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JAK2 Inhibitors RUXOLITINIB in Patients With Myelofibrosis

Primary Purpose

Myelofibrosis

Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Ruxolotinib
Sponsored by
French Innovative Leukemia Organisation
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myelofibrosis focused on measuring JAK2 inhibitor RUXOLITINIB, Primary or secondary myelofibrosis

Eligibility Criteria

18 Years - 69 Years (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • ROBIN

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

RUXOLOTINIB

Arm Description

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

Outcomes

Primary Outcome Measures

DFS
DFS is defined as the probability to be alive and in remission

Secondary Outcome Measures

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)
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

Full Information

First Posted
December 21, 2012
Last Updated
July 26, 2022
Sponsor
French Innovative Leukemia Organisation
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1. Study Identification

Unique Protocol Identification Number
NCT01795677
Brief Title
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

2. Study Status

Record Verification Date
July 2022
Overall Recruitment Status
Completed
Study Start Date
December 2012 (undefined)
Primary Completion Date
May 2018 (Actual)
Study Completion Date
March 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
French Innovative Leukemia Organisation

4. Oversight

Data Monitoring Committee
Yes

5. 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
Detailed Description
JAK2 inhibitor RUXOLITINIB before allogeneic hematopoietic stem cell transplantation (HSCT) in patients with primary or secondary myelofibrosis

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelofibrosis
Keywords
JAK2 inhibitor RUXOLITINIB, Primary or secondary myelofibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
78 (Actual)

8. Arms, Groups, and Interventions

Arm Title
RUXOLOTINIB
Arm Type
Experimental
Arm Description
Ruxolotinib : patient with donor HSCT 4 months later patients without donor: ruxolotinib alone
Intervention Type
Drug
Intervention Name(s)
Ruxolotinib
Other Intervention Name(s)
Kakavi
Intervention Description
Ruxolotinib doses calculated with platelets count and P450 cytochrome inhibitor HSCT for patients with donor
Primary Outcome Measure Information:
Title
DFS
Description
DFS is defined as the probability to be alive and in remission
Time Frame
24 months after inclusion
Secondary Outcome Measure Information:
Title
HSCT
Description
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)
Time Frame
24 months after inclusion
Title
PATIENTS CARACTERISTICS
Description
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
Time Frame
24 months after inclusion

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
69 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
MARIE ROBIN, MD
Organizational Affiliation
FIM/GOELAMS
Official's Role
Principal Investigator
Facility Information:
Facility Name
ROBIN
City
Paris
ZIP/Postal Code
75010
Country
France

12. IPD Sharing Statement

Plan to Share IPD
No
Links:
URL
http://www.filo-leucemie.org
Description
FILO internet site

Learn more about this trial

JAK2 Inhibitors RUXOLITINIB in Patients With Myelofibrosis

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