Study of the JAK Inhibitor Ruxolitinib Administered Orally to Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera-Myelofibrosis (PPV-MF) or Post-Essential Thrombocythemia-Myelofibrosis (PET-MF)

NCT ID: NCT01317875

Last Updated: 2025-08-22

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

69 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-03-31

Study Completion Date

2019-12-31

Brief Summary

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This is a Phase IB, open-label, dose-finding study of the JAK 1 and 2 inhibitor ruxolitinib in patients with myelofibrosis (MF). The study consists of two periods: the core study period, comprising the dose escalation stage and the safety extension phase up to Week 24, then the extension study period beyond Week 24 and up to 3 years, to further characterize the safety and efficacy of ruxolitinib in this patient population. The dose escalation phase will enroll successive cohorts of patients who receive increasing doses of ruxolitinib until the maximum safe starting dose (MSSD) is determined. In the safety expansion phase, additional patients will be treated with ruxolitinib at the MSSD defined during dose escalation. The primary objective is to establish the MSSD of ruxolitinib in patients with MF and starting platelet counts \< 100 x 10 \^9/L

Detailed Description

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Conditions

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Myelofibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Stratum -1

Participants with baseline Platelet counts of 75-99 x10\^9/L

Group Type EXPERIMENTAL

Ruxolitinib

Intervention Type DRUG

Starting dose of ruxolitinib for cohort 1 in dose escalation phase - 5mg twice a day (BID)

Doses will be increased a total of approximately 5mg for successive dosing cohorts based on baseline platelet count

Stratum -2

Participants with baseline Platelet counts of 50-74 x10\^9/L

Group Type EXPERIMENTAL

Ruxolitinib

Intervention Type DRUG

Starting dose of ruxolitinib for cohort 1 in dose escalation phase - 5mg twice a day (BID)

Doses will be increased a total of approximately 5mg for successive dosing cohorts based on baseline platelet count

Interventions

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Ruxolitinib

Starting dose of ruxolitinib for cohort 1 in dose escalation phase - 5mg twice a day (BID)

Doses will be increased a total of approximately 5mg for successive dosing cohorts based on baseline platelet count

Intervention Type DRUG

Other Intervention Names

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INCB018424

Eligibility Criteria

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Inclusion Criteria

* Require treatment for MF and classified at least as intermediate risk level 1 defined by the International Working Group.
* Platelet count \< 100x10 \^9/L at screening or at Study Day 1.

Exclusion Criteria

* Received platelet transfusion within 14 days prior to Screening evaluations.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis

INDUSTRY

Sponsor Role collaborator

Incyte Corporation

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Mark Jones, MD

Role: STUDY_DIRECTOR

Incyte Corporation

Locations

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Winter Park, Florida, United States

Site Status

Baltimore, Maryland, United States

Site Status

Houston, Texas, United States

Site Status

Vienna, , Austria

Site Status

Nanjing, Jiangsu, China

Site Status

Chengdu, Sichuan, China

Site Status

Hangzhou, Zhejiang, China

Site Status

Beijing, , China

Site Status

Angers, , France

Site Status

Paris, , France

Site Status

Pierre-Bénite, , France

Site Status

Leipzig, , Germany

Site Status

Florence, , Italy

Site Status

Milan, , Italy

Site Status

Terni, , Italy

Site Status

Rotterdam, , Netherlands

Site Status

Belfast, , United Kingdom

Site Status

London, , United Kingdom

Site Status

Countries

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United States Austria China France Germany Italy Netherlands United Kingdom

References

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Guglielmelli P, Kiladjian JJ, Vannucchi AM, Duan M, Meng H, Pan L, He G, Verstovsek S, Boyer F, Barraco F, Niederwieser D, Pungolino E, Liberati AM, Harrison C, Roussou P, Wroclawska M, Karumanchi D, Sinclair K, Te Boekhorst PAW, Gisslinger H. Efficacy and safety of ruxolitinib in patients with myelofibrosis and low platelet count (50 x 109/L to <100 x 109/L) at baseline: the final analysis of EXPAND. Ther Adv Hematol. 2022 Sep 10;13:20406207221118429. doi: 10.1177/20406207221118429. eCollection 2022.

Reference Type DERIVED
PMID: 36105914 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2010-023055-29

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CINC424A2201

Identifier Type: -

Identifier Source: org_study_id

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