Study of Pacritinib for Patients with Severe Thrombocytopenia in Myelofibrosis Conditions

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What is this study about?

This clinical trial is focused on studying a group of diseases known as myelofibrosis, which includes Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, and Post-Essential Thrombocythemia Myelofibrosis. These conditions are characterized by the scarring of bone marrow, which can lead to severe thrombocytopenia, a condition where there are low levels of platelets in the blood. The study is testing a treatment called Pacritinib, which is a potent and selective inhibitor of certain enzymes involved in the disease process. The trial will compare the effectiveness of Pacritinib against other treatments chosen by doctors, referred to as “physician’s choice” therapies. These other treatments may include medications like Hydroxycarbamide, Ruxolitinib, Methylprednisolone, Danazol, Prednisolone, and Dexamethasone.

The purpose of the study is to evaluate how well Pacritinib works in reducing the size of the spleen and improving symptoms in patients with myelofibrosis. The study will last for a period of 24 weeks, during which patients will receive either Pacritinib or one of the physician’s choice therapies. Throughout the study, patients will undergo regular assessments, including imaging tests like MRI or CT scans, to measure changes in spleen size and symptom severity. The study aims to determine if Pacritinib can achieve a significant reduction in spleen volume and improve the overall symptom score compared to the other treatments.

Participants in the study will be monitored for any side effects or adverse events, and their overall health will be assessed through various tests and evaluations. The study will also track the time it takes for any significant health changes to occur, such as improvements in symptoms or any serious health issues. The trial is expected to provide valuable information on the effectiveness and safety of Pacritinib for patients with severe thrombocytopenia due to myelofibrosis.

1 joining the study

Upon joining the study, you will be randomly assigned to receive either pacritinib or a treatment chosen by your doctor. This is to compare the effectiveness of the treatments.

You will be informed about the specific treatment you will receive and any necessary instructions.

2 treatment administration

If you are assigned to pacritinib, you will take it orally as a capsule. The dosage and frequency will be provided by your healthcare provider.

If you are assigned to the treatment chosen by your doctor, it may include medications such as hydroxycarbamide, ruxolitinib, methylprednisolone, danazol, prednisolone, or dexamethasone. These are also taken orally, and your doctor will provide specific instructions.

3 regular assessments

Throughout the study, you will undergo regular assessments to monitor your health and the effectiveness of the treatment.

These assessments may include imaging tests like MRI or CT scans to measure changes in spleen size and symptom evaluations using a questionnaire.

4 symptom tracking

You will be asked to complete symptom assessments using a patient-reported outcome instrument. This helps track any changes in your symptoms over time.

It is important to provide accurate information to help evaluate the treatment’s impact on your condition.

5 end of treatment evaluation

At the end of the treatment period, which is approximately 24 weeks, your progress will be evaluated.

The evaluation will focus on the reduction in spleen size and symptom improvement, as well as any side effects experienced during the study.

6 follow-up

After completing the treatment, there will be a follow-up period to monitor your health and any long-term effects of the treatment.

This may include additional assessments and communication with your healthcare provider.

Who Can Join the Study?

  • Must have one of the following conditions: Primary Myelofibrosis, Post-Essential Thrombocythaemia Myelofibrosis, or Post-Polycythaemia Vera Myelofibrosis.
  • Must have a platelet count (a type of blood cell) of less than 50,000 per microliter at the time of screening.
  • Must be classified as Intermediate-1, Intermediate-2, or High-Risk according to the Dynamic International Prognostic Scoring System.
  • Must have a palpable spleen enlargement of at least 5 cm below the rib cage as determined by a physical exam.
  • Must have a Total Symptom Score (TSS) of 10 or more, or a single symptom score of 5 or more, or two symptoms scoring 3 or more, including symptoms like left upper quadrant pain, bone pain, itching, or night sweats.
  • Must be at least 18 years old.
  • Must have an Eastern Cooperative Oncology Group performance status of 0 to 2, which measures daily living abilities.
  • Must have a peripheral blast count (immature blood cells) of less than 10% during the screening period.
  • Must have an absolute neutrophil count (a type of white blood cell) of at least 500 per microliter.
  • Must have a left ventricular cardiac ejection fraction of 50% or more, which measures heart function, as determined by an echocardiogram or similar test.
  • Must have adequate liver and kidney function, with specific blood test results within certain limits.
  • Must have adequate blood clotting ability, as shown by specific blood tests.
  • If capable of having children, must agree to use highly effective birth control methods during the study.
  • Must be willing to have and able to tolerate frequent MRI or CT scans during the study.
  • Must be able to understand and willing to complete symptom assessments using a patient-reported outcome tool.
  • Must provide signed informed consent to participate in the study.

Who Cannot Join the Study?

  • Patients with any other serious medical condition that might affect their participation in the study.
  • Patients who have had a recent heart attack or stroke.
  • Patients with uncontrolled high blood pressure.
  • Patients with severe liver disease.
  • Patients with severe kidney disease.
  • Patients who are pregnant or breastfeeding.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had an allergic reaction to the study medication or similar medications in the past.
  • Patients with a history of certain types of cancer.
  • Patients with active infections that require treatment.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
Centre Hospitalier Universitaire De Nimes Nimes France
Centre Hospitalier Universitaire De Poitiers Poitiers France
Azienda Sanitaria Universitaria Friuli Centrale Udine Italy
Hospital Universitario 12 De Octubre Madrid Spain
Hospital General Universitario Morales Meseguer Murcia Spain
Istituto Tumori Bari Giovanni Paolo II Bari Italy
Fondazione IRCCS San Gerardo Dei Tintori Monza Italy
Azienda Socio Sanitaria Territoriale Dei Sette Laghi Varese Italy
Fejer Varmegyei Szent Gyoergy Egyetemi Oktato Korhaz Szekesfehervar Hungary
University Multiprofile Hospital For Active Treatment Saint Georgi EAD Plovdiv Bulgaria
Fakultni Nemocnice Plzen Plzen Czechia
Pratia Hematologia Sp. z o.o. Katowice Poland
Somogy Varmegyei Kaposi Mor Oktato Korhaz Kaposvar Hungary
Spitalul Clinic Coltea Bucharest Romania
Uniwersytecki Szpital Kliniczny W Bialymstoku Bialystok Poland
Virgen del Rocío University Hospital Sevilla Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Hospital Clinic De Barcelona Barcelona Spain
Fakultni Nemocnice Brno Brno Czechia
Fakultni Nemocnice Kralovske Vinohrady Prague Czechia
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie Cracow Poland
Institute Of Oncology Prof. Dr. Ion Chiricuta Cluj-Napoca Cluj Napoca Romania
Samodzielny Publiczny Szpital Kliniczny Nr 1 W Lublinie Lublin Poland
Azienda Ospedaliero-Universitaria Maggiore Della Carita Novara Italy
Onco Card S.R.L. Brasov Romania
University Hospital Olomouc Olomouc Czechia
Centre Hospitalier Lyon Sud Pierre Benite France
Hopital Beaujon Clichy France
Universita Degli Studi Di Brescia Brescia Italy
Multi-profile Hospital for Active Treatment Heart and Brain EAD Pleven Bulgaria
National Specialised Hospital For Active Treatment Of Haematological Diseases Sofia Bulgaria
Fundeni Clinical Institute Bucharest Romania
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Abotdxt Owoqxyklrsg Orxcmnyp Rxtwhpt Vhcgv Stfhb Cqhdmqny Palermo Italy
Seahsxy Sjowxauxxcrwvyj Ivr Jujskysc Spkhndsqsxrj W Nyocq Szhpb Nowy Sacz Poland
Iyyxgfsk Rqtwajxmy Pue Ld Svstwk Dzt Tdfgoa Drlo Azambxw Iymt Seuwat Meldola Italy
Aetepby Ozcdidrchir Unwfvclacimwu Cglebcytaxus Dfmcc Sjybfb E Dqhlj Sedmkcm De Timogy Turin Italy
Avbqapj Uwldl Swuyqlrbq Leagpk Dp Biwecrp Bologna Italy
Mreiuses Mzukujb Arcvmoj Pleven Bulgaria
Umbndfjmzfhxsr Ccpulwg Kidqvmmsc Gdansk Poland
Ulnalsudtp Doqqa Sggko Dn Rakw Lr Skbnozxc Rome Italy
Hlhnwjvc Vrpt dgukqbtw Barcelona Spain
Sripafcprav Pihkaauin Sjjstrf Krqdraygd Iavjhzkxuca Mqvzvigcelu Smb W Ktfkglynen Katowice Poland

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Bulgaria Bulgaria
Not recruiting
10.08.2019
Czechia Czechia
Not recruiting
10.08.2019
France France
Not recruiting
10.08.2019
Hungary Hungary
Not recruiting
10.08.2019
Italy Italy
Not recruiting
10.08.2019
Poland Poland
Not recruiting
10.08.2019
Romania Romania
Not recruiting
10.08.2019
Spain Spain
Not recruiting
10.08.2019

Trial locations

Pacritinib is a medication being studied for its potential to help people with a type of blood cancer called myelofibrosis, especially those who have very low platelet counts. Myelofibrosis is a condition where the bone marrow, which is responsible for producing blood cells, becomes scarred and doesn’t work properly. This can lead to an enlarged spleen and other symptoms. Pacritinib is designed to target specific pathways in the body that contribute to the disease, with the goal of reducing the size of the spleen and improving symptoms.

Physician’s Choice Therapy refers to a treatment option where the doctor selects the best available therapy for the patient based on their individual needs and medical history. This could include a variety of medications or therapies that are already approved and commonly used for treating myelofibrosis. The idea is to compare the effectiveness of pacritinib against these standard treatments to see which works better for patients with severe thrombocytopenia, a condition where there are fewer platelets in the blood than normal.

Primary Myelofibrosis – Primary Myelofibrosis is a chronic disorder where the bone marrow is replaced by fibrous tissue, leading to a decrease in blood cell production. This condition often begins with an enlarged spleen and progresses with symptoms such as fatigue, weakness, and anemia. Over time, the fibrous tissue in the bone marrow increases, further reducing the production of blood cells. Patients may experience weight loss, night sweats, and bone pain as the disease advances. The condition can lead to significant changes in blood cell counts, including low red blood cells, white blood cells, and platelets. As the disease progresses, it can cause complications related to blood cell deficiencies.

Post-Essential Thrombocythaemia Myelofibrosis – Post-Essential Thrombocythaemia Myelofibrosis is a condition that develops in some patients who have had essential thrombocythaemia, a disorder characterized by an overproduction of platelets. Over time, the bone marrow becomes fibrotic, leading to a reduction in blood cell production. This progression results in symptoms such as fatigue, anemia, and an enlarged spleen. Patients may also experience weight loss, night sweats, and bone pain. The disease causes significant changes in blood cell counts, including low levels of red blood cells and platelets. As the condition advances, it can lead to complications associated with blood cell deficiencies.

Post-Polycythaemia Vera Myelofibrosis – Post-Polycythaemia Vera Myelofibrosis occurs in some individuals who have had polycythaemia vera, a condition where there is an overproduction of red blood cells. Over time, the bone marrow becomes fibrotic, reducing its ability to produce blood cells. This leads to symptoms such as fatigue, anemia, and an enlarged spleen. Patients may also experience weight loss, night sweats, and bone pain. The disease results in significant changes in blood cell counts, including low levels of red blood cells and platelets. As the condition progresses, it can cause complications related to blood cell deficiencies.

Trial ID:
2024-515953-52-00
Protocol code:
PAC303
NCT ID:
NCT03165734
Trial Phase:
Therapeutic confirmatory (Phase III)

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