Trial Outcomes & Findings for Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste) (NCT NCT05176717)
NCT ID: NCT05176717
Last Updated: 2024-08-07
Results Overview
Change from baseline in mean sensitivity (based on static perimetry) at 12 months of treatment versus sham-procedure
Recruitment status
TERMINATED
Study phase
PHASE2/PHASE3
Target enrollment
5 participants
Primary outcome timeframe
12 months
Results posted on
2024-08-07
Participant Flow
Participant milestones
| Measure |
QR-421a 180/60 µg
180 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter
QR-421a: RNA antisense oligonucleotide for intravitreal injection
|
QR-421a 60/60 µg
60 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter
QR-421a: RNA antisense oligonucleotide for intravitreal injection
|
Sham-procedure
Sham-procedure (no experimental drug administered) on Day 1, Month 3 and every 6 months thereafter
Sham-procedure: Sham-procedure (no experimental drug administered)
|
|---|---|---|---|
|
Overall Study
STARTED
|
1
|
2
|
2
|
|
Overall Study
COMPLETED
|
0
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
1
|
2
|
2
|
Reasons for withdrawal
| Measure |
QR-421a 180/60 µg
180 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter
QR-421a: RNA antisense oligonucleotide for intravitreal injection
|
QR-421a 60/60 µg
60 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter
QR-421a: RNA antisense oligonucleotide for intravitreal injection
|
Sham-procedure
Sham-procedure (no experimental drug administered) on Day 1, Month 3 and every 6 months thereafter
Sham-procedure: Sham-procedure (no experimental drug administered)
|
|---|---|---|---|
|
Overall Study
Sponsor Decision
|
1
|
2
|
2
|
Baseline Characteristics
Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)
Baseline characteristics by cohort
| Measure |
QR-421a 180/60 µg
n=1 Participants
180 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter
QR-421a: RNA antisense oligonucleotide for intravitreal injection
|
QR-421a 60/60 µg
n=2 Participants
60 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter
QR-421a: RNA antisense oligonucleotide for intravitreal injection
|
Sham-procedure
n=2 Participants
Sham-procedure (no experimental drug administered) on Day 1, Month 3 and every 6 months thereafter
Sham-procedure: Sham-procedure (no experimental drug administered)
|
Total
n=5 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
13 Years
n=99 Participants
|
34 Years
STANDARD_DEVIATION 11.3 • n=107 Participants
|
27 Years
STANDARD_DEVIATION 19.8 • n=206 Participants
|
27 Years
STANDARD_DEVIATION 14.3 • n=7 Participants
|
|
Age, Customized
Age Group · Adult (>= 18 years)
|
0 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
3 Participants
n=7 Participants
|
|
Age, Customized
Age Group · Pediatric (<18 years)
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
2 Participants
n=7 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
5 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
4 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
5 Participants
n=7 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Genotype - Heterozygous
|
1 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
5 Participants
n=7 Participants
|
|
Phenotype
Syndromic
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
3 Participants
n=7 Participants
|
|
Phenotype
Non-Syndromic
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
2 Participants
n=7 Participants
|
|
Baseline Best Corrected Visual Acuity (BCVA) - Treated Eye
|
73 Letters
n=99 Participants
|
78.5 Letters
STANDARD_DEVIATION 0.7 • n=107 Participants
|
74 Letters
STANDARD_DEVIATION 1.4 • n=206 Participants
|
75.6 Letters
STANDARD_DEVIATION 2.8 • n=7 Participants
|
|
Baseline Best Corrected Visual Acuity (BCVA) - Contralateral Eye
|
74.0 Letters
n=99 Participants
|
83.5 Letters
STANDARD_DEVIATION 6.4 • n=107 Participants
|
74 Letters
STANDARD_DEVIATION 8.5 • n=206 Participants
|
77.8 Letters
STANDARD_DEVIATION 7.4 • n=7 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: Study prematurely terminated due to sponsor decision for reasons unrelated to safety
Change from baseline in mean sensitivity (based on static perimetry) at 12 months of treatment versus sham-procedure
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 27 monthsEllipzoid Zone area (EZ) as measured by Spectral Domain optical coherence tomography SD-OCT
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 27 monthsChange from baseline in Best Corrected Visual Acuity (BCVA)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 27 monthsChange from baseline in Spectral domain optical coherence tomography (SD-OCT) (other measures)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 27 monthsChange from baseline in Low Luminance Visual Acuity (LLVA) using the ETDRS vision chart
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 27 monthsChange from baseline in other measures of static perimetry on the Octopus 900 as assessed by a central reading center
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 27 monthsChange from baseline in Full-field Stimulus Threshold (FST) on the Diagnosys FST as assessed by a central reading center
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 27 monthsChange from baseline in PRO measure as assessed by Michigan Retinal Degeneration Questionnaire (MRDQ)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 27 monthsChange from baseline in PRO measures as assessed by Patient Global Impressions of Severity (PGI-S)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 27 monthsChange from baseline in PRO measures as assessed by Patient Global Impressions of Change (PGI-C)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 27 monthsOcular and non-ocular adverse events (AEs)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 27 monthsMaximum concentration (Cmax) of QR-421a in serum
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: 27 monthsChange from baseline in mobility course score
Outcome measures
Outcome data not reported
Adverse Events
QR-421a 180/60 µg
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
QR-421a 60/60 µg
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Sham-procedure
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
QR-421a 180/60 µg
n=1 participants at risk
180 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter
QR-421a: RNA antisense oligonucleotide for intravitreal injection
|
QR-421a 60/60 µg
n=2 participants at risk
60 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter
QR-421a: RNA antisense oligonucleotide for intravitreal injection
|
Sham-procedure
n=2 participants at risk
Sham-procedure (no experimental drug administered) on Day 1, Month 3 and every 6 months thereafter
Sham-procedure: Sham-procedure (no experimental drug administered)
|
|---|---|---|---|
|
Eye disorders
Conjunctival haemorrhage
|
0.00%
0/1 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
100.0%
2/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
50.0%
1/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
|
Eye disorders
Eye irritation
|
0.00%
0/1 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
50.0%
1/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
0.00%
0/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
|
Eye disorders
Photophobia
|
100.0%
1/1 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
0.00%
0/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
0.00%
0/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
|
Eye disorders
Retinal pigmentation
|
0.00%
0/1 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
50.0%
1/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
0.00%
0/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
|
Eye disorders
Vitreous detachment
|
0.00%
0/1 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
50.0%
1/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
0.00%
0/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
|
Eye disorders
Vitreous floaters
|
0.00%
0/1 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
50.0%
1/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
0.00%
0/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
|
Eye disorders
Lacrimation increased
|
100.0%
1/1 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
0.00%
0/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
0.00%
0/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
100.0%
1/1 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
0.00%
0/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
0.00%
0/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
|
Skin and subcutaneous tissue disorders
Erythema
|
100.0%
1/1 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
0.00%
0/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
0.00%
0/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
|
Eye disorders
Eye pain
|
0.00%
0/1 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
50.0%
1/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
0.00%
0/2 • 7 months, 18 days - Study was terminated prematurely, therefore follow-up maximum range is from the Screening visit to the End of Study visit, for each participant.
Any event/condition noted once the subject receives their first dose of study drug until the End of study visit (30 days +/- 14 days post last dose of study drug).
|
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