Trial Outcomes & Findings for Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene (NCT NCT03140969)

NCT ID: NCT03140969

Last Updated: 2024-10-15

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

11 participants

Primary outcome timeframe

1 year

Results posted on

2024-10-15

Participant Flow

Participant milestones

Participant milestones
Measure	QR-110 Low Dose One 160 μg loading dose, followed by up to three 80 μg maintenance doses administered every 3 months, for up to 4 doses per subject.	QR-110 Mid Dose One 320 μg loading dose, followed by up to three 160 μg maintenance doses administered every 3 months, for up to 4 doses per subject.
Overall Study STARTED	6	5
Overall Study COMPLETED	6	5
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	QR-110 Low Dose n=6 Participants 160/80 μg Cohort	QR-110 Mid Dose n=5 Participants 320/160 μg Cohort	Total n=11 Participants Total of all reporting groups
Age, Continuous	23.7 years STANDARD_DEVIATION 15.06 • n=5 Participants	19.4 years STANDARD_DEVIATION 6.88 • n=7 Participants	21.7 years STANDARD_DEVIATION 11.71 • n=5 Participants
Age, Customized 6 to < 18 years	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Age, Customized 18 to < 65 years	3 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	4 Participants n=7 Participants	6 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	1 Participants n=7 Participants	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants	5 Participants n=7 Participants	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants	5 Participants n=7 Participants	11 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Outcome measures

Outcome measures
Measure	QR-110 Low Dose n=6 Participants 160/80 μg Cohort	QR-110 Mid Dose n=5 Participants 320/160 μg Cohort
Frequency and Severity of Ocular Adverse Events in the Treatment and Contralateral Eyes	6 Participants	5 Participants

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome measures
Measure	QR-110 Low Dose n=6 Participants 160/80 μg Cohort	QR-110 Mid Dose n=5 Participants 320/160 μg Cohort
Frequency and Severity of Non-ocular Adverse Events	6 Participants	5 Participants

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome measures
Measure	QR-110 Low Dose n=6 Participants 160/80 μg Cohort	QR-110 Mid Dose n=5 Participants 320/160 μg Cohort
Change in Best-corrected Visual Acuity (BCVA) Baseline (Absolute Values)	2.597 logMAR Standard Deviation 1.2738	2.990 logMAR Standard Deviation 1.4153
Change in Best-corrected Visual Acuity (BCVA) Changes from Baseline - Visit 8 (M3)	-0.813 logMAR Standard Deviation 1.0073	-0.126 logMAR Standard Deviation 0.2165
Change in Best-corrected Visual Acuity (BCVA) Changes from Baseline - Visit 13 (M6)	-0.903 logMAR Standard Deviation 1.0189	-0.122 logMAR Standard Deviation 0.1801
Change in Best-corrected Visual Acuity (BCVA) Changes from Baseline - Visit 17 (M9)	-0.923 logMAR Standard Deviation 1.0417	0.386 logMAR Standard Deviation 0.9610
Change in Best-corrected Visual Acuity (BCVA) Changes from Baseline - Visit 21 (M12)	-0.927 logMAR Standard Deviation 1.0492	-0.106 logMAR Standard Deviation 0.1545

SECONDARY outcome

Timeframe: 1 year

Average Red Light Score

Outcome measures

Outcome measures
Measure	QR-110 Low Dose n=6 Participants 160/80 μg Cohort	QR-110 Mid Dose n=4 Participants 320/160 μg Cohort
Change in Full-field Stimulus Test (FST)	-0.7 log cd/m2 Standard Deviation 0.33	-1.2 log cd/m2 Standard Deviation 0.82

SECONDARY outcome

Timeframe: 1 year

Average Blue Light Score

Outcome measures

Outcome measures
Measure	QR-110 Low Dose n=6 Participants 160/80 μg Cohort	QR-110 Mid Dose n=4 Participants 320/160 μg Cohort
Change in Full-field Stimulus Test (FST)	-0.6 log cd/m2 Standard Deviation 0.76	-0.9 log cd/m2 Standard Deviation 0.82

Adverse Events

QR-110 Low Dose

Serious events: 2 serious events

Other events: 6 other events

Deaths: 0 deaths

QR-110 Mid Dose

Serious events: 4 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	QR-110 Low Dose n=6 participants at risk 160/80 μg Cohort	QR-110 Mid Dose n=5 participants at risk 320/160 μg Cohort
Eye disorders Cataract	33.3% 2/6 • From the start of the study through to study completion (approx 1 year 11 months)	80.0% 4/5 • From the start of the study through to study completion (approx 1 year 11 months)

Other adverse events

Additional Information

Zuhal Butuner - Chief Medical Officer

Sepul Bio

Phone: (905) 599 7887

Email: [email protected]

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

Measure	QR-110 Low Dose n=6 participants at risk 160/80 μg Cohort	QR-110 Mid Dose n=5 participants at risk 320/160 μg Cohort
Eye disorders Conjunctival haemorrhage	66.7% 4/6 • From the start of the study through to study completion (approx 1 year 11 months)	80.0% 4/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Cataract	50.0% 3/6 • From the start of the study through to study completion (approx 1 year 11 months)	80.0% 4/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Lenticular opacities	16.7% 1/6 • From the start of the study through to study completion (approx 1 year 11 months)	60.0% 3/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Conjunctival hyperaemia	16.7% 1/6 • From the start of the study through to study completion (approx 1 year 11 months)	40.0% 2/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Retinal degeneration	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	40.0% 2/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Anterior chamber cell	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Cataract cortical	16.7% 1/6 • From the start of the study through to study completion (approx 1 year 11 months)	0.00% 0/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Cataract subcapsular	16.7% 1/6 • From the start of the study through to study completion (approx 1 year 11 months)	0.00% 0/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Conjunctival oedema	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Corneal lesion	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Cystoid macular oedema	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Diplopia	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Dry eye	16.7% 1/6 • From the start of the study through to study completion (approx 1 year 11 months)	0.00% 0/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Eyelid cyst	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Eyelid oedema	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Hypotony of eye	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Injection site haemorrhage	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Lens disorder	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Metamorphopsia	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Post procedural complication	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Posterior capsule opacification	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Retinal cyst	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Vitreal cells	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)
Eye disorders Vitreous opacities	0.00% 0/6 • From the start of the study through to study completion (approx 1 year 11 months)	20.0% 1/5 • From the start of the study through to study completion (approx 1 year 11 months)