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Trial record 8 of 16 for:    Recruiting, Not yet recruiting, Active, not recruiting, Enrolling by invitation Studies | Leber Congenital Amaurosis

An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. (BRIGHTEN)

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ClinicalTrials.gov Identifier: NCT04855045
Recruitment Status : Recruiting
First Posted : April 22, 2021
Last Update Posted : April 22, 2021
Sponsor:
Information provided by (Responsible Party):
ProQR Therapeutics

Brief Summary:
PQ-110-005 (BRIGHTEN) is an open-label, dose escalation and double-masked, randomized, controlled study evaluating safety and tolerability of sepofarsen administered via intravitreal (IVT) injection in pediatric subjects (<8 years of age) with LCA10 due to the c.2991+1655A>G mutation over 24 months of treatment.

Condition or disease Intervention/treatment Phase
Leber Congenital Amaurosis 10 Blindness Leber Congenital Amaurosis Vision Disorders Sensation Disorders Neurologic Manifestations Eye Diseases Eye Disorders Congenital Retinal Disease Retinal Degeneration Retinal Dystrophies Drug: sepofarsen Phase 2 Phase 3

Detailed Description:

This is an open-label, dose escalation and double-masked, randomized, controlled study evaluating safety and tolerability of sepofarsen administered via intravitreal (IVT) injection in pediatric subjects (<8 years of age) with LCA10 due to the c.2991+1655A>G mutation. The study consists of two parts: an open-label dose escalation part, followed by a double-masked randomized part.

In the open label part; subjects will be assigned to one of 3 planned dose groups using a staggered dose escalation design. After at least 1 patient is dosed in each group; the Data Monitoring Committee (DMC) will review at least 4 weeks of safety data post dosing; and may recommend initiation of the next dose group. The DMC may recommend initiation of the double-masked randomized part of the study after completion of the last dose group in the dose escalation part of the study.

In the double-masked, randomized, controlled part of the study; subjects will be randomized to one of 2 planned dose groups .

Subjects will receive a unilateral IVT injection of sepofarsen on Day 1. Thereafter a 6-monthly dosing schedule is planned.

After each dosing subjects will be assessed for safety and tolerability at follow up visits.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: The study consists of two parts: an open-label dose escalation part, followed by a double-masked randomized part.
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: An Open-Label, Dose Escalation and Double-Masked, Randomized, Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects <8 Years of Age With Leber Congenital Amaurosis Type 10 (LCA10) Due to the c.2991 +1655A>G (p.Cys998X) Mutation.
Actual Study Start Date : March 23, 2021
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2023

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Group 1 - open label Drug: sepofarsen
RNA antisense oligonucleotide for intravitreal injection
Other Name: QR-110

Experimental: Group 2 - open label Drug: sepofarsen
RNA antisense oligonucleotide for intravitreal injection
Other Name: QR-110

Experimental: Group 3: open label Drug: sepofarsen
RNA antisense oligonucleotide for intravitreal injection
Other Name: QR-110

Experimental: Group 4: double-masked, randomized to one of 2 dose cohorts Drug: sepofarsen
RNA antisense oligonucleotide for intravitreal injection
Other Name: QR-110




Primary Outcome Measures :
  1. Incidence and severity of ocular adverse events (AEs) [ Time Frame: 24 months ]
    Incidence and severity of ocular adverse events (AEs)

  2. Incidence and severity of non-ocular adverse events (AEs) [ Time Frame: 24 months ]
    Incidence and severity of non-ocular adverse events (AEs)


Secondary Outcome Measures :
  1. Change from baseline to Month 12 in Best-corrected visual acuity (BCVA) [ Time Frame: 12 months ]
    Mean change in BCVA relative to baseline after 12 months of treatment

  2. Change from baseline to Month 12 in retinal sensitivity measured by Full-field stimulus testing (FST) [ Time Frame: 12 months ]
    Mean change in retinal sensitivity measured by FST relative to baseline after 12 months of treatment



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 7 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female child, <8 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, based on genotyping analysis at Screening. A historic genotyping report from a certified laboratory are acceptable with Sponsor approval.
  • BCVA equal to or better than Logarithm of the Minimum Angle of Resolution (logMAR) + 4.0 (Light Perception), and equal to or worse than logMAR + 0.4 in the treatment eye.
  • Detectable outer nuclear layer (ONL) in the area of the macula.

Exclusion Criteria:

  • Presence of any significant ocular or non-ocular disease/disorder which may put the subject at risk because of participation in the trial' may influence the results of the trial, or the subject's ability to participate in the trial.
  • Receipt within 1 month prior to Screening of any intraocular or periocular surgery (including refractive surgery), or an IVT injection or planned intraocular surgery or procedure during the course of the trial.
  • Current treatment or treatment within the past 12 months with therapies known to influence the immune system (including but not limited to cytostatics, interferons, TNF-binding proteins, drugs acting on immunophilins, or antibodies with known impact on the immune system).
  • Current treatment or treatment within the past 3 months or planned treatment with drugs known to be toxic to the lens, retina, or the optic nerve.
  • Use of any investigational drug or device within 3 months or 5 half-lives of Day 1, whichever is longer, or plans to participate in another study of a drug or device during the trial period.
  • Any prior receipt of genetic or stem-cell therapy for ocular or non-ocular disease.

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): NCT04855045


Contacts
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Contact: ProQR Clinical Trials Manager +31881667000 info@proqr.com

Locations
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Belgium
Universitair Ziekenhuis Gent (UZ) Recruiting
Ghent, Belgium, 9000
Contact: Bart Leroy       bart.leroy@ugent.be   
Principal Investigator: Bart Leroy         
Germany
Justus-Liebig Universität - Department of Ophthalmology Not yet recruiting
Gießen, Germany, 35392
Contact: Lyubomyr Lytvynchuk    +49-641-985-43803    lyubomyr.Lytvynchuk@augen.med.uni-giessen.de   
Principal Investigator: Lyubomyr Lytvynchuk         
University of Tübingen - Institute for Ophthalmic Research Not yet recruiting
Tübingen, Germany, 72076
Contact: Andrea Rindtorff    +49 7071 29 87747    andrea.rindtorff@stz-eyetrial.de   
Principal Investigator: Katarina Stingl, MD         
Netherlands
Amsterdam University Medica Center - Locatie AMC Recruiting
Amsterdam, Netherlands, 1105 AZ
Contact: Monique Wezel    +31 205668618    m.wezel@amsterdamumc.nl   
Principal Investigator: Camiel Boon         
United Kingdom
Moorfields Eye Hospital - NHS Foundation Trust Not yet recruiting
London, United Kingdom, EC1V 2PD
Contact: Flora Kakanou    +44 0207 253 3411 ext 2109    flora.kakanou@nhs.net   
Principal Investigator: Michel Michaelides         
Sponsors and Collaborators
ProQR Therapeutics
Investigators
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Study Director: ProQR Medical Monitor ProQR Therapeutics
Additional Information:
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Responsible Party: ProQR Therapeutics
ClinicalTrials.gov Identifier: NCT04855045    
Other Study ID Numbers: PQ-110-005
First Posted: April 22, 2021    Key Record Dates
Last Update Posted: April 22, 2021
Last Verified: April 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by ProQR Therapeutics:
Leber Congenital Amaurosis
LCA10
CEP290
p.Cys998X
c.2991+1655A>G
Antisense oligonucleotide
RNA therapy
QR-110
sepofarsen
pediatric
children
Additional relevant MeSH terms:
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Leber Congenital Amaurosis
Congenital Abnormalities
Blindness
Neurologic Manifestations
Vision Disorders
Sensation Disorders
Eye Diseases
Retinal Diseases
Retinal Degeneration
Retinal Dystrophies
Eye Abnormalities
Disease
Pathologic Processes
Nervous System Diseases
Eye Diseases, Hereditary