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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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03140969
Recruitment Status : Completed
First Posted : May 4, 2017
Results First Posted : December 27, 2022
Last Update Posted : December 27, 2022
Sponsor:
Information provided by (Responsible Party):
ProQR Therapeutics

Study Description
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Brief Summary:
The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.

Condition or disease Intervention/treatment Phase
Leber's Congenital Amaurosis Drug: QR-110 Phase 1 Phase 2

Detailed Description:
The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation. Subjects will receive QR-110 in one eye every 3 months, for a maximum of 4 doses. Up to 3 dose levels of QR-110 will be evaluated.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 participants
Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: QR-110
Administered every 3 months
 Drug: QR-110
RNA antisense oligonucleotide for intravitreal injection
Other Name: Sepofarsen


Outcome Measures
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Primary Outcome Measures :
  1. Frequency and Severity of Ocular Adverse Events in the Treatment and Contralateral Eyes [ Time Frame: 1 year ]

Secondary Outcome Measures :
  1. Frequency and Severity of Non-ocular Adverse Events [ Time Frame: 1 year ]
  2. Change in Best-corrected Visual Acuity (BCVA) [ Time Frame: 1 year ]
  3. Change in Full-field Stimulus Test (FST) [ Time Frame: 1 year ]
    Average Red Light Score

  4. Change in Full-field Stimulus Test (FST) [ Time Frame: 1 year ]
    Average Blue Light Score


Eligibility Criteria
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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
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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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
Belgium
Ghent University Hospital and Ghent University
Ghent, Belgium, B-9000
Sponsors and Collaborators
ProQR Therapeutics
Investigators
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Study Director: ProQR Study Director ProQR Therapeutics
  Study Documents (Full-Text)

Documents provided by ProQR Therapeutics:
Study Protocol  [PDF] August 8, 2018
Statistical Analysis Plan  [PDF] August 9, 2019

More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: ProQR Therapeutics
ClinicalTrials.gov Identifier: NCT03140969    
Other Study ID Numbers: PQ-110-001
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by ProQR Therapeutics:
CEP290
p.Cys998X
c.2991+1655A>G
 RNA therapy
Antisense oligonucleotide
Leber's congenital amaurosis
Additional relevant MeSH terms:
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Blindness
Leber Congenital Amaurosis
Vision Disorders
Sensation Disorders
Neurologic Manifestations
 Nervous System Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Diseases