Extension Study to Study PQ-110-001 (NCT03140969) (INSIGHT)
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ClinicalTrials.gov Identifier: NCT03913130 |
Recruitment Status :
Active, not recruiting
First Posted : April 12, 2019
Last Update Posted : February 21, 2021
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Condition or disease | Intervention/treatment | Phase |
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Leber Congenital Amaurosis 10 Blindness Leber Congenital Amaurosis Vision Disorders Sensation Disorders Neurologic Manifestations Eye Diseases Eye Diseases, Hereditary Eye Disorders Congenital Retinal Disease | Drug: QR-110 | Phase 1 Phase 2 |
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject.
Subjects will be given the opportunity to enroll into this extension study for continued dosing if available data support current and/or future benefits for the subject. The Investigator, in consultation and agreement with the Medical Monitor, will decide on enrollment of each individual subject, as well as on dosing of the first treated eye and treatment initiation of the contralateral eye. Continued subject treatment in this study is desirable, but cannot be guaranteed, since it will depend on the risks and benefit of further treatment on a case-by-case basis, as discussed and agreed upon with the Medical Monitor.
The contralateral eye and the first treated eye will be injected 3 months apart. The injection interval of 3 months between both eyes will limit burden for the subjects, with a 3 month-visit frequency during the course of the study. This between-eye interval could be adapted if safety data are supportive, and for logistic reasons, and in agreement with the Medical Monitor.
The same safety monitoring protocol and efficacy assessments will apply to both eyes.
QR-110 will be administered via intravitreal (IVT) injection.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 11 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open-Label, Extension Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene |
Actual Study Start Date : | May 13, 2019 |
Estimated Primary Completion Date : | March 2021 |
Estimated Study Completion Date : | March 2021 |

Arm | Intervention/treatment |
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Experimental: Drug QR-110
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
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Drug: QR-110
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration |
- Frequency of ocular AEs [ Time Frame: 24 months ]Frequency of ocular adverse events (AEs)
- Frequency of non-ocular AEs [ Time Frame: 24 months ]Frequency of non-ocular AEs
- Change in BCVA [ Time Frame: 24 months ]Change in Best Corrected Visual Acuity (BCVA)
- Change in Mobility course score [ Time Frame: 24 months ]Change in Mobility course score
- Change in photoreceptor outer segment layer thickness [ Time Frame: 24 months ]Change in photoreceptor outer segment layer thickness by Optical Coherence Tomography (OCT)
- Change in OCI [ Time Frame: 24 months ]Change in Oculomotor Instability (OCI)
- Change in FST blue [ Time Frame: 24 months ]Change in Full-Field Stimulus Testing (FST) - blue stimuli
- Change in FST red [ Time Frame: 24 months ]Change in Full-Field Stimulus Testing (FST) - red stimuli
- Change in VFQ-25 [ Time Frame: 24 months ]Change in Visual Function Questionnaire-25 (VFQ-25) score (adult subjects)
- Change in CVAQ [ Time Frame: 24 months ]Change in Cardiff Visual Ability Questionnaire for Children (CVAQC) score (pediatric subjects)
- Change in PLR [ Time Frame: 24 months ]Change in Pupillary Light Reflex (PLR) (latency and amplitude)
- Change in NIRAF [ Time Frame: 24 months ]Change in Near Infrared AutoFluorescence (NIRAF)

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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 |
Inclusion Criteria:
- Subjects who completed participation in the study PQ-110-001 and who may derive benefit from continued treatment with QR 110, as assessed by the Investigator, in consultation with the Medical Monitor
- Persistence of detectable outer nuclear layer (ONL) in the area of the macula in the opinion of the Investigator, as determined by OCT.
- Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging, as assessed by the Investigator.
- An adult (≥ 18 years) willing and able to provide informed consent for participation OR a minor (6 to < 18 years) with a parent or legal guardian willing and able to provide written permission for the subject's participation prior to performing any study related procedures, and pediatric subjects able to provide age-appropriate assent for study participation.
- Female subjects who have reached menarche and male subjects must either practice true abstinence in accordance with their preferred and usual lifestyle, or agree to use acceptable, highly effective methods of contraception for up to 3 months following their last dose QR-110. Acceptable methods of contraception are defined in the protocol. Women of non-childbearing potential may be included without the use of adequate birth control, provided they meet the criteria in the protocol.
Exclusion Criteria:
- Any contraindication to IVT injection according to the Investigator's clinical judgment and international guidelines (Avery 2014).
- Safety issue during study PQ-110-001 that may compromise subject safety when continued dosing, as determined by the Investigator, and in consultation with the Medical Monitor.
- Any ocular or systemic disease or condition (including medications and laboratory test abnormalities) that could compromise subject safety or interfere with assessment of efficacy and safety, as determined by the Investigator and in consultation with the Medical Monitor.
- Pregnant or breast-feeding female.

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): NCT03913130
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 |
Study Director: | ProQR Medical Monitor | ProQR Therapeutics |
Responsible Party: | ProQR Therapeutics |
ClinicalTrials.gov Identifier: | NCT03913130 |
Other Study ID Numbers: |
PQ-110-002 |
First Posted: | April 12, 2019 Key Record Dates |
Last Update Posted: | February 21, 2021 |
Last Verified: | February 2021 |
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 |
LCA10 CEP290 p.Cys998X c.2991+1655A>G |
Leber's Congenital Amaurosis Antisense oligonucleotide RNA therapy |
Blindness Neurologic Manifestations Vision Disorders Sensation Disorders Eye Diseases Retinal Diseases Leber Congenital Amaurosis |
Eye Diseases, Hereditary Eye Abnormalities Genetic Diseases, Inborn Disease Pathologic Processes Nervous System Diseases Congenital Abnormalities |