Ignite Creation Date:
2024-10-26 @ 3:37 PM
Last Modification Date:
2024-10-26 @ 3:37 PM
Study NCT ID:
NCT06538181
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-07-31
Brief Title:
Pacritinib in Vacuoles E1 Ubiqutin-activating Enzyme X-linked Autoinflammatory Somatic VEXAS Syndrome
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Phase 1 Study of Pacritinib in Vacuoles E1 Ubiqutin-activating Enzyme X-linked Autoinflammatory Somatic VEXAS Syndrome
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
VEXAS vacuoles E1 ubiqutin-activating enzyme X-linked autoinflammatory somatic syndrome is a recently described disorder with severe hematologic and rheumatologic manifestations caused by somatic variants in the ubiquitin- activating enzyme gene UBA1 that is acquired in hematopoietic progenitor cells Patients are often debilitated by autoinflammatory symptoms and there is currently no standard of care available There is a clinically unmet need for better therapies in VEXAS Syndrome There have been no prospective clinical trials of JAK-I in VEXAS syndrome The investigators hypothesize that pacritinib as a JAK2IRAK1 inhibitor with a manageable safety profile in myelofibrosis patients with thrombocytopenia will improve the autoinflammatory and hematologic manifestations of VEXAS syndrome with a tolerable toxicity profile
The investigators propose a single arm pilot Phase 1 study evaluating the safety and tolerability of pacritinib in patients with VEXAS syndrome with an initial safety run-in phase of 6 patients treated with pacritinib 200mg twice daily BID on days 1-28 of a continuous 28 day cycle If no more than 1 patient experiences a dose-limiting toxicity DLT the investigators will enroll an expansion cohort to gain additional toxicity and efficacy data for a total enrollment of 15 patients If more than 1 patient experiences a DLT during the safety run-in phase the investigators will decrease the dose to 100 mg BID and if no more than 1 of 6 patients experiences a DLT the investigators will complete the expansion cohort as above for up to a total enrollment of 15 patients If more than 1 patient experiences a DLT at 100 mg BID the investigators will discontinue the study Patients will be treated for up to 12 cycles
The investigators propose a single arm pilot Phase 1 study evaluating the safety and tolerability of pacritinib in patients with VEXAS syndrome with an initial safety run-in phase of 6 patients treated with pacritinib 200mg twice daily BID on days 1-28 of a continuous 28 day cycle If no more than 1 patient experiences a dose-limiting toxicity DLT the investigators will enroll an expansion cohort to gain additional toxicity and efficacy data for a total enrollment of 15 patients If more than 1 patient experiences a DLT during the safety run-in phase the investigators will decrease the dose to 100 mg BID and if no more than 1 of 6 patients experiences a DLT the investigators will complete the expansion cohort as above for up to a total enrollment of 15 patients If more than 1 patient experiences a DLT at 100 mg BID the investigators will discontinue the study Patients will be treated for up to 12 cycles
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None