Eye Diseases Clinical Trial
— BRIGHTENOfficial 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.
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.
Status | Recruiting |
Enrollment | 15 |
Est. completion date | December 2023 |
Est. primary completion date | December 2023 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 7 Years |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
Belgium | Universitair Ziekenhuis Gent (UZ) | Ghent | |
Brazil | INRET Clinica e Centro de Pesquisa / Santa Casa BH | Belo Horizonte | |
Brazil | Federal University of Sao Paulo - Hospital Sao Paulo | São Paulo | |
Canada | University of Alberta | Edmonton | Alberta |
Germany | Justus-Liebig Universität - Department of Ophthalmology | Gießen | |
Germany | University of Tübingen - Institute for Ophthalmic Research | Tübingen | |
Italy | Eye Clinic University of Campania Liugi Vanvitelli | Naples | |
Netherlands | Amsterdam University Medica Center - Locatie AMC | Amsterdam | |
United Kingdom | Moorfields Eye Hospital - NHS Foundation Trust | London |
Lead Sponsor | Collaborator |
---|---|
ProQR Therapeutics |
Belgium, Brazil, Canada, Germany, Italy, Netherlands, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Incidence and severity of ocular adverse events (AEs) | Incidence and severity of ocular adverse events (AEs) | 24 months | |
Primary | Incidence and severity of non-ocular adverse events (AEs) | Incidence and severity of non-ocular adverse events (AEs) | 24 months | |
Secondary | Change from baseline to Month 12 in Best-corrected visual acuity (BCVA) | Mean change in BCVA relative to baseline after 12 months of treatment | 12 months | |
Secondary | Change from baseline to Month 12 in retinal sensitivity measured by Full-field stimulus testing (FST) | Mean change in retinal sensitivity measured by FST relative to baseline after 12 months of treatment | 12 months |
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