A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Advanced Vision Loss

A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Advanced Vision Loss - Sirius (PQ-421a-003)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON52147

Enrollment

Registered

2022-01-12

Start date

2022-07-15

Completion date

Unknown

Last updated

2025-08-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

progressive pigmentary retinopathy rod-cone dystrophy RP

Interventions

The current study will evaluate 2 dose levels. Subjects will receive loading dose administered on Day 1, and maintenance dose administered at Month 3 and every 6 months. The intended route of admini

Eligibility

Age

12 Years to 99 Years

Inclusion criteria

Inclusion criteria: - An adult (>= 18 years) willing and able to provide informed consent for participation prior to performing any study related procedure. OR A minor (>12

Exclusion criteria

Exclusion criteria: - Presence of any significant ocular or non-ocular disease/disorder (or medication and/or 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. - Known hypersensitivity to antisense oligonucleotides or any constituents of the injection.

Design outcomes

Primary

Measure	Time frame
- Change from baseline in best corrected visual acuity (BCVA) (based on the Early Treatment Diabetic Retinopathy Study (ETDRS) chart) at 18 months of treatment versus sham-procedur	—

Secondary

Measure	Time frame
- Change from baseline in the following outcome measures: Other measures of BCVA Spectral domain optical coherence tomography (SD-OCT) Low Luminance Visual Acuity (LLVA) Microperimetry Static perimetry Full-field Stimulus Threshold (FST) - Change from baseline in PRO measures, as assessed by: Veteran Administration Low Vision Visual Functioning Questionnaire (VA LV VFQ-20) Patient Global Impressions of Severity (PGI-S) Patient Global Impressions of Change (PGI-C) - Ocular and non-ocular adverse events (AEs) - Exposure of QR-421a in serum	—

Measure

Time frame

- Change from baseline in the following outcome measures: Other measures of BCVA Spectral domain optical coherence tomography (SD-OCT) Low Luminance Visual Acuity (LLVA) Microperimetry Static perimetry Full-field Stimulus Threshold (FST) - Change from baseline in PRO measures, as assessed by: Veteran Administration Low Vision Visual Functioning Questionnaire (VA LV VFQ-20) Patient Global Impressions of Severity (PGI-S) Patient Global Impressions of Change (PGI-C) - Ocular and non-ocular adverse events (AEs) - Exposure of QR-421a in serum

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Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)