogMAR value of 0.7.

Time Frame Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Untreated Control Eye
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: participants
0
11.Secondary Outcome
Title Number of Participants Who Experienced an Increase in Either the Rod or Cone Electroretinogram (ERG) Responses of More Than 75% Post-Implantation in the Study Eye.
Hide Description Full-field ERGs were recorded according to International Society of Clinical Electrophysiology of Vision Standards (ISCEV).
Time Frame Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title NT-501 CNTF-releasing Implant
Hide Arm/Group Description:
Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: participants
0
12.Secondary Outcome
Title Number of Participants Who Experienced an Increase in Either the Rod or Cone Electroretinogram (ERG) Responses of More Than 75% Post-Implantation in the Untreated Control Eye.
Hide Description Full-field ERGs were recorded according to International Society of Clinical Electrophysiology of Vision Standards (ISCEV).
Time Frame Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Untreated Control Eye
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: participants
0
13.Secondary Outcome
Title Number of Participants Who Experienced an Improvement in Color Discrimination and/or Matching Post-Implantation in the Study Eye.
Hide Description Color hue discrimination was tested by the Nagel anomaloscope, American Optical Hardy Rand Rittler (AOHRR) color plates, and a low vision version of the Cambridge Color Test (LvCCT) implemented on a ViSaGe System (Cambridge Research Systems Ltd., Rochester, UK) using custom-written software. Hardy Rand Rittler testing followed the guidelines accompanying the test and administered under a Macbeth Lamp at 300 lux.
Time Frame Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title NT-501 CNTF-releasing Implant
Hide Arm/Group Description:
Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: participants
0
14.Secondary Outcome
Title Number of Participants Who Experienced an Improvement in Color Discrimination and/or Matching Post-Implantation in the Untreated Control Eye.
Hide Description Color hue discrimination was tested by the Nagel anomaloscope, American Optical Hardy Rand Rittler (AOHRR) color plates, and a low vision version of the Cambridge Color Test (LvCCT) implemented on a ViSaGe System (Cambridge Research Systems Ltd., Rochester, UK) using custom-written software. Hardy Rand Rittler testing followed the guidelines accompanying the test and administered under a Macbeth Lamp at 300 lux.
Time Frame Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Untreated Control Eye
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: participants
0
Time Frame Adverse events were collected at all time points until study completion (up to Week 156 post-implantation).
Adverse Event Reporting Description The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.
 
Arm/Group Title NT-501 CNTF-releasing Implant Events ≤ 6 Months Post-implant NT-501 CNTF-releasing Implant Events > 6 Months Post-implant
Hide Arm/Group Description Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline. The events listed reflect the primary outcome endpoint of adverse events reported within 6 months post-implantation. Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline. The events listed are adverse events reported after the primary outcome endpoint of 6 months post-implantation.
All-Cause Mortality
NT-501 CNTF-releasing Implant Events ≤ 6 Months Post-implant NT-501 CNTF-releasing Implant Events > 6 Months Post-implant
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
NT-501 CNTF-releasing Implant Events ≤ 6 Months Post-implant NT-501 CNTF-releasing Implant Events > 6 Months Post-implant
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/5 (20.00%)      1/5 (20.00%)    
Eye disorders     
Uveitis  1  1/5 (20.00%)  1 0/5 (0.00%)  0
Retinal Detachment  2  0/5 (0.00%)  0 1/5 (20.00%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (16.0)
2
Term from vocabulary, MedDRA (18.1)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
NT-501 CNTF-releasing Implant Events ≤ 6 Months Post-implant NT-501 CNTF-releasing Implant Events > 6 Months Post-implant
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/5 (100.00%)      2/5 (40.00%)    
Ear and labyrinth disorders     
Vertigo  3  0/5 (0.00%)  0 1/5 (20.00%)  1
Eye disorders     
Delayed Dark Adaptation  1 [1]  4/5 (80.00%)  4 0/5 (0.00%)  0
Uveitis  1  1/5 (20.00%)  1 0/5 (0.00%)  0
Vitreous detachment  1  1/5 (20.00%)  1 0/5 (0.00%)  0
Vitritis  2  2/5 (40.00%)  2 0/5 (0.00%)  0
Lenticular Opacities  3  0/5 (0.00%)  0 1/5 (20.00%)  1
Injury, poisoning and procedural complications     
Arthropod Bite  1  1/5 (20.00%)  1 0/5 (0.00%)  0
Surgical and medical procedures     
Shoulder Operation  3  0/5 (0.00%)  0 1/5 (20.00%)  1
Uterine operation  3  0/5 (0.00%)  0 1/5 (20.00%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (16.0)
2
Term from vocabulary, MedDRA (15.1)
3
Term from vocabulary, MedDRA (18.1)
[1]
The event coded to a MedDRA v16 Preferred Term of Night Blindness but the verbatim Lower Level Terminology is delayed dark adaptation. As the MedDRA term may misrepresent participants as being blind, the verbatim event will be utilized, not MedDRA.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Paul A Sieving, MD, PhD
Organization: National Eye Institute
Phone: 301-496-2234
EMail: ps261o@nih.gov
Layout table for additonal information
Responsible Party: Paul A. Sieving, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT01648452     History of Changes
Other Study ID Numbers: 120167
12-EI-0167
First Submitted: July 20, 2012
First Posted: July 24, 2012
Results First Submitted: January 9, 2014
Results First Posted: February 25, 2014
Last Update Posted: November 21, 2016