Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
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|ClinicalTrials.gov Identifier: NCT03140969|
Recruitment Status : Completed
First Posted : May 4, 2017
Results First Posted : December 27, 2022
Last Update Posted : December 27, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Leber's Congenital Amaurosis||Drug: QR-110||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||11 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-label, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene|
|Actual Study Start Date :||October 16, 2017|
|Actual Primary Completion Date :||October 2, 2019|
|Actual Study Completion Date :||October 2, 2019|
Administered every 3 months
RNA antisense oligonucleotide for intravitreal injection
Other Name: Sepofarsen
- Frequency and Severity of Ocular Adverse Events in the Treatment and Contralateral Eyes [ Time Frame: 1 year ]
- Frequency and Severity of Non-ocular Adverse Events [ Time Frame: 1 year ]
- Change in Best-corrected Visual Acuity (BCVA) [ Time Frame: 1 year ]
- Change in Full-field Stimulus Test (FST) [ Time Frame: 1 year ]Average Red Light Score
- Change in Full-field Stimulus Test (FST) [ Time Frame: 1 year ]Average Blue Light Score
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|Ages Eligible for Study:||6 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Male or female, ≥ 6 years of age at Screening with a clinical diagnosis of LCA and a molecular diagnosis of homozygosity or compound heterozygosity for the CEP290 p.Cys998X mutation.
- Best-corrected visual acuity greater than or equal to light perception in both eyes and equal to or worse than LogMAR +1.0 (Snellen notation 20/200) in the worse eye and equal to or worse than LogMAR +0.7 (Snellen notation 20/100) in the contralateral eye.
- Detectable outer nuclear layer (ONL) in the area of the macula.
- An electroretinogram (ERG) result consistent with LCA.
- Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging.
- Syndromic disease.
- Pregnant or breast-feeding female.
- Any clinically significant cardiac disease or defect.
- One or more coagulation parameters outside of the normal range.
- Any ocular disease or condition that could compromise treatment safety, visual acuity or interfere with assessment of efficacy and safety.
- Prior receipt of intraocular surgery or intravitreal injection within 3 months prior to study start or planned intraocular surgery or procedure during the course of the study.
- Use of any investigational drug or device within 90 days or 5 half-lives of Day 1, whichever is longer, or plans to participate in another study of a drug or device during the PQ-110-001 study period.
- Any prior receipt of genetic therapy for LCA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03140969
|United States, Iowa|
|University of Iowa|
|Iowa City, Iowa, United States, 52242|
|United States, Pennsylvania|
|Scheie Eye Institute, University of Pennsylvania|
|Philadelphia, Pennsylvania, United States, 19104|
|Ghent University Hospital and Ghent University|
|Ghent, Belgium, B-9000|
|Study Director:||ProQR Study Director||ProQR Therapeutics|
Documents provided by ProQR Therapeutics:
|Responsible Party:||ProQR Therapeutics|
|Other Study ID Numbers:||
2017-000813-22 ( EudraCT Number )
|First Posted:||May 4, 2017 Key Record Dates|
|Results First Posted:||December 27, 2022|
|Last Update Posted:||December 27, 2022|
|Last Verified:||December 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Leber's congenital amaurosis
Leber Congenital Amaurosis
Nervous System Diseases
Eye Diseases, Hereditary