NCT05269771

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.

Trial Health

65
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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
8 countries

25 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 16, 2022

Completed
20 days until next milestone

First Posted

Study publicly available on registry

March 8, 2022

Completed
Last Updated

November 5, 2025

Status Verified

October 1, 2025

First QC Date

February 16, 2022

Last Update Submit

October 31, 2025

Conditions

Keywords

INC424RuxolitinibJakaviMAPManage access program

Interventions

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.

Also known as: Jakavi

Eligibility Criteria

Age12 Years - 99 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Male or female aged 12 or over
  • Confirmed diagnosis of PV based on World Health Organization (WHO) criteria
  • Patients must have a treatment history for PV that meets the definition of resistance or intolerance to hydroxyurea (HU) or other cytoreductive therapy
  • Patients with a peripheral blood blast count of 0% at screening
  • Patients must have recovered or stabilized sufficiently from adverse drug reactions associated with prior treatments before beginning treatment with ruxolitinib
  • 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.

You may not qualify if:

  • 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 (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.
  • History of progressive multifocal leuko-encephalopathy (PML).
  • 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, 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.
  • Pregnancy
  • Not able to understand and to comply with treatment instructions and requirements

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (25)

Novartis Investigative Site

Orange, New South Wales, 2800, Australia

Location

Novartis Investigative Site

Leuven, 3000, Belgium

Location

Novartis Investigative Site

Calgary, Alberta, T2N 2T9, Canada

Location

Novartis Investigative Site

Tehran, 11367, Iran

Location

Novartis Investigative Site

Kfar Saba, Israel, 44281, Israel

Location

Novartis Investigative Site

Kfar Saba, 44281, Israel

Location

Novartis Investigative Site

Rehovot, 7610001, Israel

Location

Novartis Investigative Site

Tel Aviv, 6423906, Israel

Location

Novartis Investigative Site

Tel Giborim Holon, 58100, Israel

Location

Novartis Investigative Site

Groningen, 9700RB, Netherlands

Location

Novartis Investigative Site

Gdansk, 80-214, Poland

Location

Novartis Investigative Site

Katowice, 40-027, Poland

Location

Novartis Investigative Site

Krakow, 31-501, Poland

Location

Novartis Investigative Site

Warsaw, 00-791, Poland

Location

Novartis Investigative Site

Warsaw, 04-141, Poland

Location

Novartis Investigative Site

Wałbrzych, 58-309, Poland

Location

Novartis Investigative Site

Wroclaw, 50-367, Poland

Location

Novartis Investigative Site

Hereford, HR1 2ER, United Kingdom

Location

Novartis Investigative Site

Kent, CT9 4AN, United Kingdom

Location

Novartis Investigative Site

London, NW3 2PF, United Kingdom

Location

Novartis Investigative Site

London, SE5 8AD, United Kingdom

Location

Novartis Investigative Site

London, WC1E 6HX, United Kingdom

Location

Novartis Investigative Site

Oxford, OX3 7LJ, United Kingdom

Location

Novartis Investigative Site

Poole, BH15 2JB, United Kingdom

Location

Novartis Investigative Site

Salisbury, SP2 8BJ, United Kingdom

Location

MeSH Terms

Conditions

Polycythemia Vera

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Bone Marrow NeoplasmsHematologic NeoplasmsNeoplasms by SiteNeoplasmsBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesMyeloproliferative Disorders

Study Design

Study Type
expanded access
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 16, 2022

First Posted

March 8, 2022

Last Updated

November 5, 2025

Record last verified: 2025-10

Locations