Trial Outcomes & Findings for Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa (NCT NCT01530659)

Last Updated: 2023-02-14

Results Overview

Average of cone spacing (nearest neighbor distance) at all regions of interest with at least 50 contiguous unambiguous cones identified over the central 5.7 degrees of the macula using confocal AOSLO at two baseline visits within each eye. Cone spacing measures were converted to Z scores based on normal mean values at similar distances from the fovea from a database of 27 age-similar normal eyes. The mean of 2 baseline cone spacing Z-score values were subtracted from the cone spacing Z score values obtained at post-op month 36 A Z-score of 0 represents the mean cone spacing value at the distance from the fovea measured from 27 healthy subjects. A Z-score greater than +2 represents an abnormally increased cone spacing value at the distance from the fovea where the measurement was performed. This suggests fewer cones are present than normal at that location.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

22 participants

Primary outcome timeframe

Post-op Month 36

Results posted on

2023-02-14

Participant Flow

Eligible subjects were \>18 years old with retinitis pigmentosa or Usher syndrome and adaptive optics scanning laser ophthalmoscopy (AOSLO) images in each eye with at least 7 regions having at least 50 contiguous unambiguous cones in the central 5.7 degrees of the macula at 2 baseline visits. Exclusion criteria included visual acuity worse than 20/40, lens opacity, \> -6.00 D myopia, nystagmus, cystoid macular edema, retinal vascular disease, unstable fixation, and severe hearing loss.

Unit of analysis: Eyes

Participant milestones

Participant milestones
Measure	NT-501 Implant A pre-determined randomization scheme will be utilized to designate each of the patient's eyes as the primary eye (to receive the NT-501 implant) or the fellow eye (Control-to receive sham surgery). Primary eye will receive the NT-501 implant	Sham Surgery A pre-determined randomization scheme will be utilized to designate each of the patient's eyes as the primary eye (to receive the NT-501 implant) or the fellow eye (Control-to receive sham surgery).
Overall Study STARTED	22 22	22 22
Overall Study COMPLETED	22 22	22 22
Overall Study NOT COMPLETED	0 0	0 0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	NT-501 Implant n=22 Eyes A pre-determined randomization scheme will be utilized to designate each of the patient's eyes as the primary eye (to receive the NT-501 implant) or the fellow eye (Control-to receive sham surgery). Primary eye will receive the NT-501 implant	Sham Surgery n=22 Eyes A pre-determined randomization scheme will be utilized to designate each of the patient's eyes as the primary eye (to receive the NT-501 implant) or the fellow eye (Control-to receive sham surgery).	Total n=44 Eyes Total of all reporting groups
Age, Continuous	39.8 Years STANDARD_DEVIATION 11.9 • n=5 Participants	39.8 Years STANDARD_DEVIATION 11.9 • n=7 Participants	39.8 Years STANDARD_DEVIATION 11.9 • n=5 Participants
Sex: Female, Male Female	9 Eyes n=40 Eyes	9 Eyes n=105 Eyes	18 Eyes n=210 Eyes
Sex: Female, Male Male	13 Eyes n=40 Eyes	13 Eyes n=105 Eyes	26 Eyes n=210 Eyes
Ethnicity (NIH/OMB) Hispanic or Latino	2 Eyes n=40 Eyes	2 Eyes n=105 Eyes	4 Eyes n=210 Eyes
Ethnicity (NIH/OMB) Not Hispanic or Latino	20 Eyes n=40 Eyes	20 Eyes n=105 Eyes	40 Eyes n=210 Eyes
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Eyes n=40 Eyes	0 Eyes n=105 Eyes	0 Eyes n=210 Eyes
Race (NIH/OMB) American Indian or Alaska Native	1 Eyes n=40 Eyes	1 Eyes n=105 Eyes	2 Eyes n=210 Eyes
Race (NIH/OMB) Asian	2 Eyes n=40 Eyes	2 Eyes n=105 Eyes	4 Eyes n=210 Eyes
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Eyes n=40 Eyes	1 Eyes n=105 Eyes	2 Eyes n=210 Eyes
Race (NIH/OMB) Black or African American	0 Eyes n=40 Eyes	0 Eyes n=105 Eyes	0 Eyes n=210 Eyes
Race (NIH/OMB) White	18 Eyes n=40 Eyes	18 Eyes n=105 Eyes	36 Eyes n=210 Eyes
Race (NIH/OMB) More than one race	0 Eyes n=40 Eyes	0 Eyes n=105 Eyes	0 Eyes n=210 Eyes
Race (NIH/OMB) Unknown or Not Reported	0 Eyes n=40 Eyes	0 Eyes n=105 Eyes	0 Eyes n=210 Eyes
Region of Enrollment United States	22 Eyes n=40 Eyes	22 Eyes n=105 Eyes	44 Eyes n=210 Eyes

PRIMARY outcome

Timeframe: Post-op Month 36

Population: Measure type: difference from mean baseline to 36 months in change between intervention and control eyes in mean cone spacing Z score averaged over regions of interest in each eye.

Outcome measures

Outcome measures
Measure	NT-501 Implant n=22 Eyes A pre-determined randomization scheme will be utilized to designate each of the patient's eyes as the primary eye (to receive the NT-501 implant) or the fellow eye (Control-to receive sham surgery). Primary eye will receive the NT-501 implant	Sham Surgery n=22 Eyes A pre-determined randomization scheme will be utilized to designate each of the patient's eyes as the primary eye (to receive the NT-501 implant) or the fellow eye (Control-to receive sham surgery).
Mean Change in Cone Spacing in Arcminutes (Z Score) of 2 Baseline Values Were Compared With Measurements Obtained at Post-op Month 36	1.13 Difference in change in Z score Standard Deviation 1.03	0.85 Difference in change in Z score Standard Deviation 1.78

SECONDARY outcome

Timeframe: Post-op Month 36

Population: Difference in change in logMAR visual acuity between NT-501 and contralateral sham-treated eyes.

Difference in change in logMAR visual acuity between NT-501 and contralateral sham-treated eyes. Change in visual acuity was measured based on the number of letters read on a vision chart using a standard protocol. The log of the mean angle of resolution (logMAR) was used to describe the size of the smallest letters that the patient could read. A logMAR value of 0.00 corresponds to visual acuity of 20/20, a logMAR value of 0.3 corresponds to visual acuity of 20/40, a logMAR value of 0.7 corresponds to visual acuity of 20/100, and a logMAR value of 1.00 corresponds to visual acuity of 20/200.

Outcome measures

Outcome measures
Measure	NT-501 Implant n=22 Eyes A pre-determined randomization scheme will be utilized to designate each of the patient's eyes as the primary eye (to receive the NT-501 implant) or the fellow eye (Control-to receive sham surgery). Primary eye will receive the NT-501 implant	Sham Surgery n=22 Eyes A pre-determined randomization scheme will be utilized to designate each of the patient's eyes as the primary eye (to receive the NT-501 implant) or the fellow eye (Control-to receive sham surgery).
Difference in logMAR Visual Acuity Change Between CNTF- and Sham Treated Eyes	0.004 logMAR Standard Deviation 0.010	0.014 logMAR Standard Deviation 0.082

Adverse Events

NT-501 Implant

Serious events: 4 serious events

Other events: 2 other events

Deaths: 0 deaths

Sham Surgery

Serious events: 4 serious events

Other events: 1 other events

Deaths: 0 deaths

Prior to Randomization

Serious events: 0 serious events

Other events: 0 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	NT-501 Implant n=22 participants at risk A pre-determined randomization scheme will be utilized to designate each of the patient's eyes as the primary eye (to receive the NT-501 implant) or the fellow eye (Control-to receive sham surgery). Primary eye will receive the NT-501 implant	Sham Surgery n=22 participants at risk A pre-determined randomization scheme will be utilized to designate each of the patient's eyes as the primary eye (to receive the NT-501 implant) or the fellow eye (Control-to receive sham surgery).	Prior to Randomization One participant was consented and enrolled but died before being randomized to receive the study intervention.
Pregnancy, puerperium and perinatal conditions Pregnancy	4.5% 1/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	4.5% 1/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	— 0/0 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.
Cardiac disorders Reversible Cardiac Vasospasm	4.5% 1/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	4.5% 1/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	— 0/0 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.
Eye disorders Total Eye Disorders	18.2% 4/22 • Number of events 5 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	4.5% 1/22 • Number of events 1 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	— 0/0 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.
Eye disorders Clinically Significant Cystoid Macular Edema	4.5% 1/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	0.00% 0/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	— 0/0 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.
Eye disorders Secondary Surgical Intervention (excluding Posterior Capsulotomy)	4.5% 1/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	4.5% 1/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	— 0/0 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.
Eye disorders Ocular discomfort	4.5% 1/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	0.00% 0/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	— 0/0 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.
Eye disorders Reduced peripheral visual field	4.5% 1/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	0.00% 0/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	— 0/0 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.

Other adverse events

Other adverse events
Measure	NT-501 Implant n=22 participants at risk A pre-determined randomization scheme will be utilized to designate each of the patient's eyes as the primary eye (to receive the NT-501 implant) or the fellow eye (Control-to receive sham surgery). Primary eye will receive the NT-501 implant	Sham Surgery n=22 participants at risk A pre-determined randomization scheme will be utilized to designate each of the patient's eyes as the primary eye (to receive the NT-501 implant) or the fellow eye (Control-to receive sham surgery).	Prior to Randomization One participant was consented and enrolled but died before being randomized to receive the study intervention.
Eye disorders Epiretinal membrane	4.5% 1/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	0.00% 0/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	— 0/0 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.
Musculoskeletal and connective tissue disorders Pelvic fracture	4.5% 1/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	4.5% 1/22 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.	— 0/0 • Serious and Other (Not Including Serious) Adverse Events were collected only for randomized participants through study completion, an average of 36 months. However, mortality is noted for one participant who passed away due to unrelated issues before being randomized. Non-ocular, Non-serious adverse events were not required to be collected per protocol. All-Cause Mortality and Non-Ocular Serious Adverse Events are reported at the participant level. Ocular Serious and Other (Not Including Serious) Adverse Events are presented with total number of eyes monitored/assessed as the at Risk population.

Additional Information

Neurotech, Sr. Director of Clinical Operations

Neurotech USA, Inc.

Phone: 629-333-5804

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place