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NCT06538181 · ClinicalTrials.gov registry record · Phase 1
Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome
A Phase 1 study of VEXAS and Vexas Syndrome, sponsored by Washington University School of Medicine.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
- 1
- Study location
NCT06538181 is a Phase 1 study of VEXAS and Vexas Syndrome that is actively recruiting participants, run by Washington University School of Medicine. The registered enrollment target is 15 participants. The trial reports 1 study location across 1 state.
The verdict
NCT06538181, a Phase 1 study of VEXAS and Vexas Syndrome, is actively recruiting participants, sponsored by Washington University School of Medicine.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
- 1
- Study location
Study 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 JAK2/IRAK1 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.
Conditions Studied
Interventions
- DRUG Pacritinib
Study Locations (1)
Missouri
- Washington University School of Medicine - St Louis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2025-02-13 |
| Est. Completion | 2029-02-28 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06538181
The ClinicalTrials.gov registry entry for NCT06538181 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 15 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Washington University School of Medicine, which has 1,502 total studies on file at ClinicalTrials.gov.
The record links to 3 conditions, with VEXAS appearing as the primary indexed condition, and to 1 intervention - of which Pacritinib is the first listed.
NCT06538181 reports 1 study location spanning 1 distinct geographic area - top geographies include Missouri.
Frequently Asked Questions
What is clinical trial NCT06538181 about?
NCT06538181 is a clinical study titled "Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome". 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 progeni...
What is the current status of trial NCT06538181?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2025-02-13. Estimated completion is 2029-02-28.
What conditions does trial NCT06538181 study?
This clinical trial studies the following conditions: VEXAS, Vexas Syndrome, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome.
What interventions are being tested in trial NCT06538181?
The interventions under investigation include: Pacritinib (DRUG).
Who is sponsoring clinical trial NCT06538181?
This trial is sponsored by Washington University School of Medicine, which has 1,502 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06538181 being conducted?
This trial has 1 study location across Missouri. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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