Study of Ruxolitinib (INCB018424) Sustained Release Formulation in Myelofibrosis Patients

NCT01340651 | Completed Phase 2 Results

• 1 other identifier: 18424-260
• Study Type: interventional
• Target: 41
• Locations: 1 country
• Sites: 3

Brief Summary

The purpose of this study is to determine the safety and tolerability of ruxolitinib (INCB018424) sustained release (SR) formulation in participants with primary myelofibrosis (PMF), post-polycythemia vera MF (PPV-MF), and post-essential thrombocythemia MF (PET-MF).

Conditions

Myelofibrosis

Keywords

myelofibrosis; myeloproliferative neoplasms; PMF; PET-MF; PPV-MF

Outcome Measures

Primary Outcomes (2)

• Percentage of Participants With at Least 1 Adverse Event From Baseline Through Week 16

  Baseline to Week 16

• Overall Response (OR) at Week 16

  The investigator graded OR according to the International Working Group for Myelofibrosis Research and Therapy criteria for treatment response. As bone marrow biopsies were not taken after baseline, the best achievable response was clinical improvement which required 1 of the following in the absence of progressive disease (PD): (1) A ≥ 2 g/dL increase in hemoglobin level or (2) either a palpable ≥ 50% reduction of splenomegaly of a spleen ≥ 10 cm at baseline or a spleen palpable at \> 5 cm at baseline becoming not palpable. PD required 1 of the following: (1) Progressive splenomegaly defined by the appearance of previously absent splenomegaly that was palpable at \> 5 cm below the left costal margin or a ≥ 100% increase in palpable distance for baseline splenomegaly of 5-10 cm or a ≥ 50% increase in palpable distance for baseline splenomegaly of \> 10 cm or (2) an increase in peripheral blood blast percentage to ≥ 20% that lasted for ≥ 8 weeks. Stable disease: None of the above.

  Baseline to Week 16

Secondary Outcomes (8)

• Change From Baseline in Spleen Volume at Week 16

  Baseline to Week 16

• Change From Baseline in Spleen Length at Week 16

  Baseline to Week 16

• Percentage of Participants With ≥ 35% Reduction in Spleen Volume at Week 16 From Baseline

  Baseline to Week 16

• Change From Baseline in the Total Symptom Score at Week 16

  Baseline to Week 16

• Percentage of Participants With a ≥ 50% Reduction From Baseline in the Total Symptom Score at Week 16

  Baseline to Week 16

Study Arms (1)

Ruxolitinib 25 mg SR/10, 15, or 20 mg IR

EXPERIMENTAL

Participants began administration with 25 mg ruxolitinib sustained release (SR) once daily (QD). After 8 weeks, if there was inadequate efficacy, the dose level could be titrated to 50 mg SR QD or 25 mg SR every other day (QOD) alternating with 50 mg SR QOD. At Week 16, participants transitioned to ruxolitinib 10, 15, or 20 mg immediate release (IR) orally twice daily. Participants who continued to demonstrate benefit in the opinion of the investigator could remain on ruxolitinib IR until the last participant completed Week 36 or the commercial availability of ruxolitinib IR, whichever was earlier; the dose received was based on platelet counts at the time of transition.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

Ruxolitinib was supplied as SR and IR formulated tablets.

Also known as: INCB018424

Ruxolitinib 25 mg SR/10, 15, or 20 mg IR

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants 18 years of age or older.
• Participants must be diagnosed with primary myelofibrosis (PMF), post-essential thrombocythemia myelofibrosis (PPV-MF), or post-polycythemia vera myelofibrosis (PET-MF).
• Participants with myelofibrosis requiring therapy must be classified as high risk (3 or more prognostic factors), intermediate risk level 2 (2 prognostic factors), or intermediate risk level 1 (1 prognostic factor)defined by International Working Group for Myelofibrosis Research and Treatment (IWG-MRT).
• Participants must have a palpable spleen measuring 5 cm or greater below the costal margin.

You may not qualify if:

• Participants with a life expectancy of less than 6 months.
• Participants of childbearing potential who are unwilling to take appropriate precautions to avoid pregnancy or fathering a child.
• Participants with inadequate bone marrow reserve.
• Participants with history of platelet counts \< 50,000/μL, platelet transfusion(s), or an absolute neutrophil count \< 500/μL in the month prior to Screening.
• Participants with inadequate liver or renal function at Screening and Baseline visits.

Sponsors & Collaborators

• Incyte Corporation: lead

Study Sites (3)

Unknown Facility

Scottsdale, Arizona, United States

Location

Unknown Facility

Winter Park, Florida, United States

Location

Unknown Facility

Houston, Texas, TX, United States

Location

MeSH Terms

Conditions

Primary Myelofibrosis; Myeloproliferative Disorders

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Results Point of Contact

• Title: Study Director
• Organization: Incyte Corporation

855 463-3463

Study Officials

• Srdan Verstovsek, MD, PhD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

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: April 21, 2011
• First Posted: April 22, 2011
• Study Start: March 1, 2011
• Primary Completion: July 1, 2012
• Study Completion: July 1, 2012
• Last Updated: March 10, 2014
• Results First Posted: September 25, 2013

Record last verified: 2013-12

Locations

US (3)