Trial Outcomes & Findings for Extension Study to Study PQ-110-001 (NCT03140969) (NCT NCT03913130)
NCT ID: NCT03913130
Last Updated: 2024-10-15
Results Overview
Frequency of ocular adverse events (AEs)
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
9 participants
Primary outcome timeframe
24 months
Results posted on
2024-10-15
Participant Flow
Participant milestones
| Measure |
Drug QR-110
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
QR-110: First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
|
|---|---|
|
Overall Study
STARTED
|
9
|
|
Overall Study
COMPLETED
|
8
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Drug QR-110
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
QR-110: First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
|
|---|---|
|
Overall Study
Sponsor Decision
|
1
|
Baseline Characteristics
Extension Study to Study PQ-110-001 (NCT03140969)
Baseline characteristics by cohort
| Measure |
Drug QR-110
n=9 Participants
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
QR-110: First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
|
|---|---|
|
Age, Customized
Age (Mean)
|
24.7 Years
STANDARD_DEVIATION 12.7 • n=99 Participants
|
|
Age, Customized
Age (Median)
|
23.0 Years
n=99 Participants
|
|
Age, Customized
<18 years
|
3 Participants
n=99 Participants
|
|
Age, Customized
>= 18 Years
|
6 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Weight (Mean)
|
64.26 kg
STANDARD_DEVIATION 23.19 • n=99 Participants
|
|
Weight (Median)
|
61.55 kg
n=99 Participants
|
|
Height (Mean)
|
164.9 cm
STANDARD_DEVIATION 11.8 • n=99 Participants
|
|
Height (Median)
|
166.3 cm
n=99 Participants
|
|
BMI (Mean)
|
23.02 kg/m2
STANDARD_DEVIATION 5.20 • n=99 Participants
|
|
BMI (Median)
|
21.89 kg/m2
n=99 Participants
|
|
Genotype
Compound Heterozygous
|
8 Participants
n=99 Participants
|
|
Genotype
Homozygous
|
1 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 24 monthsFrequency of ocular adverse events (AEs)
Outcome measures
| Measure |
Drug QR-110
n=9 Participants
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
QR-110: First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
|
|---|---|
|
Frequency of Ocular AEs
|
7 Participants
|
PRIMARY outcome
Timeframe: 24 monthsFrequency of non-ocular AEs
Outcome measures
| Measure |
Drug QR-110
n=9 Participants
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
QR-110: First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
|
|---|---|
|
Frequency of Non-ocular AEs
|
6 Participants
|
SECONDARY outcome
Timeframe: 24 monthsChange in Best Corrected Visual Acuity (BCVA) in First Treated Eye
Outcome measures
| Measure |
Drug QR-110
n=7 Participants
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
QR-110: First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
|
|---|---|
|
Change in BCVA in First Treated Eye
|
0.03 logMAR
Standard Deviation 0.17
|
SECONDARY outcome
Timeframe: 24 monthsChange in Mobility course score
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 monthsChange in photoreceptor outer segment layer thickness by Optical Coherence Tomography (OCT)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 monthsChange in Oculomotor Instability (OCI)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 monthsChange in Full-Field Stimulus Testing (FST) - blue stimuli
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 monthsChange in Full-Field Stimulus Testing (FST) - red stimuli
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 monthsChange in Visual Function Questionnaire-25 (VFQ-25) score (adult subjects)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 monthsChange in Cardiff Visual Ability Questionnaire for Children (CVAQC) score (pediatric subjects)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 monthsChange in Pupillary Light Reflex (PLR) (latency and amplitude)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 monthsChange in Near Infrared AutoFluorescence (NIRAF)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 monthsChange in Best Corrected Visual Acuity (BCVA) in Treated Contralateral Eye
Outcome measures
| Measure |
Drug QR-110
n=5 Participants
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
QR-110: First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
|
|---|---|
|
Change in BCVA in Treated Contralateral Eye
|
-0.11 logMAR
Standard Deviation 0.23
|
SECONDARY outcome
Timeframe: 24 monthsChange in Best Corrected Visual Acuity (BCVA) in Non-Treated Contralateral Eye
Outcome measures
| Measure |
Drug QR-110
n=2 Participants
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
QR-110: First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
|
|---|---|
|
Change in BCVA in Non-Treated Contralateral Eye
|
0.05 logMAR
Standard Deviation 0.21
|
Adverse Events
Drug QR-110
Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Drug QR-110
n=9 participants at risk
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
QR-110: First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
|
|---|---|
|
Injury, poisoning and procedural complications
Lumbar vertebral fracture
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
Other adverse events
| Measure |
Drug QR-110
n=9 participants at risk
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
QR-110: First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
|
|---|---|
|
Eye disorders
Cataract
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Cataract cortical
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Cataract subcapsular
|
22.2%
2/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Conjunctival haemorrhage
|
22.2%
2/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Cystoid macular oedema
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Dry eye
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Eye pain
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Eye pruritus
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Lacrimation increased
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Lenticular opacities
|
22.2%
2/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Posterior capsule opacification
|
33.3%
3/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Retinal cyst
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Retinal degeneration
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Visual acuity reduced
|
33.3%
3/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Infections and infestations
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Infections and infestations
Blebitis
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Surgical and medical procedures
Posterior lens capsulotomy
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
General disorders
Fatigue
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Immune system disorders
Seasonal allergy
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Infections and infestations
Cystitis
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Infections and infestations
Gastroenteritis
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Infections and infestations
Influenza
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Infections and infestations
Paronychia
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Injury, poisoning and procedural complications
Contusion
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Investigations
Coronavirus test positive
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Nervous system disorders
Headache
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Nervous system disorders
Petit mal epilepsy
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
22.2%
2/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
22.2%
2/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Surgical and medical procedures
Dermabrasion
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Surgical and medical procedures
Wisdom teeth removal
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
|
Eye disorders
Diplopia
|
11.1%
1/9 • Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.
In the first treated eye all events/conditions will be captured as an AE as treatment continues from the preceding study.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Institution shall be free to publish, present, or use any Data and results arising out of its performance of the protocol. At least 30 days prior to submission for publication, institution shall submit to Sponsor for review and comment any proposed oral or written publication. Institution will consider any such comments in good faith but is under no obligation to incorporate Sponsor's suggestions. The review period for abstracts or poster presentations shall be 30 days.
- Publication restrictions are in place
Restriction type: OTHER