NCT01445769

Brief Summary

The purpose of this study was to evaluate the effect of an alternative dosing strategy of ruxolitinib in subjects with primary myelofibrosis (PMF), post-polycythemia vera-myelofibrosis (PPV-MF) and post essential thrombocythemia-myelofibrosis (PET-MF) in order to minimize the development of anemia and thrombocytopenia.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
45

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Sep 2011

Geographic Reach
1 country

21 active sites

Status
completed

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

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2011

Completed
22 days until next milestone

First Submitted

Initial submission to the registry

September 23, 2011

Completed
11 days until next milestone

First Posted

Study publicly available on registry

October 4, 2011

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2013

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2013

Completed
1.5 years until next milestone

Results Posted

Study results publicly available

September 19, 2014

Completed
Last Updated

March 27, 2019

Status Verified

March 1, 2019

Enrollment Period

1.5 years

First QC Date

September 23, 2011

Results QC Date

March 31, 2014

Last Update Submit

March 18, 2019

Conditions

Primary MyelofibrosisPost-Polycythemia Vera MyelofibrosisPost-Essential Thrombocythemia Myelofibrosis

Keywords

MyelofibrosisPrimary MyelofibrosisPMFPost-Polycythemia Vera MyelofibrosisPPV-MFPost-Essential Thrombocythemia MyelofibrosisPET-MFOpen labelRuxolitinib18424Jak inhibitor

Outcome Measures

Primary Outcomes (2)

  • Mean Percentage Change From Baseline in Spleen Volume at Week 24

    Spleen volume was measured using magnetic resonance imaging (MRI) or computed tomography (CT) scan. The MRIs were read in the central imaging laboratory. Spleen volume was obtained by outlining the circumference of the organ and determining the volume using the technique of least squares. MRI was the preferred method for obtaining spleen volume data. CT scans were performed if the participant was not a candidate for MRI. The CT scans were processed by the same central laboratory used for MRIs. The same method (MRI or CT) was used for all visits for a given participant unless a new contraindication to the use of MRI (eg, pacemaker insertion) occurred.

    Baseline to Week 24

  • Median Percent Change From Baseline in Spleen Volume at Week 24

    Spleen volume was measured using magnetic resonance imaging (MRI) or computed tomography (CT) scan. The MRIs were read in the central imaging laboratory. Spleen volume was obtained by outlining the circumference of the organ and determining the volume using the technique of least squares. MRI was the preferred method for obtaining spleen volume data. CT scans were performed if the participant was not a candidate for MRI. The CT scans were processed by the same central laboratory used for MRIs. The same method (MRI or CT) was used for all visits for a given participant unless a new contraindication to the use of MRI (eg, pacemaker insertion) occurred.

    Baseline to Week 24

Secondary Outcomes (12)

  • Mean Percentage Change From Baseline in the Total Symptom Score at Week 24

    Baseline to Week 24

  • Median Percent Change From Baseline in the Total Symptom Score at Week 24

    Baseline to Week 24

  • Percentage of Participants With a ≥ 35% Reduction From Baseline in Spleen Volume at Week 24

    Baseline to Week 24

  • Percentage of Participants With a ≥ 10% Reduction From Baseline in Spleen Volume at Week 24

    Baseline to Week 24

  • Percentage of Participants With a ≥ 50% Improvement From Baseline in Total Symptom Score at Week 24

    Baseline to Week 24

  • +7 more secondary outcomes

Other Outcomes (1)

  • Dose Distribution at Week 24

    Week 24

Study Arms (1)

Ruxolitinib

EXPERIMENTAL

Participants initially received ruxolitinib 10 mg twice a day (bid) for 24 weeks. Dose increases of 5 mg bid were possible at Weeks 12 and 18 up to a maximum dose of 20 mg bid.

Drug: Ruxolitinib

Interventions

RuxolitinibDRUG

Ruxolitinib was provided as 5 mg tablets. Dose increases were only permitted at wks 12 \& 18 for lack of efficacy. Increases were restricted to patients who didn't meet criteria for a dose hold over the prior 6 wks, had a platelet count ≥ 100 x 10\^9/L at wk 12 or ≥ 150 x 10\^9/L at wk 18, and had a self-reported PGIC score of 3 (minimally improved) to 7 (very much worse) OR the subject's palpable spleen length below the costal margin was reduced by less than 40% relative to Baseline. Dose increases were elective and not required. Subjects were permitted a dose increase of 5 mg BID to 15 mg BID at wk 12 and to a maximum of 20 mg BID at wk 18. The protocol required dose decreases for thrombocytopenia (platelets \<100 x 10\^9/L) or protocol-defined anemia (decline in hemoglobin of at least 2 g/dL to a level \< 8 g/dL, development of transfusion dependence, or a 50% increase in transfusion requirements for transfusion dependent subjects).

Also known as: INCB018424, INC424
Ruxolitinib

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (PPV-MF), or Post-Essential Thrombocythemia Myelofibrosis (PET-MF) as confirmed by bone marrow biopsy.
  • Must score at least 2 points on the Dynamic International Prognostic Scoring System (DIPSS) scale for prognostic risk factors.
  • Peripheral blast count \< 5% at both Screening and Baseline hematology assessments.
  • Must discontinue all drugs used to treat underlying myelofibrosis (MF) disease no later than Day -1 (the day prior to starting ruxolitinib).
  • Must have hemoglobin value ≥ 6.5 g/dL and be willing to receive blood transfusions.
  • Platelet count ≥ 100\*10\^9/L.
  • Must have a palpable spleen.

You may not qualify if:

  • Inadequate liver or bone marrow reserves, end stage renal disease on dialysis, clinically significant concurrent infections requiring therapy, or unstable cardiac function.
  • Invasive malignancies over the previous 5 years (except treated early stage carcinomas of the skin, completely resected intraepithelial carcinoma of the cervix, and completely resected papillary thyroid and follicular thyroid cancers).
  • Splenic irradiation within 6 months prior to receiving the first dose of study medication.
  • Life expectancy less than 6 months.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (21)

Unknown Facility

Highland, California, United States

Location

Unknown Facility

La Jolla, California, United States

Location

Unknown Facility

Los Angeles, California, United States

Location

Unknown Facility

Jacksonville, Florida, United States

Location

Unknown Facility

Orange City, Florida, United States

Location

Unknown Facility

Winter Park, Florida, United States

Location

Unknown Facility

Atlanta, Georgia, United States

Location

Unknown Facility

Augusta, Georgia, United States

Location

Unknown Facility

Iowa City, Iowa, United States

Location

Unknown Facility

Baltimore, Maryland, United States

Location

Unknown Facility

Ann Arbor, Michigan, United States

Location

Unknown Facility

Southfield, Michigan, United States

Location

Unknown Facility

Morristown, New Jersey, United States

Location

Unknown Facility

Armonk, New York, United States

Location

Unknown Facility

Hickory, North Carolina, United States

Location

Unknown Facility

Canton, Ohio, United States

Location

Unknown Facility

Hazleton, Pennsylvania, United States

Location

Unknown Facility

Hershey, Pennsylvania, United States

Location

Unknown Facility

Charleston, South Carolina, United States

Location

Unknown Facility

Sioux Falls, South Dakota, United States

Location

Unknown Facility

San Antonio, Texas, United States

Location

Related Publications (1)

  • Talpaz M, Erickson-Viitanen S, Hou K, Hamburg S, Baer MR. Evaluation of an alternative ruxolitinib dosing regimen in patients with myelofibrosis: an open-label phase 2 study. J Hematol Oncol. 2018 Aug 7;11(1):101. doi: 10.1186/s13045-018-0642-0.

    PMID: 30086777DERIVED

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Myeloproliferative DisordersBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Limitations and Caveats

This was a single arm pilot study. The patient population differed from Phase III studies of ruxolitinib in myelofibrosis and therefore comparisons to standard dosing cannot be made from this study.

Results Point of Contact

Title
Study Director
Organization
Incyte Corporation
855 463-3463

Study Officials

  • William V Williams, MD

    Incyte Corporation

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 23, 2011

First Posted

October 4, 2011

Study Start

September 1, 2011

Primary Completion

March 1, 2013

Study Completion

April 1, 2013

Last Updated

March 27, 2019

Results First Posted

September 19, 2014

Record last verified: 2019-03

Locations

US(21)