PXS-5505 for Myelofibrosis

Recruiting in Palo Alto (17 mi)

+26 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Syntara

No Placebo Group

Trial Summary

What is the purpose of this trial?This trial tests a new drug, PXS-5505, for safety and tolerance in patients with specific bone marrow disorders. It aims to find the best dose and see if combining it with another medication improves treatment.

Eligibility Criteria

This trial is for patients with primary myelofibrosis or those who developed it after essential thrombocythemia/polycythemia vera. They should not be candidates for stem cell transplantation and must have stopped certain treatments like ruxolitinib or fedratinib. Participants need to have a specific risk level of disease, show symptoms, and have good organ function. Men and women must agree to use contraception.

Inclusion Criteria

I am not eligible for a stem cell transplant.

My condition is classified as intermediate-2 or high-risk by DIPSS.

You are expected to live for at least six more months.

+14 more

Exclusion Criteria

Participation in an investigational drug or device trial within two weeks prior to study Day 1 or within five times the half-life of the investigational agent in the other clinical study, if known

I haven't had surgery in the last two weeks and don't plan to during or right after the study.

I have had an aneurysm in the past.

+9 more

Participant Groups

The study tests PXS-5505's safety and how the body responds to different doses in people with myelofibrosis that started on its own or after other blood conditions. It's an early-stage trial (phase 1/2a) where everyone gets the drug openly without any placebo control.

5Treatment groups

Experimental Treatment

Group I: PXS-5505, Expansion PhaseExperimental Treatment1 Intervention

All patients will receive PXS-5505 at the selected twice daily dose for a period of 24 weeks, or until progressive disease, unacceptable toxicity, dose-limiting toxicity or withdrawal of consent.

Group II: PXS-5505, Dose Level 3, Escalation Phase (Cohort C)Experimental Treatment1 Intervention

Patients will receive PXS-5505 dose level 3, twice daily for a period of 4 weeks.

Group III: PXS-5505, Dose Level 2, Escalation Phase (Cohort B)Experimental Treatment1 Intervention

Patients will receive PXS-5505 dose level 2, twice daily for a period of 4 weeks.

Group IV: PXS-5505, Dose Level 1, Escalation Phase (Cohort A)Experimental Treatment1 Intervention

Patients will receive PXS-5505 dose level 1, twice daily for a period of 4 weeks.

Group V: PXS-5505, Add-on PhaseExperimental Treatment1 Intervention

Patients already receiving a stable dose of ruxolitinib for at least 12 weeks, will receive PXS-5505 (the dose used in the cohort expansion phase) on top of their ruxolitinib dose for up to 52 weeks or until progressive disease, unacceptable toxicity, dose-limiting toxicity, or withdrawal of consent.