Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 (INCB018424) Tablets Versus Best Available Care: The RESPONSE Trial

Condition(s) targeted: Polycythemia Vera

Intervention: INC424 tablets (Drug); Best Available Therapy (BAT) (Other)

Phase: Phase 3

Status: Recruiting

Sponsored by: Incyte Corporation

Official(s) and/or principal investigator(s):
Srdan Verstovsek, MD,PhD, Study Director, Affiliation: M.D. Anderson Cancer Center

Overall contact:
William Garrett, MBA, Phone: 302-498-6987, Email: wgarrett@incyte.com

This pivotal phase III trial (CINC424B2301) is designed to compare the efficacy and safety of INC424 to Best Available Therapy (BAT) in subjects with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea (HU).

Official title: Randomized, Open Label, Multicenter Phase III Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 Tablets Versus Best Available Care

Study design: Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Proportion of subjects achieving a response at Week 32. 'Response' is defined as having achieved both: (1) the absence of protocol-defined phlebotomy eligibility and (2) a ≥ 35% reduction from baseline in spleen volume as determined by imaging

Secondary outcome:

Proportion of subjects achieving complete hematologic remission at Week 32.

Proportion of all randomized subjects who both achieve the Week 32 primary response endpoint and maintain response for ≥ 48 weeks from the time that response was initially documented.

Minimum age: 18 Years. Maximum age: N/A. Gender(s): Both.

Criteria:

Inclusion Criteria:

- Subjects diagnosed with PV for at least 24 weeks prior to screening according to the

2008 World Health Organization criteria

- Subjects resistant to or intolerant of hydroxyurea

- Subjects with a phlebotomy requirement

- Subjects with a palpable splenomegaly ≥ 5 cm below the costal margin

- Subjects with elevated white blood cell and/or platelet counts

- Subjects with an ECOG performance status of 0, 1 or 2

Exclusion Criteria:

- Women who are pregnant or nursing

- Subjects with inadequate liver or renal function

- Subjects with significant bacterial, fungal, parasitic, or viral infection requiring

treatment

- Subjects with an active malignancy within the past 5 years, excluding specific skin

cancers

- Subjects with known active hepatitis or HIV positivity

- Subjects who have previously received treatment with a JAK inhibitor

- Subjects being treated with any investigational agent

William Garrett, MBA, Phone: 302-498-6987, Email: wgarrett@incyte.com

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Debrecen, Hungary; Recruiting

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Ft Meyers, Florida, United States; Recruiting

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Winter Park, Florida, United States; Recruiting

Barrington, Illinois, United States; Recruiting

Chicago, Illinois, United States; Recruiting

Joliet, Illinois, United States; Not yet recruiting

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Lafayette, Louisiana, United States; Recruiting

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Starting date: October 2010
Last updated: December 5, 2011