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New drug aims to reverse bone marrow scarring in rare blood cancer

NCT ID NCT07394153

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jul 29, 2026 · Updated 4 times

Summary

This phase 2 trial tests the drug pacritinib in 30 people with myelofibrosis, a rare bone marrow cancer, who also have low platelet counts. The goal is to see if pacritinib can reduce scarring (fibrosis) in the bone marrow and improve symptoms. Participants take pacritinib pills twice daily for up to two years, with regular check-ups and scans.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Pacritinib
What this could lead to
If successful, this could show that pacritinib reduces bone marrow scarring and improves blood cell counts in myelofibrosis patients with low platelets.
What could go wrong
This is a small, early-phase study with only 30 participants and no comparison group. The drug may cause side effects or fail to improve fibrosis.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 30 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2026

Expected to finish

Jun 2028

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Signed written and voluntary informed consent. 2. Age ≥18 years 3. Patients with a confirmed diagnosis of myelofibrosis, either primary myelofibrosis (PMF) or post polycythemia vera (PPV-MF) or post essential thrombocythemia (PET-MF). 4. Patients with thrombocytopenia, delimited by platelets counts between 50 - 120 x 109/L. 5. Patients who require JAK-2 inhibitor therapy in the opinion of the investigator and are eligible to start treatment with pacritinib either in the first line (JAK2 inhibitor-naive) or in second line setting (after no response or loss of response or intolerance to one prior JAK2 inhibitor ). Note: patients should have recovered to grade ≤ 1 from any toxicity from previous treatment. 6. Have a Eastern Cooperative Oncology Group Performance Status (ECOG-PS) of 0 - 2. 7. Have a dynamic international prognostic scoring system (DIPSS) Intermediate-1, Intermediate-2, or High risk. 8. Peripheral blasts count \< 5% and absolute neutrophil count (ANC) of ≥500/μL. 9. Adequate liver and renal function, defined by: 1. liver transaminases, including alanine aminotransferase (ALT or GOT) and aspartate aminotransferase (AST or GOT) ≤ 3 x upper limit normal (ULN). AST/ALT ≤5 × ULN if transaminase elevation is related to MF. 2. Total bilirubin and/or direct bilirubin ≤ 4 x ULN. 3. Estimated glomerular filtration rate (eGFR) \> 30 mL/min. 10. Adequate coagulation defined by prothrombin time/international normalized ratio and partial thromboplastin time ≤ 1.5 × ULN. 11. If fertile, willing to use effective birth control methods during the study and up to 30 days after the last dose of pacritinib. 12. Willing to undergo and able to tolerate frequent MRI during the study and BM biopsy 13. Able to understand and willing to complete symptom assessments. Exclusion Criteria: 1. Life expectancy \<6 months. 2. Splenic irradiation within the last 6 months. 3. Previously treated with pacritinib. 4. Concurrent enrollment in another interventional trial. 5. Treatment with an experimental therapy within 28 days prior to the first dose of study treatment. 6. Systemic treatment with a strong CYP3A4 inhibitor or inducer and the treatment cannot be either discontinued or switched to a different medication within 5 half-lifes prior to study entry. 7. Severe (Child-Pugh C) liver impairment. 8. Significant recent bleeding history defined as NCI CTCAE grade ≥2 within 3 months prior to first dose of study treatment, or with active bleeding, unless precipitated by an inciting event (e.g., surgery, trauma, or injury). 9. Conditions or medications that increase the risk of bleeding, except for aspirin (dosages of ≤100 mg per day). Patients treated with "direct-acting oral anticoagulants (DOACs), could be considered for inclusion (may be consulted with the Sponsor, GEMFIN). 10. Any history of CTCAE grade ≥2 dysrhythmias or non-dysrhythmia cardiac conditions within 6 months prior to the first dose of study treatment. Patients with non-dysrhythmia or non-QTc grade 2 cardiovascular conditions , may be considered for inclusion, if stable , asymptomatic and unlikely to affect patient safety. 11. QT corrected by the Fridericia method (QTcF) prolongation \>480 ms or other factors that increase the risk for QTcF interval prolongation (e.g., heart failure, hypokalemia or history of long QT interval syndrome). 12. New York Heart Association Class II, III, or IV congestive heart failure. 13. Active or uncontrolled inflammatory or chronic functional bowel disorder such as Crohn's disease, inflammatory bowel disease, chronic diarrhea or constipation 14. Other malignancy within 3 years prior to treatment Day 1, other than curatively treated basal cell or squamous cell skin or corneal cancer; curatively treated carcinoma in situ of the cervix. The exception is if patients have been disease-free for at least 5 years, and are deemed by the investigator to be at low risk for recurrence of that malignancy. 15. Known seropositivity for human immunodeficiency virus. Known active hepatitis B, or C virus infection. 16. Women who are pregnant or lactating 17. Uncontrolled intercurrent illness, including, but not limited to, ongoing active infection, psychiatric illness, or social situation that, in the judgment of the treating physician, would limit compliance with study requirements. 18. Any active GI or metabolic condition that could interfere with absorption of oral medication.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    13 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Fundación Jimenez Díaz

    RECRUITING

    Madrid, Madrid, 28040, Spain

  • Hospital Clinic de Barcelona

    RECRUITING

    Barcelona, Barcelona, 08036, Spain

  • Hospital Clínico Universitario Valencia

    RECRUITING

    Valencia, Valencia, 46010, Spain

  • Hospital General Universitario Gregorio Marañon

    RECRUITING

    Madrid, Madrid, 28007, Spain

  • Hospital General Universitario Morales Meseguer

    RECRUITING

    Murcia, Murcia, 30008, Spain

  • Hospital General Universitario de Valencia

    RECRUITING

    Valencia, Valencia, 46014, Spain

  • Hospital Universitario 12 de Octubre

    RECRUITING

    Madrid, Madrid, 28041, Spain

  • Hospital Universitario Doctor Peset

    NOT_YET_RECRUITING

    Valencia, Valencia, 46017, Spain

  • Hospital Universitario Ramon y Cajal

    RECRUITING

    Madrid, Madrid, 28034, Spain

  • Hospital Universitario Vall d´Hebron

    RECRUITING

    Barcelona, Barcelona, 08035, Spain

  • Hospital Universitario de Jerez

    RECRUITING

    Jerez de la Frontera, Cádiz, 11407, Spain

  • Hospital Universitario de Salamanca

    NOT_YET_RECRUITING

    Salamanca, Salamanca, 37007, Spain

  • Hospital del Mar Barcelona

    RECRUITING

    Barcelona, Barcelona, 08003, Spain

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