JAK2 Inhibitors RUXOLITINIB in Patients With Myelofibrosis

Sponsor

French Innovative Leukemia Organisation (Other)

Overall Status

Completed

CT.gov ID

NCT01795677

Collaborator

(none)

Enrollment

Location

Arm

74.9

Duration (Months)

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Myelofibrosis	Drug: Ruxolotinib	Phase 2

Detailed Description

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

Study Design

Study Type:

Interventional

Actual Enrollment :

78 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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 Start Date :

Dec 1, 2012

Actual Primary Completion Date :

May 1, 2018

Actual Study Completion Date :

Mar 1, 2019

Arms and Interventions

Arm	Intervention/Treatment
Experimental: RUXOLOTINIB Ruxolotinib : patient with donor HSCT 4 months later patients without donor: ruxolotinib alone	Drug: Ruxolotinib Ruxolotinib doses calculated with platelets count and P450 cytochrome inhibitor HSCT for patients with donor Other Names: Kakavi

Arm

Intervention/Treatment

Experimental: RUXOLOTINIB

Ruxolotinib : patient with donor HSCT 4 months later patients without donor: ruxolotinib alone

Drug: Ruxolotinib

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

Other Names:

Kakavi

Outcome Measures

Primary Outcome Measures

DFS [24 months after inclusion]
DFS is defined as the probability to be alive and in remission

Secondary Outcome Measures

HSCT [24 months after inclusion]
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)
PATIENTS CARACTERISTICS [24 months after inclusion]
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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 69 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	ROBIN	Paris		France	75010

Sponsors and Collaborators

French Innovative Leukemia Organisation

Investigators

Principal Investigator: MARIE ROBIN, MD, FIM/GOELAMS

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

FILO internet site

Publications

None provided.

Responsible Party:

French Innovative Leukemia Organisation

ClinicalTrials.gov Identifier:

NCT01795677

Other Study ID Numbers:

JAK ALLO STUDY

First Posted:

Feb 21, 2013

Last Update Posted:

Jul 27, 2022

Last Verified:

Jul 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Keywords provided by French Innovative Leukemia Organisation

JAK2 inhibitor RUXOLITINIB

Primary or secondary myelofibrosis

Additional relevant MeSH terms:

Primary Myelofibrosis

Study Results

No Results Posted as of Jul 27, 2022