Clinical trial NCT01340651: Study of Ruxolitinib (INCB018424) Sustained Release Formulation in Myelofibrosis Patients
Official title: An Open-label Assessment of Once-daily Dosing of a Sustained Release (SR) Formulation of INCB018424 in Patients With Primary Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis, and Post-polycythemia Vera Myelofibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Myelofibrosis
Keywords: myelofibrosis
myeloproliferative neoplasms
PMF
PET-MF
PPV-MF
Lead sponsor: Incyte Corporation
Sponsor class: INDUSTRY
First posted: 2011-04-22
Enrollment: 41
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).

Detailed description: The study will enroll approximately 40 participants with PMF, PPV-MF or PET-MF. Participants will take ruxolitinib SR once daily for 16 consecutive weeks and then transition to a comparable twice daily dose regimen of ruxolitinib using the immediate release (IR) tablets which have been under investigation in controlled Phase 1, 2, and 3 clinical trials.

Participants receiving benefit from treatment with ruxolitinib may continue further participation with IR tablets up to the time when the last participant completed Week 36 or the commercial availability of ruxolitinib IR, whichever was earlier. Follow-up will occur at least 30 days following the last dose of ruxolitinib.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Percentage of Participants With at Least 1 Adverse Event From Baseline Through Week 16
timeFrame: Baseline to Week 16
measure: Overall Response (OR) at Week 16
timeFrame: Baseline to Week 16
Secondary outcomes:
measure: Change From Baseline in Spleen Volume at Week 16
timeFrame: Baseline to Week 16
measure: Change From Baseline in Spleen Length at Week 16
timeFrame: Baseline to Week 16
measure: Percentage of Participants With ≥ 35% Reduction in Spleen Volume at Week 16 From Baseline
timeFrame: Baseline to Week 16
measure: Change From Baseline in the Total Symptom Score at Week 16
timeFrame: Baseline to Week 16
measure: Percentage of Participants With a ≥ 50% Reduction From Baseline in the Total Symptom Score at Week 16
timeFrame: Baseline to Week 16
measure: Maximum Observed Plasma Concentration (Cmax) of Ruxolitinib 25 mg SR on Day 1
timeFrame: Day 1
measure: Time to Reach the Maximum Plasma Concentration (Tmax) of Ruxolitinib 25 mg SR on Day 1
timeFrame: Day 1
measure: Area Under the Plasma Concentration-time Curve (AUC) of Ruxolitinib 25 mg SR on Day 1
timeFrame: Day 1
Other outcomes:
Locations:
city: Scottsdale
country: United States
state: Arizona
city: Winter Park
country: United States
state: Florida
city: Houston
country: United States
state: Texas
Study officials:
affiliation: M.D. Anderson Cancer Center
name: Srdan Verstovsek, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: