JAK2 Inhibitors RUXOLITINIB in Patients With Myelofibrosis

Myelofibrosis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01795677
• Other study ID #: JAK ALLO STUDY
• Status: Completed
• Phase: Phase 2
• Start date: December 2012
• Est. completion date: March 2019

Study information

• Verified date: July 2022
• Source: French Innovative Leukemia Organisation
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 78
• Est. completion date: March 2019
• Est. primary completion date: May 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 69 Years

Eligibility

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

Related Conditions & MeSH terms

• Myelofibrosis
• Primary Myelofibrosis

Intervention

Drug:
• Ruxolotinib
Ruxolotinib doses calculated with platelets count and P450 cytochrome inhibitor HSCT for patients with donor

Locations

Country	Name	City	State
France	ROBIN	Paris

Sponsors (1)

Lead Sponsor	Collaborator
French Innovative Leukemia Organisation

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	DFS	DFS is defined as the probability to be alive and in remission	24 months after inclusion
Secondary	HSCT	Rate of pre-graft splenectomy; Co-morbidity score defined by Sorror et al before RUXOLITINIB and after 4-month treatment just before transplantation; Post-graft haematological recovery: time to neutrophil engraftment, platelet and red blood cells transfusion independency; Acute GVHD grade II-IV incidence; Chronic GVHD incidence; Overall survival, disease-free survival, non-relapse mortality; JAK2V617E allele burden and status at registration, 3, 7, 16 months after inclusion (centralization)	24 months after inclusion
Secondary	PATIENTS CARACTERISTICS	Patients with and without donor; Rate of patients with donor who benefit from a transplantation: Comorbidity score at registration and after 3 months; Platelet and red blood cells transfusion independency; Performance status evolution (ECOG); General symptoms related to myelofibrosis (questionnaire MF SAF); Comparison of haematological response in patients with or without donor; Spleen size evolution; Comparison of quality of life in patients with and without (questionnaire EORTC); Comparison of overall survival in patients with and without donor; Incidence of severe infections; Cytokine measure at registration, 3, and 7 months after inclusion (centralization); MPL JAK status (at registration, centralization	24 months after inclusion

External Links

•   FILO internet site