Extension Study to Study PQ-110-001 (NCT03140969)

Eye Diseases Clinical Trial

— INSIGHT  

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03913130
• Other study ID #: PQ-110-002
• Secondary ID: 2018-003500-40
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: May 13, 2019
• Est. completion date: October 3, 2022

Study information

• Verified date: December 2022
• Source: ProQR Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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. Study PQ-110-002 will provide long-term safety, tolerability, pharmacokinetic (PK), and efficacy data of QR-110.

Description:

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 9
• Est. completion date: October 3, 2022
• Est. primary completion date: October 3, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Blindness
• Disease
• Eye Abnormalities
• Eye Diseases
• Eye Diseases, Hereditary
• Eye Disorders Congenital
• Genetic Diseases, Inborn
• Leber Congenital Amaurosis
• Leber Congenital Amaurosis 10
• Neurologic Manifestations
• Retinal Disease
• Retinal Diseases
• Sensation Disorders
• Vision Disorders

Intervention

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

Locations

Country	Name	City	State
Belgium	Ghent University Hospital and Ghent University	Ghent
United States	University of Iowa	Iowa City	Iowa
United States	Scheie Eye Institute, University of Pennsylvania	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
ProQR Therapeutics

Countries where clinical trial is conducted

United States,  Belgium, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Frequency of Ocular AEs	Frequency of ocular adverse events (AEs)	24 months
Primary	Frequency of Non-ocular AEs	Frequency of non-ocular AEs	24 months
Secondary	Change in BCVA in First Treated Eye	Change in Best Corrected Visual Acuity (BCVA) in First Treated Eye	24 months
Secondary	Change in Mobility Course Score	Change in Mobility course score	24 months
Secondary	Change in Photoreceptor Outer Segment Layer Thickness	Change in photoreceptor outer segment layer thickness by Optical Coherence Tomography (OCT)	24 months
Secondary	Change in OCI	Change in Oculomotor Instability (OCI)	24 months
Secondary	Change in FST Blue	Change in Full-Field Stimulus Testing (FST) - blue stimuli	24 months
Secondary	Change in FST Red	Change in Full-Field Stimulus Testing (FST) - red stimuli	24 months
Secondary	Change in VFQ-25	Change in Visual Function Questionnaire-25 (VFQ-25) score (adult subjects)	24 months
Secondary	Change in CVAQ	Change in Cardiff Visual Ability Questionnaire for Children (CVAQC) score (pediatric subjects)	24 months
Secondary	Change in PLR	Change in Pupillary Light Reflex (PLR) (latency and amplitude)	24 months
Secondary	Change in NIRAF	Change in Near Infrared AutoFluorescence (NIRAF)	24 months
Secondary	Change in BCVA in Treated Contralateral Eye	Change in Best Corrected Visual Acuity (BCVA) in Treated Contralateral Eye	24 months
Secondary	Change in BCVA in Non-Treated Contralateral Eye	Change in Best Corrected Visual Acuity (BCVA) in Non-Treated Contralateral Eye	24 months

External Links

•   Sponsor website