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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT05158296
Other study ID # PQ-421a-003
Secondary ID 2021-002729-74
Status Active, not recruiting
Phase Phase 2/Phase 3
First received
Last updated
Start date December 8, 2021
Est. completion date December 2024

Study information

Verified date September 2022
Source ProQR Therapeutics
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.


Description:

The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. The below dose levels of ultevursen will be evaluated with the loading dose administered at Day 1 and maintenance dose administered at Month 3 and every 6 months thereafter: 1. Loading dose of 180 µg, maintenance dose of 60 µg 2. Loading dose of 60 µg, maintenance dose of 60 µg Dose levels will include subjects randomized to sham-procedure or treatment with ultevursen.


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Study Design


Intervention

Drug:
Ultevursen
RNA antisense oligonucleotide for intravitreal injection
Other:
Sham-procedure
Sham-procedure (no experimental drug administered)

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Sponsors (1)

Lead Sponsor Collaborator
ProQR Therapeutics

Countries where clinical trial is conducted

United States,  Germany,  Netherlands,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Mean change from baseline in BCVA Mean change from baseline in best corrected visual acuity(BCVA) based on the Early Treatment Diabetic Retinopathy Study (ETDRS) chart 18 months of treatment versus sham-procedure
Secondary Proportion of patients who maintain vision defined by BCVA loss less than 15 Letters (ETDRS) Proportion of patients who maintain vision defined by BCVA loss less than 15 Letters based on ETDRS 27 months
Secondary Change from baseline in other analyses of BCVA Change from baseline in other analyses of BCVA 27 months
Secondary Change from baseline in ellipsoid zone (EZ) area and width as imaged by spectral domain optical coherence tomography (SD-OCT) Change from baseline in ellipsoid zone (EZ) area and width as imaged by spectral domain optical coherence tomography (SD-OCT) 27 months
Secondary Change from baseline in Low Luminance Visual Acuity (LLVA) Change from baseline in Low Luminance Visual Acuity (LLVA) 27 months
Secondary Change from baseline in Microperimetry Change from baseline in Microperimetry 27 months
Secondary Change from baseline in Full-field Stimulus Threshold (FST) Change from baseline in Full-field Stimulus Threshold (FST) 27 months
Secondary Change from baseline in PRO measures As assessed by the Veteran Affairs Low Vision Visual Functioning Questionnaire (VA LV VFQ-20), Patient Global Impressions of Severity (PGI-S) and Patient Global Impressions of Change (PGI-C) 27 months
Secondary Ocular and non-ocular adverse events (AEs) Ocular and non-ocular adverse events (AEs) 27 months
Secondary Cmax of ultevursen in serum Maximum concentration (Cmax) of ultevursen in serum 27 months
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