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MAP to Provide Access to Ruxolitinib, for Patients With PMF or PPV MF or PET-MF

Primary Purpose

Primary Myelofibrosis (PMF), Post Polycythemia Myelofibrosis (PPV MF), Thrombocythemia Myelofibrosis (PET-MF)

Status
Available
Phase
Locations
Study Type
Expanded Access
Intervention
Ruxolitinib
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Primary Myelofibrosis (PMF) focused on measuring Primary Myelofibrosis, PMF, Post Polycythemia Myelofibrosis, PPV MF, Post Essential Thrombocythemia Myelofibrosis, PET-MF, Ruxolitinib, Jakavi

Eligibility Criteria

12 Years - 99 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion criteria

Patients eligible for inclusion in this Treatment Plan have to meet all of the following criteria:

Written patient informed consent must be obtained prior to start of treatment. Patients ineligible for other ruxolitinib trials could be eligible for this MAP.

  1. Patients must be ≥12 years and diagnosed with PMF, according to the 2016 revised International Standard Criteria (Arber et al. 2016), PPV MF or PET-MF (Barosi 2008), irrespective of JAK2 mutation status.
  2. Patients with a peripheral blood blast percentage count of < 10%.
  3. Patients must have recovered or stabilized sufficiently from any adverse drug reactions associated with prior treatments before beginning treatment with ruxolitinib.

    • Patients who were pre-treated with Fedratinib should have complete physical examination including full neurologic examination and cardiology assessment including thiamine levels. MRI of the brain should be considered if indicated based on the signs or symptoms.

Exclusion criteria

Patients eligible for this Treatment Plan must not meet any of the following criteria:

  1. History of hypersensitivity to any drugs or metabolites of similar chemical classes as ruxolitinib.
  2. Presence of an active uncontrolled infection including significant bacterial, fungal, viral
  3. History of progressive multifocal leuko-encephalopathy.
  4. Presence of severely impaired renal function defined by serum creatinine > 2 mg/dL (>176.8 μmol/L), or have estimated creatinine clearance < 30 ml/min measured or calculated by Cockroft Gault equation.
  5. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant,

    • women whose sexual orientation precludes intercourse with a male partner.
    • women whose partners have been sterilized by vasectomy or other means.
    • using a highly effective method of birth control
  6. Not able to understand and to comply with treatment instructions and requirements.

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    February 5, 2021
    Last Updated
    January 24, 2023
    Sponsor
    Novartis Pharmaceuticals
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04745637
    Brief Title
    MAP to Provide Access to Ruxolitinib, for Patients With PMF or PPV MF or PET-MF
    Official Title
    Managed Access Program (MAP) to Provide Access to Ruxolitinib, for Patients With Primary Myelofibrosis (PMF) or Post Polycythemia Myelofibrosis (PPV MF) or Post-Essential Thrombocythemia Myelofibrosis (PET-MF)
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    January 2023
    Overall Recruitment Status
    Available
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Novartis Pharmaceuticals

    4. Oversight

    5. Study Description

    Brief Summary
    The purpose of this Cohort Treatment Plan is to allow access to ruxolitinib for eligible patients diagnosed with PMF, PPV MF or PET MF. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations. The requesting Treating Physician submitted a request for access to drug (often referred to as Compassionate Use) to Novartis which was reviewed and approved by the medical team experienced with the drug and indication.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Primary Myelofibrosis (PMF), Post Polycythemia Myelofibrosis (PPV MF), Thrombocythemia Myelofibrosis (PET-MF)
    Keywords
    Primary Myelofibrosis, PMF, Post Polycythemia Myelofibrosis, PPV MF, Post Essential Thrombocythemia Myelofibrosis, PET-MF, Ruxolitinib, Jakavi

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    Ruxolitinib
    Intervention Description
    provide access to ruxolitinib, for Patients with Primary Myelofibrosis (PMF) or Post Polycythemia Myelofibrosis (PPV MF) or Post-Essential Thrombocythemia Myelofibrosis (PET-MF)

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    12 Years
    Maximum Age & Unit of Time
    99 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion criteria Patients eligible for inclusion in this Treatment Plan have to meet all of the following criteria: Written patient informed consent must be obtained prior to start of treatment. Patients ineligible for other ruxolitinib trials could be eligible for this MAP. Patients must be ≥12 years and diagnosed with PMF, according to the 2016 revised International Standard Criteria (Arber et al. 2016), PPV MF or PET-MF (Barosi 2008), irrespective of JAK2 mutation status. Patients with a peripheral blood blast percentage count of < 10%. Patients must have recovered or stabilized sufficiently from any adverse drug reactions associated with prior treatments before beginning treatment with ruxolitinib. Patients who were pre-treated with Fedratinib should have complete physical examination including full neurologic examination and cardiology assessment including thiamine levels. MRI of the brain should be considered if indicated based on the signs or symptoms. Exclusion criteria Patients eligible for this Treatment Plan must not meet any of the following criteria: History of hypersensitivity to any drugs or metabolites of similar chemical classes as ruxolitinib. Presence of an active uncontrolled infection including significant bacterial, fungal, viral History of progressive multifocal leuko-encephalopathy. Presence of severely impaired renal function defined by serum creatinine > 2 mg/dL (>176.8 μmol/L), or have estimated creatinine clearance < 30 ml/min measured or calculated by Cockroft Gault equation. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, women whose sexual orientation precludes intercourse with a male partner. women whose partners have been sterilized by vasectomy or other means. using a highly effective method of birth control Not able to understand and to comply with treatment instructions and requirements.
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Novartis Pharmaceuticals
    Phone
    +41613241111
    Email
    novartis.email@novartis.com
    First Name & Middle Initial & Last Name or Official Title & Degree
    Novartis Pharmaceuticals

    12. IPD Sharing Statement

    Learn more about this trial

    MAP to Provide Access to Ruxolitinib, for Patients With PMF or PPV MF or PET-MF

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