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QR-1123

Phase 1

Autosomal Dominant Retinitis Pigmentosa | Small molecule | Ophthalmology |ProQR Therapeutics N.V.|Last Updated: May 6, 2022

Success Probability
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Market & Valuation
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Trial Design
RandomizedDouble-BlindCONTROLLEDDMC
Total Trials1
Total Enrollment11
FDA Designations
No designations recorded
Clinical trial landscape

QR-1123 · 1 trial · 8 indications

Phase 1 1
NCT04123626A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO GeneAutosomal Dominant Retinitis Pigmentosa
ACTIVE NOT_RECRUITING11 Analytics
PHASE1ACTIVE NOT_RECRUITING
A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene
Autosomal Dominant Retinitis PigmentosaUnlock trial analytics
Study Endpoints
Primary Endpoints
Incidence and Severity of ocular AEs
up to 12 months

Incidence and severity of ocular adverse events scored based on CTCAC in the study and fellow eye

Incidence and Severity of non-ocular AEs
up to 12 months

Incidence and severity of non-ocular adverse events scored based on CTCAC in the study and fellow eye

Secondary Endpoints
Changes in BCVA
up to 12 months
Changes in LLVA
up to 12 months
Changes in DAC perimetry
up to 12 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
QR-1123 Single dose - dose level 1EXPERIMENTALOpen label Single dose cohort: dose level 1
QR-1123 Single dose - dose level 2EXPERIMENTALOpen label Single dose cohort: dose level
QR-1123 Single dose - dose level 3EXPERIMENTALOpen label Single dose cohort: dose level 3
QR-1123 Single dose - dose level 4EXPERIMENTALOpen label Single dose cohort: dose level 4
QR-1123 Single dose - dose level 5EXPERIMENTALOpen label Single dose cohort: dose level 5
Repeat dose cohort 1EXPERIMENTALDouble-masked, randomized, sham controlled, Repeat dose cohort. Dose levels will be determined following DMC review of obtained safety and efficacy data.
Interventions
NameTypeDescription
QR-1123DRUGunilateral IVT injection
Sham procedureOTHERSham procedures (i.e. no penetration of the globe) closely mimic the active injection and serve to mask subjects to treatment assignment
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Eligibility Criteria
Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites5

Main Inclusion Criteria: 1. Male or female, ≥ 18 years of age. 2. Clinical presentation consistent with adRP, based on ophthalmic examinations. 3. Impairment on VF in the opinion of the Investigator, as determined by perimetry. 4. A molecular diagnosis of autosomal dominant form of RP with the P23H...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04123626primaryCompletionDate: changed
LOWMay 24, 2026NCT04123626studyFirstPostDate: changed