Trial Outcomes & Findings for CNTF Implants for CNGB3 Achromatopsia (NCT NCT01648452)

NCT ID: NCT01648452

Last Updated: 2016-11-21

Results Overview

The primary outcome is the total number of adverse events reported within six months post-implantation.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 5 participants
• Primary outcome timeframe: Day 1, Week 1, Week 4, Week 12, Week 24 post-implantation
• Results posted on: 2016-11-21

Participant Flow

Participant milestones

Measure	NT-501 CNTF-releasing Implant Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Overall Study STARTED	5
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

CNTF Implants for CNGB3 Achromatopsia

Baseline characteristics by cohort

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Age, Continuous	35.6 years STANDARD_DEVIATION 11.37 • n=5 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24 post-implantation

The primary outcome is the total number of adverse events reported within six months post-implantation.

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Number of Adverse Events at Six Months Post-Implantation	10 Adverse Events

PRIMARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24 post-implantation

The number of severe adverse events reported within six months post-implantation.

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Number of Severe Adverse Events at Six Months Post-Implantation	1 Adverse Events

PRIMARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24 post-implantation

The number of eye-related adverse events reported within six months post-implantation.

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Number of Ocular Adverse Events at Six Months Post-Implantation	9 Adverse Events

PRIMARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24 post-implantation

The number of non eye-related adverse events reported within six months post-implantation.

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Number of Non-Ocular Adverse Events at Six Months Post-Implantation	1 Adverse Events

SECONDARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation

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

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Number of Adverse Events at All Time Points Post-Implantation	15 Adverse Events

SECONDARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation

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."

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Number of Severe Adverse Events at All Time Points Post-Implantation	1 Adverse Events

SECONDARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation

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

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Number of Ocular Adverse Events at All Time Points Post-Implantation	11 Adverse Events

SECONDARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation

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

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Number of Non-Ocular Adverse Events at All Time Points Post-Implantation	4 Adverse Events

SECONDARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation

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.

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
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.	0 participants

SECONDARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation

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.

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
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.	0 participants

SECONDARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation

Full-field ERGs were recorded according to International Society of Clinical Electrophysiology of Vision Standards (ISCEV).

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
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.	0 participants

SECONDARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation

Full-field ERGs were recorded according to International Society of Clinical Electrophysiology of Vision Standards (ISCEV).

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
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.	0 participants

SECONDARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation

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.

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Number of Participants Who Experienced an Improvement in Color Discrimination and/or Matching Post-Implantation in the Study Eye.	0 participants

SECONDARY outcome

Timeframe: Day 1, Week 1, Week 4, Week 12, Week 24, Week 52, Week 78, Week 156 post-implantation

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.

Outcome measures

Measure	NT-501 CNTF-releasing Implant n=5 Participants Participants received an ocular implantation of a NT-501 CNTF-releasing capsule (20 ng/day) in one eye (the study eye) at baseline.
Number of Participants Who Experienced an Improvement in Color Discrimination and/or Matching Post-Implantation in the Untreated Control Eye.	0 participants

Adverse Events

NT-501 CNTF-releasing Implant Events ≤ 6 Months Post-implant

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

NT-501 CNTF-releasing Implant Events > 6 Months Post-implant

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Measure	NT-501 CNTF-releasing Implant Events ≤ 6 Months Post-implant n=5 participants at risk 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.	NT-501 CNTF-releasing Implant Events > 6 Months Post-implant n=5 participants at risk 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.
Eye disorders Uveitis	20.0% 1/5 • Number of events 1 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.	0.00% 0/5 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.
Eye disorders Retinal Detachment	0.00% 0/5 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.	20.0% 1/5 • Number of events 1 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.

Other adverse events

Measure	NT-501 CNTF-releasing Implant Events ≤ 6 Months Post-implant n=5 participants at risk 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.	NT-501 CNTF-releasing Implant Events > 6 Months Post-implant n=5 participants at risk 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.
Injury, poisoning and procedural complications Arthropod Bite	20.0% 1/5 • Number of events 1 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.	0.00% 0/5 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.
Eye disorders Delayed Dark Adaptation	80.0% 4/5 • Number of events 4 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.	0.00% 0/5 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.
Eye disorders Uveitis	20.0% 1/5 • Number of events 1 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.	0.00% 0/5 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.
Eye disorders Vitreous detachment	20.0% 1/5 • Number of events 1 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.	0.00% 0/5 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.
Eye disorders Vitritis	40.0% 2/5 • Number of events 2 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.	0.00% 0/5 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.
Surgical and medical procedures Shoulder Operation	0.00% 0/5 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.	20.0% 1/5 • Number of events 1 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.
Eye disorders Lenticular Opacities	0.00% 0/5 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.	20.0% 1/5 • Number of events 1 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.
Ear and labyrinth disorders Vertigo	0.00% 0/5 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.	20.0% 1/5 • Number of events 1 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.
Surgical and medical procedures Uterine operation	0.00% 0/5 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.	20.0% 1/5 • Number of events 1 • Adverse events were collected at all time points until study completion (up to Week 156 post-implantation). The primary outcome is the number and severity of adverse events and systemic and ocular toxicities within six months post-implantation.

Additional Information

Paul A Sieving, MD, PhD

National Eye Institute

Phone: 301-496-2234

Email: ps261o@nih.gov

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place