Trial Outcomes & Findings for RESCUE and REVERSE Long-term Follow-up (NCT NCT03406104)
NCT ID: NCT03406104
Last Updated: 2025-09-09
Results Overview
Number of Eyes with Ocular Adverse events related to study treatment or study procedures as judged by the investigator reported from year 2 to year 5 post treatment
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
62 participants
Primary outcome timeframe
from year 2 to year 5 post treatment
Results posted on
2025-09-09
Participant Flow
Participant milestones
| Measure |
All Patient Treated in RECUE and REVERSE Studies
all patients previously treated in RESCUE and REVERSE studies entered in this long term follow up study.
|
|---|---|
|
Overall Study
STARTED
|
62
|
|
Overall Study
COMPLETED
|
55
|
|
Overall Study
NOT COMPLETED
|
7
|
Reasons for withdrawal
| Measure |
All Patient Treated in RECUE and REVERSE Studies
all patients previously treated in RESCUE and REVERSE studies entered in this long term follow up study.
|
|---|---|
|
Overall Study
Lost to Follow-up
|
2
|
|
Overall Study
Withdrawal by Subject
|
2
|
|
Overall Study
due to Covid patient did not want to travel
|
1
|
|
Overall Study
Death
|
2
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Lenadogene Nolparvovec and Sham All Participants
n=62 Participants
All participants who were enrolled and received both study treatments, GS010 and Sham. All participants in the study received both GS010 and the sham procedure simultaneously. Participants were randomly assigned to receive GS010 in either the right or left eye. The same participants also received the sham procedure in the eye not assigned to GS010, at the same study visit.
GS010: Either the right or left eye received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 µL. The injection was performed in the vitreous humor under local anesthesia.
Sham procedure: Either the right or left eye (the eye not randomly assigned to GS010) received the sham procedure. One single sham IVT injection was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.
|
|---|---|
|
Age, Categorical
<=18 years
|
7 Participants
n=62 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
55 Participants
n=62 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=62 Participants
|
|
Age, Continuous
|
35.9 years
STANDARD_DEVIATION 15.3 • n=62 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=62 Participants
|
|
Sex: Female, Male
Male
|
49 Participants
n=62 Participants
|
|
Region of Enrollment
United States
|
31 participants
n=62 Participants
|
|
Region of Enrollment
Italy
|
1 participants
n=62 Participants
|
|
Region of Enrollment
United Kingdom
|
5 participants
n=62 Participants
|
|
Region of Enrollment
France
|
16 participants
n=62 Participants
|
|
Region of Enrollment
Germany
|
9 participants
n=62 Participants
|
|
Best Corrected Visual Acuity
|
1.40 LogMAR
STANDARD_DEVIATION 0.50 • n=62 Participants
|
PRIMARY outcome
Timeframe: from year 2 to year 5 post treatmentNumber of Eyes with Ocular Adverse events related to study treatment or study procedures as judged by the investigator reported from year 2 to year 5 post treatment
Outcome measures
| Measure |
GS010 Treated Eyes
n=62 eyes
All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.
The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.
|
Sham Treated Eyes
n=62 eyes
All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.
The same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.
|
|---|---|---|
|
Ocular Adverse Events (AEs)
|
5 eyes
|
1 eyes
|
SECONDARY outcome
Timeframe: Nadir to 5 Years post-treatment, Assessed at Baseline in RESCUE and REVERSE studies to year 5 post-treatmentPopulation: All efficacy analyses were conducted on the modified intent-to-treat population and additionally in subset populations, as appropriate.The modified intent-to-treat (mITT) population consisted of all subjects who received the IMP in RESCUE or REVERSE Phase III studies, consented to be enrolled in this LTFU study, and provided visual acuity data at Visit 2 (Year 2.5).
Change from Nadir to 5 Years post-treatment, Assessed at Baseline in RESCUE and REVERSE studies to year 5 post-treatment, expressed in LogMAR. On chart visual acuity: Visual acuity inferior or equal to LogMAR +1.6 Off chart visual acuity: Visual acuity superior to LogMar +1.7 Normal vision LogMar: 0 and less than 0
Outcome measures
| Measure |
GS010 Treated Eyes
n=62 eyes
All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.
The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.
|
Sham Treated Eyes
n=62 eyes
All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.
The same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.
|
|---|---|---|
|
Visual Acuity
|
-0.44 LogMar
Standard Deviation 0.46
|
-0.39 LogMar
Standard Deviation 0.36
|
SECONDARY outcome
Timeframe: Nadir to 5 Years post-treatment, Assessed at Baseline in RESCUE and REVERSE studies to year 5 post-treatmentPopulation: All efficacy analyses were conducted on the modified intent-to-treat population and additionally in subset populations, as appropriate.The modified intent-to-treat (mITT) population consisted of all subjects who received the IMP in RESCUE or REVERSE Phase III studies, consented to be enrolled in this LTFU study, and provided visual acuity data at Visit 2 (Year 2.5).
CRR clinically relevant recovery from Nadir defined as i/ for eyes on chart at Nadir, an improvement of at least -0.2 LogMar from Nadir and ii/ for eyes off chart at Nadir eyes that became on chart. Off chart visual acuity expressed in LogMar: more than +1.7 On chart visual acuity expressed in LogMar: less than 1.6 Normal vision LogMar: 0 and less than 0
Outcome measures
| Measure |
GS010 Treated Eyes
n=62 eyes
All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.
The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.
|
Sham Treated Eyes
n=62 eyes
All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.
The same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.
|
|---|---|---|
|
Responder Analysis: Clinically Relevant Recovery
|
60 percentage of eye
|
66 percentage of eye
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Year 5 post-treatmentPopulation: All efficacy analyses were conducted on the modified intent-to-treat population and additionally in subset populations, as appropriate.The modified intent-to-treat (mITT) population consisted of all subjects who received the IMP in RESCUE or REVERSE Phase III studies, consented to be enrolled in this LTFU study, and provided visual acuity data at Visit 2 (Year 2.5).
Definition: Visual acuity inferior or equal to LogMAR +1.6 at 5 Years post treatment
Outcome measures
| Measure |
GS010 Treated Eyes
n=62 eyes
All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.
The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.
|
Sham Treated Eyes
n=62 eyes
All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.
The same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.
|
|---|---|---|
|
Eyes on Chart
|
76 percentage of eyes
|
79 percentage of eyes
|
Adverse Events
Subjects
Serious events: 8 serious events
Other events: 19 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Subjects
n=62 participants at risk
All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.
The same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.
|
|---|---|
|
Injury, poisoning and procedural complications
hip fracture
|
3.2%
2/62 • Number of events 2 • from 2 to 5 years
|
|
Musculoskeletal and connective tissue disorders
humerus fracture
|
1.6%
1/62 • Number of events 1 • from 2 to 5 years
|
|
Cardiac disorders
cardiac arrest
|
1.6%
1/62 • Number of events 1 • from 2 to 5 years
|
|
Gastrointestinal disorders
ileus
|
1.6%
1/62 • Number of events 1 • from 2 to 5 years
|
|
Musculoskeletal and connective tissue disorders
lower limb fracture
|
1.6%
1/62 • Number of events 1 • from 2 to 5 years
|
|
Injury, poisoning and procedural complications
limb injury
|
1.6%
1/62 • Number of events 1 • from 2 to 5 years
|
|
Nervous system disorders
cerebral haemorrage
|
1.6%
1/62 • Number of events 1 • from 2 to 5 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
glioblastoma multiforme
|
1.6%
1/62 • Number of events 1 • from 2 to 5 years
|
|
Infections and infestations
appenditis perforated
|
1.6%
1/62 • Number of events 1 • from 2 to 5 years
|
|
Infections and infestations
pneumonia
|
1.6%
1/62 • Number of events 1 • from 2 to 5 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
neoplasm progression
|
1.6%
1/62 • Number of events 1 • from 2 to 5 years
|
Other adverse events
| Measure |
Subjects
n=62 participants at risk
All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.
The same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.
|
|---|---|
|
Eye disorders
Cataract
|
9.7%
6/62 • Number of events 13 • from 2 to 5 years
|
|
Investigations
Gamma-Glutamyltransferase Increased
|
8.1%
5/62 • Number of events 5 • from 2 to 5 years
|
|
Eye disorders
Intraocular Pressure Increased
|
6.5%
4/62 • Number of events 8 • from 2 to 5 years
|
|
Infections and infestations
Covid-19
|
6.5%
4/62 • Number of events 4 • from 2 to 5 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place