Clinical trial NCT01633372: An Open Label Study of Itacitinib Administered Orally in Patients With Myelofibrosis
Official title: An Open-Label, Multiple Simon 2-Stage Study of Itacitinib Administered Orally to Subjects With Primary Myelofibrosis (PMF), Post Polycythemia Vera Myelofibrosis (PPV-MF) or Post Essential Thrombocythemia Myelofibrosis (PET-MF)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: MPN (Myeloproliferative Neoplasms)
Keywords: Primary Myelofibrosis
PMF
Post Polycythemia Vera Fibrosis
PPV-MF
Post Essential Thrombocythemia Myelofibrosis
PET-MF
Lead sponsor: Incyte Corporation
Sponsor class: INDUSTRY
First posted: 2012-07-04
Enrollment: 87
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a study of itacitinib (INCB039110) in patients with myelofibrosis. This study will evaluate safety and efficacy parameters of itacitinib (INCB039110).


Eligibility criteria: Inclusion Criteria:

* Must be diagnosed with PMF, PPV-MF or PET-MF as confirmed by bone marrow biopsy.
* Must score at least 1 point on the Dynamic International Prognostic Scoring System (DIPSS) for prognostic risk factors and have peripheral blast count \<10% at both Screening and Baseline hematology assessments.
* Subjects must discontinue all drugs used to treat underlying MF disease no later than Day -14.
* Subjects must have hemoglobin value \>/= 8.0g/dL and be willing to receive blood transfusions, have a platelet count \>/=50x10\^9/L and absolute neutrophil count (ANC) \>/= 1x10\^9/L.
* Subjects must have palpable spleen or history of splenectomy
* Active symptoms at the screening visit

Exclusion Criteria:

* Women who are pregnant or breastfeeding, and men and women who cannot comply with requirements to avoid fathering a child or becoming pregnant, respectively.
* Subjects with impaired liver function, end stage renal disease on dialysis or clinically significant concurrent infections requiring therapy.
* Subjects with unstable cardiac function or invasive malignancies over the previous 2 years except treated basal or squamous carcinomas of the skin, completely resected intraepithelial carcinoma of the cervix and completely resected papillary thyroid and follicular thyroid cancers.

Primary outcomes:
measure: Proportion of subjects with >/= 50% reduction in total symptom score in each dose group, as measured by the modified The Myelofibrosis Symptom Assessment Form (MFSAF) v3.0 diary
timeFrame: Baseline and Week 12
Secondary outcomes:
measure: Proportion of subjects with >/= 35% reduction in spleen volume, and mean percent change in spleen volume
timeFrame: Baseline, Week 12 and Week 24
measure: Proportion of transfusion dependent subjects who exhibit changes in transfusion frequency over any 12 week period on study and proportion of transfusion independent subjects who exhibit changes in hemoglobin level
timeFrame: Baseline to Week 12; Week 13 to Week 24 through the end of study or study termination visit.
measure: Safety and tolerability of itacitinib as measured by adverse events.
timeFrame: Every 4-6 weeks through the end of study or early termination visit (approximately 33 weeks exclusive of the extension phase).
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Birmingham Hematology Oncology Associates, LLC
state: Alabama
city: Scottsdale
country: United States
facility: Mayo Clinic, Arizona
state: Arizona
city: Los Angeles
country: United States
facility: UCLA Hematology & Oncology
state: California
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Baltimore
country: United States
facility: St Agnes Hospital
state: Maryland
city: Ann Arbor
country: United States
facility: University of Michigan Cancer Center
state: Michigan
city: New York
country: United States
facility: Mount Sinai School of Medicine
state: New York
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania Health System
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Western Pennsylvania Hospital
state: Pennsylvania
city: Columbia
country: United States
facility: South Carolina Oncology & Associates
state: South Carolina
city: Memphis
country: United States
facility: Boston Baskin Cancer Foundation, Inc.
state: Tennessee
city: Nashville
country: United States
facility: Tennessee Oncology
state: Tennessee
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: Darlinghurst
country: Australia
facility: St Vincent's Hospital
state: New South Wales
city: Kogarah
country: Australia
facility: St. George Hospital
state: New South Wales
city: Box Hill
country: Australia
facility: Box Hill Hospital
state: Victoria
city: Frankston
country: Australia
facility: Frankston Hospital
state: Victoria
city: Edmonton
country: Canada
facility: Cross Cancer Institute
state: Alberta
city: Vancouver
country: Canada
facility: St. Paul's Hospital
state: British Columbia
city: Toronto
country: Canada
facility: Princess Margaret Hospital
state: Ontario
city: Montreal
country: Canada
facility: St. Mary's Hospital
state: Quebec
Study officials:
affiliation: Incyte Corporation
name: Albert Assad, MD
role: STUDY_DIRECTOR
References:
citation: Mascarenhas JO, Talpaz M, Gupta V, Foltz LM, Savona MR, Paquette R, Turner AR, Coughlin P, Winton E, Burn TC, O'Neill P, Clark J, Hunter D, Assad A, Hoffman R, Verstovsek S. Primary analysis of a phase II open-label trial of INCB039110, a selective JAK1 inhibitor, in patients with myelofibrosis. Haematologica. 2017 Feb;102(2):327-335. doi: 10.3324/haematol.2016.151126. Epub 2016 Oct 27.