Blood and Lymph Conditions

All Conditions

Neoplasms

Heart and Blood Diseases

Rare Diseases

Myeloproliferative Disorders

Polycythemia Vera

Polycythemia

Primary Myelofibrosis

Thrombocytosis

Thrombocythemia, Essential

Bone Marrow Diseases

Hematologic Diseases

Bone Marrow Neoplasms

Hematologic Neoplasms

Blood Platelet Disorders

Hemostatic Disorders

Blood Coagulation Disorders

Hemorrhagic Disorders

Chronic Graft Versus Host Disease

Chronic Myeloproliferative Disorders

Essential Thrombocythemia

SB1518 taken orally daily for 28 consecutive days in a 28-day cycle

SB1518

John Seymour, M.D.

Andrew Roberts, M.D.

Bik To, MD

Rami Komrokji, MD

Martha Wadleigh, MD

Ruben Mesa, MD

The study consists of two phases: The first portion of the study is a Phase 1 dose escalation study to determine the maximum tolerated dose and the dose limiting toxicities of SB1518 when given as a single agent orally once daily in subjects with Chronic Idiopathic Myelofibrosis (CIMF) regardless of their JAK2 mutational status. The second portion of the study is a Phase 2 study to define the efficacy and safety profile of single agent SB1518 at the recommended dose in subjects with CIMF.

A Phase 1/2 Study of Oral SB1518 in Subjects With Chronic Idiopathic Myelofibrosis

S*BIO

Symptoms and General Pathology

Syndrome

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Preleukemia

Myelodysplastic Syndromes

Leukemia, Myelomonocytic, Chronic

Leukemia, Myelomonocytic, Juvenile

Neoplasms by Histologic Type

Chronic Disease

Disease Attributes

Myelodysplastic-Myeloproliferative Diseases

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Chronic Myeloid Leukemia

Acute Graft Versus Host Disease

Chronic Myelomonocytic Leukemia

Juvenile Myelomonocytic Leukemia

Myelodysplastic/myeloproliferative Disease

Srdan Verstovsek, M.D, Ph.D

H. Joachim Deeg, M.D

Olatoyosi M. Odenike, M.D.

This study consists of two phases: the first portion of the study is a Phase 1 dose escalation study to determine the maximum tolerated dose and the dose limiting toxicities of SB1518 when given as a single agent orally once daily in subjects with advanced myeloid malignancies; the second portion of the study is a Phase 2 study to define the efficacy and safety profile of single-agent SB1518 at the recommended dose in subjects with chronic idiopathic myelofibrosis (CIMF).

A Phase 1/2 Study of SB1518 for the Treatment of Advanced Myeloid Malignancies

The effect of pacritinib treatment during 3 to 4 cycles before allo-SCT on engraftment 6 months (day +180) post allo-SCT in MF patients.

Patients receive up to 4 cycles of pacritinib before allo-SCT

Pacritinib

Peter AW te Boekhorst, M.D. PhD

The only curative treatment for patients with myelofibrosis (MF) is allogeneic stem cell transplantation (SCT). Treatment with JAK2 inhibitors like pacritinib improves condition of MF patients, decreases spleen size and might diminish graft-versus-host disease (GvHD), thereby improving the outcome of SCT.

Despite recent new therapeutic options, allogeneic Stem Cell Transplantation remains the only curative option in patients with Myelofibrosis. Therefore, optimalization of this therapy remains a major challenge. Improvement of the clinical condition of these patients, decreasing spleen size can be accomplished by JAK2 inhibitor treatment and might improve SCT outcome. In addition, selective JAK2 inhibitors might modulate GvHD which can also add to improved SCT outcome. Also, decreasing the burden/activity of the disease before allo-SCT might also improve final disease response. The first, limited, clinical data of ruxolitinib treatment before allo-SCT show controversial effects on the outcome of SCT. Therefore, additional prospective clinical trials have to be done to establish the role of JAK2 inhibition before allogeneic SCT. Since ruxolitinib has considerable myelosuppressive effects which might limit the clinical use in some MF patients, other selective JAK2 inhibitors might be useful in this setting. Pacritinib, as a JAK2/FLT3 inhibitor, has a very potent JAK2 inhibitory activity without myelosuppressive effects and might therefore be more suitable than ruxolitinib. The major possible side effects are gastro-intestinal and can be managed with medication. In several studies, pacritinib has shown to be effective in decreasing spleen size and has shown to improve clinical condition of patients. Therefore, this compound seems promising in improving the outcome of allo-SCT. Although pacritinib causes no inhibition of JAK1 activity and therefore might have limited effects in decreasing inflammatory response, this might also be of benefit since a "withdrawal syndrome" as has been described after cessation of ruxolitinib is not to be expected.

With this trial, using pacritinib treatment before allo-SCT, the issue of improvement of SCT outcome will be investigated.

Myelofibrosis Treated With Pacritinib Before aSCT. (HOVON134MF)

A Phase II Trial in Patients With Myelofibrosis (Primary, Post-ET or Post PV-MF) Treated With the Selective JAK2 Inhibitor Pacritinib Before Reduced-intensity Conditioning Allogeneic Stem Cell Transplantation

Failure can be defined by one of the following parameters:

* Primary graft failure
* Acute graft versus host disease grade 3-4
* Secondary graft failure
* Death, from any cause

Progression free survival as time between registration or SCT until progression/relapse or death from any cause

Over all survival, calculated from either registration or SCT.

Death due to the disease or after progression

Quality of life during and after treatment will be recorded by use of the MPN-SAF questionnaire.

From a total of 27 questions, the scores (from 0 to 10) from the following 10 questions are added and subsequently averaged to come to a total quality of life score.

* fatigue
* satisfction after a meal
* stomach complaints
* not able to perform activities
* concentration problems
* night sweat
* itch
* bone pain
* fever
* sudden weight loss

Dutch Cancer Society

CTI BioPharma

Stichting Hemato-Oncologie voor Volwassenen Nederland

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Phase 1/2 Study of Oral SB1518 in Subjects With Chronic Idiopathic Myelofibrosis	NCT00745550	Myelofibrosis Myeloproliferat... Polycythemia Ve... Essential Throm...	SB1518	18 Years -	S*BIO
A Phase 1/2 Study of SB1518 for the Treatment of Advanced Myeloid Malignancies	NCT00719836	Acute Myelogeno... Chronic Myeloge... Chronic Myelomo... Myelodysplastic... Myelofibrosis	SB1518	18 Years -	S*BIO
Myelofibrosis Treated With Pacritinib Before aSCT. (HOVON134MF)	NCT03645824	Myelofibrosis	Pacritinib	18 Years - 70 Years	Stichting Hemato-Oncologie voor Volwassenen Nederland
A Phase 1/2 Study of SB1518 for the Treatment of Advanced Myeloid Malignancies	NCT00719836	Acute Myelogeno... Chronic Myeloge... Chronic Myelomo... Myelodysplastic... Myelofibrosis	SB1518	18 Years -	S*BIO
Myelofibrosis Treated With Pacritinib Before aSCT. (HOVON134MF)	NCT03645824	Myelofibrosis	Pacritinib	18 Years - 70 Years	Stichting Hemato-Oncologie voor Volwassenen Nederland

Spots Global Cancer Trial Database for jak2 inhibitor