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

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Expanded Access
Eligible Ages	12 Years - 99 Years
Gender	All

More Inclusion & Exclusion 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. 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.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT04745637
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Novartis Pharmaceuticals
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	N/A
Principal Investigator Affiliation	N/A
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry
Overall Status	Available
Countries
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Primary Myelofibrosis (PMF), Post Polycythemia Myelofibrosis (PPV MF), Thrombocythemia Myelofibrosis (PET-MF)

Contact Information

This trial has no sites locations listed at this time. If you are interested in learning more, you can contact the trial's primary contact:

Novartis Pharmaceuticals

novartis.email@novartis.com

+41613241111

For additional contact information, you can also visit the trial on clinicaltrials.gov.

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