Trial Outcomes & Findings for An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (NCT NCT05085964)

Last Updated: 2024-08-07

Results Overview

Number of subjects with ocular treatment emergent adverse events (TEAEs) in the contralateral eye (CE) is presented. Frequency of individual TEAEs by system organ class and preferred term is presented in the safety section and clinical trial summary report. Time frame of reporting is the maximum followup period from first subject first visit to last end of study visit.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

21 participants

Primary outcome timeframe

1 year, 1 month

Results posted on

2024-08-07

Participant Flow

In total 21 participants were enrolled: 19 participants were enrolled after they completed the previous PQ-421a-001 (Stellar) study and 2 participants were enrolled after premature termination of the PQ-421a-004 (Celeste) study.

Participant milestones

Participant milestones
Measure	Ultevursen 180ug/60ug (N=21) 180ug loading dose of QR-421a will be first administered to the contralateral (fellow) eye (as defined in the preceding study), and 60ug will be repeated every 6 months.	Ultevursen 60ug/60ug (N=0) 60ug loading dose of QR-421a will be first administered to the contralateral (fellow) eye (as defined in the preceding study), and 60ug will be repeated every 6 months.
Overall Study STARTED	21	0
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	21	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Ultevursen 180ug/60ug (N=21) 180ug loading dose of QR-421a will be first administered to the contralateral (fellow) eye (as defined in the preceding study), and 60ug will be repeated every 6 months.	Ultevursen 60ug/60ug (N=0) 60ug loading dose of QR-421a will be first administered to the contralateral (fellow) eye (as defined in the preceding study), and 60ug will be repeated every 6 months.
Overall Study Premature termination of study	21	0

Baseline Characteristics

An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ultevursen 180ug/60ug n=21 Participants 180ug loading dose of QR-421a will be first administered to the contralateral (fellow) eye (as defined in the preceding study), and 60ug will be repeated every 6 months.	Ultevursen 60ug/60ug 60ug loading dose of QR-421a will be first administered to the contralateral (fellow) eye (as defined in the preceding study), and 60ug will be repeated every 6 months.	Total n=21 Participants Total of all reporting groups
Age, Continuous	47.7 years STANDARD_DEVIATION 13.5 • n=39 Participants	—	47.7 years STANDARD_DEVIATION 13.5 • n=35 Participants
Sex: Female, Male Female	10 Participants n=39 Participants	—	10 Participants n=35 Participants
Sex: Female, Male Male	11 Participants n=39 Participants	—	11 Participants n=35 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=39 Participants	—	1 Participants n=35 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	15 Participants n=39 Participants	—	15 Participants n=35 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	5 Participants n=39 Participants	—	5 Participants n=35 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) Asian	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) Black or African American	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) White	16 Participants n=39 Participants	0 Participants n=41 Participants	16 Participants n=35 Participants
Race (NIH/OMB) More than one race	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) Unknown or Not Reported	5 Participants n=39 Participants	0 Participants n=41 Participants	5 Participants n=35 Participants
Region of Enrollment Canada	2 Participants n=39 Participants	0 Participants n=41 Participants	2 Participants n=35 Participants
Region of Enrollment United States	11 Participants n=39 Participants	0 Participants n=41 Participants	11 Participants n=35 Participants
Region of Enrollment France	8 Participants n=39 Participants	0 Participants n=41 Participants	8 Participants n=35 Participants

PRIMARY outcome

Timeframe: 1 year, 1 month

Population: All participants who were enrolled and received at least 1 dose of ultevursen.

Outcome measures

Outcome measures
Measure	Ultevursen 180ug/60ug n=21 Participants 180ug loading dose of QR-421a will be first administered to the contralateral (fellow) eye (as defined in the preceding study), and 60ug will be repeated every 6 months.	Ultevursen 60ug/60ug 60ug loading dose of QR-421a will be first administered to the contralateral (fellow) eye (as defined in the preceding study), and 60ug will be repeated every 6 months.
Ocular Adverse Events (AEs) Contralateral Eye (CE) - TEAEs	11 Participants	0 Participants
Ocular Adverse Events (AEs) No TEAEs reported	10 Participants	—

PRIMARY outcome

Timeframe: 1 year, 1 month

Population: All participants who were enrolled and received at least 1 dose of ultevursen.

Number of subjects with non-ocular treatment emergent adverse events (TEAEs) is presented. Frequency of individual TEAEs by system organ class and preferred term is presented in the safety section and clinical trial summary report. Time frame of reporting is the maximum follow up period from first subject first visit to last end of study visit.

Outcome measures

Outcome measures
Measure	Ultevursen 180ug/60ug n=21 Participants 180ug loading dose of QR-421a will be first administered to the contralateral (fellow) eye (as defined in the preceding study), and 60ug will be repeated every 6 months.	Ultevursen 60ug/60ug 60ug loading dose of QR-421a will be first administered to the contralateral (fellow) eye (as defined in the preceding study), and 60ug will be repeated every 6 months.
Non-ocular Adverse Events (AEs)	11 Participants	0 Participants

Adverse Events

Ultevursen 180ug/60ug

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Ultevursen 60ug/60ug

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Ultevursen 180ug/60ug n=21 participants at risk 180 ug loading dose of QR-421a will be first administered to the contralateral (fellow) eye (as defined in the preceding study), and 60ug will be repeated every 6 months.	Ultevursen 60ug/60ug 60 ug loading dose of QR-421a will be first administered to the contralateral (fellow) eye (as defined in the preceding study), and 60ug will be repeated every 6 months.
Eye disorders Conjunctival haemorrhage	38.1% 8/21 • 1 year,1 month All ocular (CE) and non-ocular TEAEs reported in subjects that received at least 1 dose of study drug.	— 0/0 • 1 year,1 month All ocular (CE) and non-ocular TEAEs reported in subjects that received at least 1 dose of study drug.
Eye disorders Eye pain	33.3% 7/21 • 1 year,1 month All ocular (CE) and non-ocular TEAEs reported in subjects that received at least 1 dose of study drug.	— 0/0 • 1 year,1 month All ocular (CE) and non-ocular TEAEs reported in subjects that received at least 1 dose of study drug.
Eye disorders Conjunctival hyperaemia	9.5% 2/21 • 1 year,1 month All ocular (CE) and non-ocular TEAEs reported in subjects that received at least 1 dose of study drug.	— 0/0 • 1 year,1 month All ocular (CE) and non-ocular TEAEs reported in subjects that received at least 1 dose of study drug.
Infections and infestations COVID-19	19.0% 4/21 • 1 year,1 month All ocular (CE) and non-ocular TEAEs reported in subjects that received at least 1 dose of study drug.	— 0/0 • 1 year,1 month All ocular (CE) and non-ocular TEAEs reported in subjects that received at least 1 dose of study drug.
Infections and infestations Nasopharyngitis	9.5% 2/21 • 1 year,1 month All ocular (CE) and non-ocular TEAEs reported in subjects that received at least 1 dose of study drug.	— 0/0 • 1 year,1 month All ocular (CE) and non-ocular TEAEs reported in subjects that received at least 1 dose of study drug.

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