New drug aims to reverse bone marrow scarring in rare blood cancer
NCT ID NCT07394153
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
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 30 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Apr 2026
- Expected to finish
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Jun 2028
An estimate. End dates often move.
- Lead sponsor
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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
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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.
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The places running it
13 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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
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Fundación Jimenez Díaz
RECRUITINGMadrid, Madrid, 28040, Spain
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Hospital Clinic de Barcelona
RECRUITINGBarcelona, Barcelona, 08036, Spain
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Hospital Clínico Universitario Valencia
RECRUITINGValencia, Valencia, 46010, Spain
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Hospital General Universitario Gregorio Marañon
RECRUITINGMadrid, Madrid, 28007, Spain
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Hospital General Universitario Morales Meseguer
RECRUITINGMurcia, Murcia, 30008, Spain
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Hospital General Universitario de Valencia
RECRUITINGValencia, Valencia, 46014, Spain
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Hospital Universitario 12 de Octubre
RECRUITINGMadrid, Madrid, 28041, Spain
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Hospital Universitario Doctor Peset
NOT_YET_RECRUITINGValencia, Valencia, 46017, Spain
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Hospital Universitario Ramon y Cajal
RECRUITINGMadrid, Madrid, 28034, Spain
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Hospital Universitario Vall d´Hebron
RECRUITINGBarcelona, Barcelona, 08035, Spain
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Hospital Universitario de Jerez
RECRUITINGJerez de la Frontera, Cádiz, 11407, Spain
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Hospital Universitario de Salamanca
NOT_YET_RECRUITINGSalamanca, Salamanca, 37007, Spain
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Hospital del Mar Barcelona
RECRUITINGBarcelona, Barcelona, 08003, Spain
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Half-Matched stem cells tested as cure for myelofibrosis
- Can blood tests predict transplant complications?
- Real-World data may unlock Pacritinib's true potential in myelofibrosis
- Can a Dual-Action pill tame bone marrow tumors?
- Can a new daily pill shrink the spleen and ease myelofibrosis symptoms?
- Can a new pill tame myelofibrosis?