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Histone Deacetylase Inhibitor

PXS-5505 for Myelofibrosis

Phase 1 & 2

Recruiting

Research Sponsored by Syntara

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients who are not eligible for stem cell transplantation

Have intermediate -2, or high-risk disease according to the International Working Group prognostic scoring system (DIPSS)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 0 to follow-up visit (28 days -1 to +7days post-tx discontinuation [dose escalation phase]; day 0 to 28 days ± 3 days post-tx discontinuation [cohort expansion phase]); day 0 to follow-up visit (28 days ± 3 days post-tx discontinuation [add-on phase]

Awards & highlights

Study Summary

This trial will study PXS-5505, a potential treatment for myelofibrosis, in order to evaluate its safety and tolerability.

Who is the study for?

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.Check my eligibility

What is being tested?

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.See study design

What are the potential side effects?

While specific side effects are not listed here, phase 1/2a trials like this one generally monitor for any adverse reactions ranging from mild issues such as nausea or headaches to more serious ones including organ-related complications.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am not eligible for a stem cell transplant.

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

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My diagnosis is primary myelofibrosis or post-ET/PV myelofibrosis with at least Grade 2 marrow fibrosis.

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I can take care of myself and am up and about more than half of my waking hours.

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I agree to use approved birth control methods during and 90 days after the study.

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I've needed frequent blood transfusions or had severe side effects from ruxolitinib or fedratinib.

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I have symptoms as per the MFSAF v4.0 scale.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 0 to follow-up visit (28 days -1 to +7days post-tx discontinuation [dose escalation phase]; day 0 to 28 days ± 3 days post-tx discontinuation [cohort expansion phase]); day 0 to follow-up visit (28 days ± 3 days post-tx discontinuation [add-on phase]

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 0 to follow-up visit (28 days -1 to +7days post-tx discontinuation [dose escalation phase]; day 0 to 28 days ± 3 days post-tx discontinuation [cohort expansion phase]); day 0 to follow-up visit (28 days ± 3 days post-tx discontinuation [add-on phase]

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 0 to follow-up visit (28 days -1 to +7days post-tx discontinuation [dose escalation phase]; day 0 to 28 days ± 3 days post-tx discontinuation [cohort expansion phase]); day 0 to follow-up visit (28 days ± 3 days post-tx discontinuation [add-on phase] for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of subjects with serious and non-serious adverse events

Secondary outcome measures

Change in bone marrow (BM) fibrosis

Changes in myelofibrosis related symptoms

Changes in spleen volume

+5 more

Trial Design

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.

0 / 7Eligibility criteria met

Find a Location

Who is running the clinical trial?

SyntaraLead Sponsor

34 Previous Clinical Trials

3,050 Total Patients Enrolled

PharmaxisLead Sponsor

34 Previous Clinical Trials

3,050 Total Patients Enrolled

ParexelIndustry Sponsor

303 Previous Clinical Trials

100,787 Total Patients Enrolled

Media Library

PXS-5505 (Histone Deacetylase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04676529 — Phase 1 & 2

Myelofibrosis Research Study Groups: PXS-5505, Add-on Phase, PXS-5505, Expansion Phase, PXS-5505, Dose Level 1, Escalation Phase (Cohort A), PXS-5505, Dose Level 2, Escalation Phase (Cohort B), PXS-5505, Dose Level 3, Escalation Phase (Cohort C)

Myelofibrosis Clinical Trial 2023: PXS-5505 Highlights & Side Effects. Trial Name: NCT04676529 — Phase 1 & 2

PXS-5505 (Histone Deacetylase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04676529 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this study open to participants of advanced age?

"This trail has specific eligibility requirements, including being of a certain age. Participants between the ages 18 to 65 are welcome and there are special trials for those younger or older than this bracket."

Answered by AI

Is there an opportunity to participate in this exploration?

"Participants aged within the 18-135 range and suffering from primary myelofibrosis can qualify for this trial, which is recruiting about two dozen individuals."

Answered by AI

How many locations are administering this trial?

"At present time, the trial is accepting volunteers at 5 sites: Boston, Cleveland, Birmingham and two other locales. To reduce travel-related stressors it could be beneficial to pick a location closest to you if enrolling in this experiment."

Answered by AI

What is the current intake capacity of this clinical trial?

"This trial necessitates the participation of 24 individuals who meet all specified inclusion standards. Participants may attend either Harvard U Med School IRB #1 in Boston, Massachusetts or Cleveland Clinic - Medical Oncology/Hematology in Cleveland, Ohio to partake in this experiment."

Answered by AI

Are there still vacancies available for participation in this clinical experiment?

"Clinicaltrials.gov confirms that this medical trial is actively onboarding participants, with the initial posting taking place on February 18th 2021 and its most recent update occuring on November 28th 2022."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study to Evaluate Safety, Pharmacokinetic and Pharmacodynamic Dose Escalation and Expansion Study of PXS-5505 in Patients With Primary, Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis

2021. "Study to Evaluate Safety, Pharmacokinetic and Pharmacodynamic Dose Escalation and Expansion Study of PXS-5505 in Patients With Primary, Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04676529.

All > Essential Thrombocythemia > Polycythemia Vera