Status:
UNKNOWN
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.
Lead Sponsor:
ProQR Therapeutics
Conditions:
Leber Congenital Amaurosis 10
Blindness
Eligibility:
All Genders
Up to 7 years
Phase:
PHASE2
PHASE3
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 i...
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 subje...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
March 23 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2023
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT04855045
Start Date
March 23 2021
End Date
December 1 2023
Last Update
March 25 2022
Active Locations (9)
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1
Universitair Ziekenhuis Gent (UZ)
Ghent, Belgium, 9000
2
INRET Clinica e Centro de Pesquisa / Santa Casa BH
Belo Horizonte, Brazil
3
Federal University of Sao Paulo - Hospital Sao Paulo
São Paulo, Brazil
4
University of Alberta
Edmonton, Alberta, Canada