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

Eye Diseases Clinical Trial

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.

Status Recruiting

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04855045
Study type	Interventional
Source	ProQR Therapeutics
Contact	ProQR Clinical Trials Manager
Phone	+31881667000
Email	info@proqr.com
Status	Recruiting
Phase	Phase 2/Phase 3
Start date	March 23, 2021
Completion date	December 2023

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT05414994` - Assessment of the Ocular Microbiome in Health and Disease
Active, not recruiting	`NCT04271709` - Manhattan Vision Screening and Follow-Up Study (NYC-SIGHT)	N/A
Completed	`NCT02811692` - Study for Collection of Aflibercept Data in Routine Practice
Recruiting	`NCT06045299` - Evaluation of the Efficacy and Safety of LNZ101 and LNZ100 for the Presbyopia	Phase 3
Recruiting	`NCT01521065` - An Open-label Study to Evaluate the Clinical and Economic Benefits of I-Ray in Patients With Choroidal Neovascularization Secondary to Age-related Macular Degeneration	Phase 2
Completed	`NCT04799704` - Tear Film SARS-nCoV-2 Detection in Symptomatic and Pauci-symptomatic Patients.
Recruiting	`NCT05876689` - Swept Source OCT Imaging With the DREAM VG-OCT
Active, not recruiting	`NCT04123626` - A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene	Phase 1/Phase 2
Recruiting	`NCT04150432` - Correlation Between Exhaled Propofol Concentration With Plasma Concentration in Children	N/A
Not yet recruiting	`NCT05550740` - Repeated Low-Level Red-Light Therapy for Shortening Axial Length	N/A
Completed	`NCT02332343` - Sparing of the Fovea in Geographic Atrophy Progression	N/A
Completed	`NCT01727973` - Efficacy of Subantimicrobial Dose Doxycycline for Moderate to Severe and Active Graves' Orbitopathy	Phase 1/Phase 2
Terminated	`NCT01225146` - Study of 2.0 mg Ranibizumab in Subjects With Ischemic Central Retinal Vein Occlusion (RAVE2)	Phase 1
Completed	`NCT00333203` - Next Generation Ophthalmic Irrigating Solution Posterior Segment Study	Phase 3
Not yet recruiting	`NCT05565547` - Multimodal Equipment for Teleophthalmology Assessment (META)
Recruiting	`NCT05158699` - Effectiveness of Periocular Drug Injection in CATaract Surgery	Phase 3
Recruiting	`NCT06112340` - A Phase 2b Extension Study of Two Doses of Linsitinib in Subjects With Active, Moderate to Severe Thyroid Eye Disease (TED)	Phase 2/Phase 3
Recruiting	`NCT06451172` - Novel Antisense Oligonucleotide Eye Drops for Treating Antibiotic-Resistant Bacterial Keratitis	Early Phase 1
Completed	`NCT05211089` - Manual Versus Automated Choroidal Thickness Measurements Using Swept-source Anterior Segment OCT
Not yet recruiting	`NCT06070467` - Holistic Mixed Approaches to Capture the Real Life of Children With Rare Eye Diseases

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.