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Trial record 9 of 16 for:    Recruiting, Not yet recruiting, Active, not recruiting, Enrolling by invitation Studies | Leber Congenital Amaurosis

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 : April 14, 2021
Sponsor:
Information provided by (Responsible Party):
ProQR Therapeutics

Tracking Information
First Submitted Date  ICMJE February 21, 2019
First Posted Date  ICMJE April 12, 2019
Last Update Posted Date April 14, 2021
Actual Study Start Date  ICMJE May 13, 2019
Estimated Primary Completion Date May 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 10, 2019)
  • 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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 10, 2019)
  • 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)
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Extension Study to Study PQ-110-001 (NCT03140969)
Official Title  ICMJE 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
Brief 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.
Detailed 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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Leber Congenital Amaurosis 10
  • Blindness
  • Leber Congenital Amaurosis
  • Vision Disorders
  • Sensation Disorders
  • Neurologic Manifestations
  • Eye Diseases
  • Eye Diseases, Hereditary
  • Eye Disorders Congenital
  • Retinal Disease
Intervention  ICMJE 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
Study Arms  ICMJE 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
Intervention: Drug: QR-110
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: April 10, 2019)
11
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 2022
Estimated Primary Completion Date May 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03913130
Other Study ID Numbers  ICMJE PQ-110-002
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party ProQR Therapeutics
Study Sponsor  ICMJE ProQR Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: ProQR Medical Monitor ProQR Therapeutics
PRS Account ProQR Therapeutics
Verification Date April 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP