Official Title
Treatment Plan for Managed Access Program (MAP) to Provide Access to Ruxolitinib, for Patients With Polycythemia Vera (PV)
Brief Summary

The purpose of this Cohort Treatment Plan is to allow access to ruxolitinib for eligible patients diagnosed with Polycythemia Vera. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

Detailed Description

The requesting Treating Physician must submit a request for access to drug (often referred to
as Compassionate Use) to Novartis which will review and approve by the medical team
experienced with the drug and indication.

Available
Individual Patients
Polycythemia Vera

Drug: Ruxolitinib

Patients may self-administer daily ruxolitinib tablets qd or bid orally, without regard to food, in accordance with specified dosing schedule provided by the investigator.
Other Name: Jakavi

Eligibility Criteria

Inclusion Criteria:

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

1. Male or female aged 12 or over

2. Confirmed diagnosis of PV based on World Health Organization (WHO) criteria

3. Patients must have a treatment history for PV that meets the definition of resistance
or intolerance to hydroxyurea (HU) or other cytoreductive therapy

4. Patients with a peripheral blood blast count of 0% at screening

5. Patients must have recovered or stabilized sufficiently from adverse drug reactions
associated with prior treatments before beginning treatment with ruxolitinib

6. Patients with an Eastern Cooperative Oncology Group (ECOG) score of 0, 1 or 2 Written
patient informed consent must be obtained prior to start of treatment.

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 (including CMV, EBV, HHV-6, HBV, HCV, BK or HIV) or parasitic infection
requiring treatment. Infections are considered controlled if appropriate therapy has
been instituted and, at the time of screening, no signs of progression are present.
Progression of infection is defined as hemodynamic instability attributable to sepsis,
new symptoms, worsening physical signs or radiographic findings attributable to
infection. Persisting fever without other signs or symptoms will not be interpreted as
progressing infection. Patients with inactive chronic infection (without viral
replication) can be enrolled.

3. History of progressive multifocal leuko-encephalopathy (PML).

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, UNLESS they are

- 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 (i.e. one that results in a less
than 1% per year failure rate when used consistently and correctly, such as
implants, injectables, combined oral contraceptives, and some intrauterine
devices (IUDs); periodic abstinence (e.g. calendar, ovulation, symptothermal,
post-ovulation methods) is not acceptable throughout the period of treatment and
30 days after treatment discontinuation.

Any female aged 8 years and above is to be treated as a woman of child-bearing
potential.

Women are considered post-menopausal and not of child bearing potential if they have
had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile
(e.g. age appropriate, history of vasomotor symptoms) or six months of spontaneous
amenorrhea with serum FSH levels > 40 mIU/mL [for US only: and estradiol < 20 pg/mL]
or have had surgical bilateral oophorectomy (with or without hysterectomy) at least
six weeks ago. In the case of oophorectomy alone, only when the reproductive status of
the woman has been confirmed by follow up hormone level assessment is she considered
not of child bearing potential.

6. Pregnancy

7. Not able to understand and to comply with treatment instructions and requirements

Eligibility Gender
All
Eligibility Age
Minimum: 12 Years ~ Maximum: 99 Years
Contacts

Novartis Pharmaceuticals
+41613241111
novartis.email@novartis.com

Novartis Pharmaceuticals

Novartis Pharmaceuticals
NCT Number
Keywords
INC424
ruxolitinib
Jakavi
MAP
Manage access program
MeSH Terms
Polycythemia Vera
Polycythemia