Trial Outcomes & Findings for Safety Study to Evaluate Bilateral CNTF Implants in Subjects With MacTel Type 2 (NCT NCT04729972)
NCT ID: NCT04729972
Last Updated: 2025-03-26
Results Overview
Assess the incidence and severity of adverse events (AEs) following bilateral ocular implantation of NT-501
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
33 participants
Primary outcome timeframe
6 months
Results posted on
2025-03-26
Participant Flow
Of the 33 enrolled subjects, 32 (97.0%) received NT-501; the exception was a subject whose surgery was canceled due to an event of arterial fibrillation that occurred prior to surgery and resulted in subject discontinuation. No subject had NT-501 removed from either eye during the study, and all 32 subjects who received NT-501 in this study completed the study.
Eligible subjects must have had a positive diagnosis of MacTel and previously received NT-501 in one eye prior to or in the Phase 1/2 extension study (NTMT-01/02E) or in 1 of the 2 Phase 3 studies (NTMT-03-A or NTMT-03-B). Subjects who participated in the Phase 3 study must have completed the Month 24 visit in that study and subjects enrolled in either the Phase 1/2 extension study or the Phase 3 study must have discontinued prior to enrollment in the current study.
Participant milestones
| Measure |
NT-501 in Fellow Eye
NT-501 was implanted on Day 0 in the fellow eye of subjects who received a single NT-501 in a previous study; subjects were followed for 6 months post-implantation of NT-501 in this study.
NT-501 CNTF implant: Single implantation of CNTF-secreting NT-501 device into fellow (untreated) eye
|
|---|---|
|
Overall Study
STARTED
|
33
|
|
Overall Study
NT-501 Implanted in Study Eye
|
32
|
|
Overall Study
COMPLETED
|
32
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
NT-501 in Fellow Eye
NT-501 was implanted on Day 0 in the fellow eye of subjects who received a single NT-501 in a previous study; subjects were followed for 6 months post-implantation of NT-501 in this study.
NT-501 CNTF implant: Single implantation of CNTF-secreting NT-501 device into fellow (untreated) eye
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
Baseline Characteristics
Safety Study to Evaluate Bilateral CNTF Implants in Subjects With MacTel Type 2
Baseline characteristics by cohort
| Measure |
NT-501 in Fellow Eye
n=32 Participants
NT-501 was implanted on Day 0 in the fellow eye of subjects who received a single NT-501 in a previous study; subjects were followed for 6 months post-implantation of NT-501 in this study.
NT-501 CNTF implant: Single implantation of CNTF-secreting NT-501 device into fellow (untreated) eye
|
|---|---|
|
Age, Customized
Age (Years)
|
63.0 years
STANDARD_DEVIATION 6.55 • n=5 Participants
|
|
Age, Customized
50 to < 60 years
|
9 Participants
n=5 Participants
|
|
Age, Customized
60 to < 70 years
|
17 Participants
n=5 Participants
|
|
Age, Customized
> or = to 70 years
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
24 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
30 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
28 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
BCVA (letters) for Study Eye
Study Eye
|
68.7 letters correctly read
STANDARD_DEVIATION 11.01 • n=5 Participants
|
|
BCVA (letters) for Study Eye
Fellow Eye
|
70.1 letters correctly read
STANDARD_DEVIATION 9.58 • n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsAssess the incidence and severity of adverse events (AEs) following bilateral ocular implantation of NT-501
Outcome measures
| Measure |
NT-501 in Fellow Eye
n=32 Participants
NT-501 was implanted on Day 0 in the fellow eye of subjects who received a single NT-501 in a previous study; subjects were followed for 6 months post-implantation of NT-501 in this study.
NT-501 CNTF implant: Single implantation of CNTF-secreting NT-501 device into fellow (untreated) eye
|
|---|---|
|
Frequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events
At least 1 TEAE
|
26 Participants
|
|
Frequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events
At least 1 ocular TEAE in study eye
|
26 Participants
|
|
Frequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events
At least 1 ocular TEAE in fellow eye
|
6 Participants
|
|
Frequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events
At least 1 nonocular TEAE
|
13 Participants
|
|
Frequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events
At least 1 treatment related ocular TEAE
|
26 Participants
|
|
Frequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events
At least 1 treatment related nonocular TEAE
|
0 Participants
|
|
Frequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events
At least 1 SAE
|
2 Participants
|
|
Frequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events
NT-501 removal from either eye
|
0 Participants
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: A neutralizing antibody test to NT-501-produced CNTF was performed to verify or refute CNTF antibody detected in one participant. The neutralizing antibody test refuted that the antibody detected was to NT-501 CNTF.
Incidence of positive serum levels of CNTF and immunogenicity for NT-501 with quantification of antibodies (Ab) to CNTF, neutralizing antibodies (NAb) to CNTF, Ab to NTC-201-6A cells, and Ab to mouse dihydrofolate reductase (mDHFR) at any time.
Outcome measures
| Measure |
NT-501 in Fellow Eye
n=32 Participants
NT-501 was implanted on Day 0 in the fellow eye of subjects who received a single NT-501 in a previous study; subjects were followed for 6 months post-implantation of NT-501 in this study.
NT-501 CNTF implant: Single implantation of CNTF-secreting NT-501 device into fellow (untreated) eye
|
|---|---|
|
Serum Levels of CNTF and Immunogenicity for NT-501
CNTF Positive
|
0 Participants
|
|
Serum Levels of CNTF and Immunogenicity for NT-501
Ab Positive to CNTF
|
1 Participants
|
|
Serum Levels of CNTF and Immunogenicity for NT-501
Neutralizing Ab to CNTF
|
0 Participants
|
|
Serum Levels of CNTF and Immunogenicity for NT-501
Ab to NTC
|
0 Participants
|
|
Serum Levels of CNTF and Immunogenicity for NT-501
ab to DHRF
|
0 Participants
|
Adverse Events
NT-501 in Fellow Eye
Serious events: 2 serious events
Other events: 26 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
NT-501 in Fellow Eye
n=32 participants at risk
NT-501 was implanted on Day 0 in the fellow eye of subjects who received a single NT-501 in a previous study; subjects were followed for 6 months post-implantation of NT-501 in this study.
NT-501 CNTF implant: Single implantation of CNTF-secreting NT-501 device into fellow (untreated) eye
|
|---|---|
|
Surgical and medical procedures
Device Extrusion
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Gastrointestinal disorders
Feces discolored
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
Other adverse events
| Measure |
NT-501 in Fellow Eye
n=32 participants at risk
NT-501 was implanted on Day 0 in the fellow eye of subjects who received a single NT-501 in a previous study; subjects were followed for 6 months post-implantation of NT-501 in this study.
NT-501 CNTF implant: Single implantation of CNTF-secreting NT-501 device into fellow (untreated) eye
|
|---|---|
|
Eye disorders
Eye pain
|
28.1%
9/32 • Number of events 9 • Through 6 months post implant
|
|
Eye disorders
Ocular discomfort
|
28.1%
9/32 • Number of events 9 • Through 6 months post implant
|
|
Eye disorders
Conjunctival haemorrhage
|
25.0%
8/32 • Number of events 8 • Through 6 months post implant
|
|
Eye disorders
Eye irritation
|
12.5%
4/32 • Number of events 4 • Through 6 months post implant
|
|
Eye disorders
Dry eye
|
6.2%
2/32 • Number of events 2 • Through 6 months post implant
|
|
Eye disorders
Anterior chamber cell
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Anterior chamber flare
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Cataract
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Cataract subcapsular
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Conjunctival hyperaemia
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Conjunctival oedema
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Conjunctivitis allergic
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Corneal pigmentation
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Delayed dark adaptation
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Epiretinal membrane
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Eye pruritus
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Foreign body sensation in eyes
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Halo vision
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Iris transillumination defect
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Iritis
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Photophobia
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Photopsia
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Punctate keratitis
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Retinal haemorrhage
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Vitreous detachment
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Eye disorders
Vitreous haemorrhage
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Immune system disorders
Allergy to sutures
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Infections and infestations
Conjunctivitis
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Injury, poisoning and procedural complications
Corneal abrasion
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Investigations
Intraocular pressure decreased
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Pyogenic granuloma
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
|
Product Issues
Device extrusion
|
3.1%
1/32 • Number of events 1 • Through 6 months post implant
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place