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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 May 2022
• Main ID: NCT04123626
• Date of registration: 01/10/2019
• Prospective Registration: Yes
• Primary sponsor: ProQR Therapeutics
• Public title: 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 AURORA
• Scientific title: A Prospective First-In-Human Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa (adRP) Due to the P23H Mutation in the RHO Gene
• Date of first enrolment: October 7, 2019
• Target sample size: 11
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT04123626
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: ProQR Clinical Trial Manager
• Affiliation: ProQR Therapeutics

• Name: ProQR Medical Monitor
• Affiliation: ProQR Therapeutics

Key inclusion & exclusion criteria

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 mutation in the
RHO gene, based on genetic analysis.

5. A clear ocular media and adequate pupillary dilation to permit good quality fundus
imaging, as assessed by the Investigator.

Main Exclusion Criteria:

1. Presence of additional pathogenic mutations in genes (other than the P23H mutation in
the RHO gene) associated with inherited retinal degenerative diseases or syndromes,
based on genetic analysis (eg, Usher syndrome, Leber congenital amaurosis, etc).

2. Presence of any significant ocular or non-ocular disease/disorder (including
medication and laboratory test abnormalities) which, in the opinion of the
Investigator and with concurrence of the Medical Monitor, may either put the subject
at risk because of participation in the study, may influence the results of the study,
or the subject's ability to participate in the study.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Eye Diseases, Hereditary

Vision Tunnel

Retinal Dystrophies

Retinitis

Autosomal Dominant Retinitis Pigmentosa

Retinal Disease

Eye Diseases

Vision Disorders

Intervention(s)

Other: Sham procedure

Drug: QR-1123

Primary Outcome(s)

Incidence and Severity of non-ocular AEs [Time Frame: up to 12 months]

Incidence and Severity of ocular AEs [Time Frame: up to 12 months]

Secondary Outcome(s)

Assessment of systemic exposure after treatment with QR-1123 [Time Frame: up to 12 months]

Changes in Microperimetry [Time Frame: up to 12 months]

Changes in SD-OCT [Time Frame: up to 12 months]

Changes in Static VF [Time Frame: up to 12 months]

Changes in BCVA [Time Frame: up to 12 months]

Changes in DAC perimetry [Time Frame: up to 12 months]

Changes in FST [Time Frame: up to 12 months]

Changes in LLVA [Time Frame: up to 12 months]

Changes in Full-field ERG [Time Frame: up to 12 months]

Secondary ID(s)

PQ-1123-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors