Back to resultsMAP 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
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
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.
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT04745637
First Posted
February 5, 2021
Last Updated
January 24, 2023
Sponsor
Novartis Pharmaceuticals
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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