Ignite Creation Date:
2024-06-16 @ 11:52 AM
Last Modification Date:
2024-10-26 @ 3:32 PM
Study NCT ID:
NCT06461286
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-06-14
First Post:
2024-06-05
Brief Title:
SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy Sundew
Sponsor:
PYC Therapeutics
Organization:
PYC Therapeutics
Study Overview
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
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
Detailed Description:
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 one1 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 33 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 33 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
Following confirmation of eligibility on Day-1 one1 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 33 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 33 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None