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

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Available

CT.gov ID

NCT04745637

Collaborator

(none)

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Primary Myelofibrosis (PMF) Post Polycythemia Myelofibrosis (PPV MF) Thrombocythemia Myelofibrosis (PET-MF)	Drug: Ruxolitinib

Study Design

Study Type:

Expanded Access

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)

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years to 99 Years

Sexes Eligible for Study:

All

Accepts 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.

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,