Is participation in this St Louis clinical trial free?

Yes, all study-related care is provided at no cost to qualified participants. Many studies also offer compensation for time and travel expenses.

E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome Clinical Trial in St Louis, MO

Q: Is NCT06538181 recruiting in St Louis, MO?

Yes, NCT06538181 is currently recruiting participants at its St Louis, MO location. This e1 ubiqutin-activating enzyme, x-linked, autoinflammatory, somatic syndrome clinical trial is actively enrolling qualified participants.

Q: How do I join the e1 ubiqutin-activating enzyme, x-linked, autoinflammatory, somatic syndrome clinical trial in St Louis?

To join this clinical trial in St Louis, complete the eligibility form on this page. A study coordinator will contact you to discuss next steps and determine if you qualify.

About This E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome Study in St Louis

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.

Sponsor: Washington University School of Medicine

Who Can Participate

Inclusion Criteria

Patients must have UBA1 mutation with a variant allele frequency (VAF) of ≥ 2% detected on a next generation sequencing panel and have at least one of the following current or past clinical manifestation of VEXAS syndrome, as determined by the attending physician:

ocular/orbital inflammation (e.g., uveitis/iritis, episcleritis)

genitourinary inflammation (e.g., epididymitis/orchitis)

arthritis/arthralgias

pulmonary inflammation (e.g., alveolitis/pleural effusion,)

splenomegaly

hepatomegaly

myocarditis or pericarditis

cytopenias (defined as hemoglobin \<11 g/dL, platelets \< 100 X 10\^9 /L, OR absolute neutrophil count \<1.0 X 10\^9 /L).

Patients with VEXAS syndrome who have never been treated with a JAK-I will be eligible to enroll on study. A stable corticosteroid dose must be maintained for at least 14 days prior to start of pacritinib.

Patients who have previously been treated with a JAK-I other than pacritinib, or who are currently being treated with a JAK-I other than pacritinib, may be eligible after a 28 day washout if either (i) their symptoms are not adequately controlled, as determined by the treating physician, or (ii) they have been unable to taper corticosteroids to an equivalent of \<10 mg prednisone/day, and in the opinion of the treating physician, may benefit from a change in JAK-I. A stable corticosteroid dose must be maintained for at least 14 days prior to start of pacritinib.

At least 18 years of age.

ECOG performance status ≤ 3.

Organ function as defined below:

Absolute neutrophil count ≥ 0.5 K/cumm

Platelets ≥ 25 K/cumm

PT/PTT \<2.5 X upper limit of normal (ULN)

Total bilirubin ≤ 1.5 x IULN

AST(SGOT)/ALT(SGPT) ≤ 3.0 x IULN

Creatinine clearance ≥ 30 mL/min by Cockcroft-Gault

QTcF \< 480 msec.

The effects of pacritinib on the developing human fetus are unknown. For this reason and because pacritinib was shown to be teratogenic in animal studies, women of childbearing potential and men must agree to use highly effective contraception prior to study entry, for the duration of study participation, and for 30 days after completion of study treatment. Hormonal contraception is no longer considered highly effective alone as pacritinib is a CYP3A4 inducer and accelerated progesterone metabolism. The contraceptive methods considered highly effective for WOCBP who receive pacritinib are intrauterine devices, bilateral tubal occlusion, vasectomized partner, or total sexual abstinence. Hormonal contraceptives (e.g., Depo-Provera) alone are not considered highly effective methods of contraception on their own when in treatment with pacritinib; such hormonal contraceptives must be combined with an additional barrier method (condom, diaphragm with spermicidal gel, or condoms with spermicides to be considered highly effective. Highly effective contraceptive methods in males include vasectomy, sexual abstinence, and condoms when combined with their partner using a highly effective method (including oral contraceptives).

Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately.

Ability to understand and willingness to sign an IRB approved written informed consent document or that of legally authorized representative, if applicable.

Patients with myelodysplastic neoplasms (MDS) or plasma cell dyscrasias are eligible if they are not undergoing active treatment. Supportive care is permitted.

Exclusion Criteria

Prior use of pacritinib.

Use of another JAK inhibitor within 28 days of C1D1 of pacritinib.

Currently receiving any other investigational agents. Patients may be eligible after 28 day washout.

Thrombotic events (arterial or venous) within 60 days prior to enrollment.

Any recent clinically significant bleeding within at least 7 days prior to enrollment.

Any active or acute infection.

History of malignancy within the prior 2 years, with the exception of MDS and plasma cell dyscrasias, or non-melanoma skin cancers that have been treated.

History of clinically significant cardiovascular disease or clinically significant abnormalities in rhythm or conduction during screening EKG, including severe cardiac events, symptomatic congestive heart failure, unstable angina pectoris, uncontrolled cardiac arrhythmia, or heart failure.

Currently receiving immunosuppressants (other than corticosteroids), disease-modifying antirheumatic drugs (DMARDs), or biologic cytokine inhibitors. Patients may be eligible after 28 day washout.

A history of allergic reactions attributed to compounds of similar chemical or biologic composition to pacritinib.

Concurrent use of strong CYP3A4 inhibitors or inducers. Patients may be eligible after washout period of 28 days (or 5 half-lives, whichever is shorter).

Diagnosis or history of moderate (Child-Pugh B) and severe hepatic impairment (Child-Pugh C).

Pregnant and/or breastfeeding. Women of childbearing potential must have a negative serum pregnancy test within 7 days of C1D1 or negative urine pregnancy test within 3 days of C1D1.

Known active viral infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV).

Patients with latent tuberculosis. Patients must have a negative T-Spot during screening to be eligible.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in St Louis?

Yes, this clinical trial (NCT06538181) has an active research site in St Louis, MO that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome Treatment Options in St Louis, MO

If you're searching for e1 ubiqutin-activating enzyme, x-linked, autoinflammatory, somatic syndrome treatment options in St Louis, MO, this clinical trial (NCT06538181) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our St Louis research site is actively enrolling participants for this clinical trial. You'll receive care from experienced e1 ubiqutin-activating enzyme, x-linked, autoinflammatory, somatic syndrome specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all e1 ubiqutin-activating enzyme, x-linked, autoinflammatory, somatic syndrome clinical trials near you to find additional studies recruiting in your area.

E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome Clinical Trial in St Louis, MO

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