Clinical trial NCT01795677: JAK2 Inhibitors RUXOLITINIB in Patients With Myelofibrosis
Official title: JAK2 Inhibitors RUXOLITINIB in Patients With High or Intermediate Risk Primary or Secondary Myelofibrosis Eligible for Allogeneic Stem Cell Transplantation: a Prospective Multicentric Phase II Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Myelofibrosis
Keywords: JAK2 inhibitor RUXOLITINIB
Primary or secondary myelofibrosis
Lead sponsor: French Innovative Leukemia Organisation
Sponsor class: OTHER
First posted: 2013-02-21
Enrollment: 78
Sex: ALL
Minimum age (years): 18
Maximum age (years): 69
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: JAK2 inhibitor RUXOLITINIB before allogeneic hematopoietic stem cell transplantation (HSCT) in patients with primary or secondary myelofibrosis : a prospective phase II

Detailed description: JAK2 inhibitor RUXOLITINIB before allogeneic hematopoietic stem cell transplantation (HSCT) in patients with primary or secondary myelofibrosis

Eligibility criteria: Inclusion Criteria:

* Age between 18 and 69 years
* No comorbidity contraindicating the transplantation :

  * Severe respiratory failure defined as dyspnea grade III or more
  * Severe cardiac failure defined as EF \< or = 30%
  * Severe renal failure defined as creatinine clearance \< 30 ml/min or dialysis
  * Dementia or non-ability to give informed consent for the protocol
  * Major alteration of performance status defined as ECOG \> 2
  * Severe liver disease defined as a cirrhosis or bilirubin \> 2 x ULN, or AST/ALT \> 5 x ULN
* Primary or secondary myelofibrosis diagnosed according to WHO definition (Tefferi, et al 2007)
* Palpable splenomegaly or splenomegaly measured by any imagery (maximum size\> 15 cm by ultrasound scan, Magnetic Resonance Imaging or computer tomography)
* Disease if intermediate or high risk according to published criteria and summarized as follows:

At least one criterion among the following:

* Haemoglobin \< 100 gr/L (unrelated to medication toxicity)
* Leucocytes \< 4 G/L (unrelated to medication toxicity) or \> 25 G/L
* Poor prognosis cytogenetics : complex karyotype, abnormalities of chromosomes 5, 7 or 17 , +8, 12p-, inv(3), 11q23

Two criteria among the following criteria :

* General symptoms (weight lost \> 10% in less than 6 months, night swears, specific fever \> 37.5°C)
* Peripheral blastosis \> 1% observed at least twice
* Thrombocytopenia \< 100 G/L (unrelated to treatment toxicity)

Exclusion Criteria:

* Myelofibrosis transformed into acute leukaemia with 20% blasts of more in blood or bone marrow
* Previous treatment with JAK2 inhibitor
* Thrombopenia \< 50 G/L
* Comorbidities contraindicating the transplantation
* Comorbidity score Sorror \> 3
* Pregnant or lactating women

Primary outcomes:
measure: DFS
timeFrame: 24 months after inclusion
Secondary outcomes:
measure: HSCT
timeFrame: 24 months after inclusion
measure: PATIENTS CARACTERISTICS
timeFrame: 24 months after inclusion
Other outcomes:
Locations:
city: Paris
country: France
facility: ROBIN
Study officials:
affiliation: FIM/GOELAMS
name: MARIE ROBIN, MD
role: PRINCIPAL_INVESTIGATOR
References: