Autosomal Dominant Optic Atrophy Clinical Trial
Official title:
A Phase 1a Open-Label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered PYC-001 in Participants With Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy
A First-in-Human multi-centre, prospective, Phase1a, Single Ascending Dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.
This is a First-In-Human multi-centre, prospective, Phase 1a, single ascending dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA. Following confirmation of eligibility on Day-1, one(1) eye will be selected for study participation (the "study eye"), and the other eye will be designated as the "fellow eye". Selection of the "Study eye" will be the eye with worse vision. If both eyes have similar visual acuity and visual field information, the choice of study eye will be determined at the discretion of the Investigator in consultation with the participant. Each eligible participant will receive a single intravitreal (IVT) injection of PYC-001 in their study eye on Day 1, and will be monitored for dose limiting toxicities (DLTs) for 4 weeks. The study will use a 3+3 escalation scheme and will involve up to three PYC-001 dose groups. Cohorts of 3 participants will be initially enrolled at each dose level, and then expanded to 6 participants per cohort in the event of a DLT or any >> Grade 2 adverse events (AEs) that are deemed related to study treatment. Within each cohort, dosing will be staggered with a 7-day interval between the first participant receiving investigational product (IP), PYC-001, and the remaining 2 participants receiving IP. If >> 2 DLTs are observed in 6 participants in any cohort, and the previous lower dose cohort was not previously expanded to 6 participants per the 3+3 design rules, the lower dose cohort will be expanded to include evaluation of 6 participants. If this lower dose level has << 1 DLTs it will be considered the maximum tolerated dose (MTD). Alternatively, a dose half-way between the previous lower dose, and the dose with >> 2 participants showing DLTs may be selected for evaluation. If no DLTs or any >> Grade 2 AEs are observed in the first 3 participants treated within a cohort, then escalation to the next dose cohort can proceed following review of the collated 4-week safety data by the safety review committee (SRC). ;
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