Open Label Ruxolitinib (INCB018424) in Patients With Myelofibrosis and Post Polycythemia Vera/Essential Thrombocythemia Myelofibrosis

NCT ID: NCT00509899

Last Updated: 2018-03-12

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

154 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-06-30

Study Completion Date

2017-02-28

Brief Summary

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To determine the safety, tolerability and effectiveness of ruxolitinib (INCB018424), administered orally to patients with Primary Myelofibrosis (PMF), Post Polycythemia Vera Myelofibrosis (PPV-MF) and Essential Thrombocythemia Myelofibrosis (PET-MF).

Detailed Description

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This is a multicenter, open-label, non-randomized, dose escalation study of ruxolitinib, a small molecule Janus kinase (JAK) inhibitor, administered orally to patients with PMF, PPEV-MF or PET-MF. The study is comprised of 3 parts:

Part 1: Dose escalation and determination of maximum tolerated dose (complete).

Part 2: Exploration of alternative dosing schedules (complete).

Part 3: Further evaluation of selected dose regimens, including additional response measures to explore effect of ruxolitinib on symptoms and other parameters including daily physical activity and long-term survival (ongoing).

Conditions

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Myelofibrosis Polycythemia Vera Thrombocytosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Ruxolitinib

All participants received oral ruxolitinib. Patients began treatment with either 10 mg twice a day (bid), 15 mg bid, 25 mg bid, 50 mg bid, 25 mg once a day (qd), 50 mg qd, 100 mg qd, or 200 mg qd, depending on the time period when they entered the study. The doses were titrated based on efficacy and safety to a maximum of 25 mg bid for patients who entered the study after sufficient dosing information had been obtained to define the maximum dose for patients in the study. Patients could continue receiving treatment indefinitely if receiving benefit at a dose that continues to maintain benefit but does not exceed a maximum dose of 25 mg BID.

Group Type EXPERIMENTAL

Ruxolitinib

Intervention Type DRUG

5 and 25 mg tablets with a daily dosing range from 10 to 200 mg qd or bid.

Interventions

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Ruxolitinib

5 and 25 mg tablets with a daily dosing range from 10 to 200 mg qd or bid.

Intervention Type DRUG

Other Intervention Names

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INCB018424 Jakafi(TM)

Eligibility Criteria

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Inclusion Criteria

* Diagnosed with PMF or Post-PV/ET MF
* Patients with myelofibrosis requiring therapy
* Adequate bone marrow reserve

Exclusion Criteria

* Received anti-cancer medications or investigational therapy in the past 14 days
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Incyte Corporation

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Srdan Verstovsek, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

M.D. Anderson Cancer Center, Houston, TX

Ayalew Tefferi, MD

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic, Rochester, MN

Locations

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Rochester, Minnesota, United States

Site Status

Houston, Texas, United States

Site Status

Countries

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United States

References

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Verstovsek S, Kantarjian H, Mesa RA, Pardanani AD, Cortes-Franco J, Thomas DA, Estrov Z, Fridman JS, Bradley EC, Erickson-Viitanen S, Vaddi K, Levy R, Tefferi A. Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med. 2010 Sep 16;363(12):1117-27. doi: 10.1056/NEJMoa1002028.

Reference Type RESULT
PMID: 20843246 (View on PubMed)

Verstovsek S, Kantarjian HM, Estrov Z, Cortes JE, Thomas DA, Kadia T, Pierce S, Jabbour E, Borthakur G, Rumi E, Pungolino E, Morra E, Caramazza D, Cazzola M, Passamonti F. Long-term outcomes of 107 patients with myelofibrosis receiving JAK1/JAK2 inhibitor ruxolitinib: survival advantage in comparison to matched historical controls. Blood. 2012 Aug 9;120(6):1202-9. doi: 10.1182/blood-2012-02-414631. Epub 2012 Jun 20.

Reference Type RESULT
PMID: 22718840 (View on PubMed)

Kvasnicka HM, Thiele J, Bueso-Ramos CE, Sun W, Cortes J, Kantarjian HM, Verstovsek S. Long-term effects of ruxolitinib versus best available therapy on bone marrow fibrosis in patients with myelofibrosis. J Hematol Oncol. 2018 Mar 15;11(1):42. doi: 10.1186/s13045-018-0585-5.

Reference Type DERIVED
PMID: 29544547 (View on PubMed)

Other Identifiers

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INCB 18424-251

Identifier Type: -

Identifier Source: org_study_id

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