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JAK2 Inhibitors RUXOLITINIB in Patients With Myelofibrosis

This study is currently recruiting participants.
Verified February 2013 by Groupe Ouest Est d'Etude des Leucémies et Autres Maladies du Sang GOELAMS
Sponsor:
Information provided by (Responsible Party):
Groupe Ouest Est d'Etude des Leucémies et Autres Maladies du Sang GOELAMS
ClinicalTrials.gov Identifier:
NCT01795677
First received: December 21, 2012
Last updated: February 18, 2013
Last verified: February 2013
History of Changes
  Purpose

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


Condition Intervention Phase
Myelofibrosis
 Drug: Ruxolotinib
 Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Groupe Ouest Est d'Etude des Leucémies et Autres Maladies du Sang GOELAMS:

Primary Outcome Measures:
  • DFS [ Time Frame: 24 months after inclusion ] [ Designated as safety issue: Yes ]
    DFS is defined as the probability to be alive and in remission


Secondary Outcome Measures:
  • HSCT [ Time Frame: 24 months after inclusion ] [ Designated as safety issue: Yes ]
    • 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 [ Time Frame: 24 months after inclusion ] [ Designated as safety issue: Yes ]

    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


Estimated Enrollment: 80
Study Start Date: December 2012
Estimated Study Completion Date: December 2014
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: ?

Detailed Description:

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

  Eligibility

Ages Eligible for Study:   18 Years to 69 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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

Combination of both 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

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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01795677

Contacts
Contact: Marie ROBIN, MD 33142499639 marie.robin@sls.aphp.fr
Contact: Valerie ROLLAND NEYRET, CRA 33 476765096 VRolland-neyret@chu-grenoble.fr

Locations
France
ROBIN Recruiting
Paris, France, 75010
Contact: Valerie ROLLAND NEYRET, Mrs     +3476765096     vrOlland-neyret@chu-grenoble.fr    
Principal Investigator: Marie ROBIN, MD            
Sponsors and Collaborators
Groupe Ouest Est d'Etude des Leucémies et Autres Maladies du Sang GOELAMS
Investigators
Principal Investigator: MARIE ROBIN, MD FIM/GOELAMS
  More Information

Additional Information:
Goelams internet site  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: Groupe Ouest Est d'Etude des Leucémies et Autres Maladies du Sang GOELAMS
ClinicalTrials.gov Identifier: NCT01795677     History of Changes
Other Study ID Numbers: JAK ALLO STUDY
Study First Received: December 21, 2012
Last Updated: February 18, 2013
Health Authority: France: L’Agence nationale de sécurité du médicament et des produits de santé

Keywords provided by Groupe Ouest Est d'Etude des Leucémies et Autres Maladies du Sang GOELAMS:
JAK2 inhibitor RUXOLITINIB
Primary or secondary myelofibrosis

Additional relevant MeSH terms:
Primary Myelofibrosis
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases

ClinicalTrials.gov processed this record on May 19, 2013