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Ruxolitinib Phosphate and Danazol in Treating Anemia in Patients With Myelofibrosis

NCT ID: NCT01732445

Last Updated: 2017-10-18

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

PHASE2

Total Enrollment

14 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-04-30

Study Completion Date

2017-08-10

Brief Summary

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This phase II pilot trial studies how well ruxolitinib phosphate and danazol work in treating anemia in patients with myelofibrosis. Ruxolitinib phosphate and danazol may cause the body to make more red blood cells. They are used to treat anemia in patients with myelofibrosis.

Detailed Description

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PRIMARY OBJECTIVES:

I. To evaluate the efficacy (best overall response) of ruxolitinib (ruxolitinib phosphate) and danazol in patients with myelofibrosis suffering from anemia.

SECONDARY OBJECTIVES:

I. To evaluate the overall survival of patients with myelofibrosis suffering from anemia initiating ruxolitinib and danazol.

II. To evaluate the adverse event profile of ruxolitinib and danazol in patients with myelofibrosis suffering from anemia.

TERTIARY OBJECTIVES:

I. To evaluate quality of life (QOL) and patient-reported symptoms using the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) and European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30) with ruxolitinib and danazol for patients with myelofibrosis suffering from anemia.

OUTLINE:

Patients receive ruxolitinib phosphate orally (PO) twice daily (BID) and danazol PO thrice daily (TID) on days 1-56. Treatment repeats every 56 days for 6 courses in the absence of disease progression or unacceptable toxicity. At the treating physician's discretion, patients may continue treatment past 6 courses if they are without disease progression.

After completion of study treatment, patients are followed up every 6 months for 2 years.

Conditions

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Anemia Primary Myelofibrosis Secondary Myelofibrosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

SUPPORTIVE_CARE

Blinding Strategy

NONE

Study Groups

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Supportive care (ruxolitinib phosphate and danazol)

Patients receive ruxolitinib phosphate PO BID and danazol PO TID on days 1-56. Treatment repeats every 56 days for 6 courses in the absence of disease progression or unacceptable toxicity. At the treating physician's discretion, patients may continue treatment past 6 courses if they are without disease progression.

Group Type EXPERIMENTAL

ruxolitinib phosphate

Intervention Type DRUG

Given PO

danazol

Intervention Type DRUG

Given PO

quality-of-life assessment

Intervention Type OTHER

Ancillary studies

questionnaire administration

Intervention Type OTHER

Ancillary studies

Interventions

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ruxolitinib phosphate

Given PO

Intervention Type DRUG

danazol

Given PO

Intervention Type DRUG

quality-of-life assessment

Ancillary studies

Intervention Type OTHER

questionnaire administration

Ancillary studies

Intervention Type OTHER

Other Intervention Names

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INCB18424 Jakafi oral JAK inhibitor INCB18424 oral Janus-associated kinase inhibitor INCB18424 Chronogyn DAN Danocrine quality of life assessment

Eligibility Criteria

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Inclusion Criteria

* Histological confirmation of primary myelofibrosis (MF), post polycythemia vera (PV) or post essential thrombocythemia (ET) myelofibrosis (intermediate 1, intermediate II or high risk) requiring medical therapy
* Anemia is required for trial entry (defined as hemoglobin \< 10g/dL or transfusion dependent \[having needed a transfusion anytime in the past 6 months\])
* Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 at study entry
* Absolute neutrophil count (ANC) \>= 1000/uL
* Platelet count \>= 50,000/uL
* Serum creatinine =\< 1.5 x the upper limit of normal (ULN)
* Total bilirubin =\< 1.5 x ULN; if total bilirubin is \> 1.5 x ULN, a direct bilirubin should be performed and must be \< 1.5mg/dL
* Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =\< 3 x ULN; higher values (i.e., =\< 5 x ULN) are allowed if clinically compatible with hepatic extramedullary hematopoiesis
* Life expectancy of \>= 6 months
* Patient able to provide voluntary written informed consent to participate
* Willing to comply with scheduled visits, treatment plans, laboratory assessments, and other study-related procedures
* Negative pregnancy test done =\< 7 days prior to registration, for women of childbearing potential only

Exclusion Criteria

* Any chemotherapy (e.g., hydroxyurea), immunomodulatory drug therapy (e.g., thalidomide, interferon-alpha), immunosuppressive therapy, corticosteroids \> 10 mg/day prednisone or equivalent, or growth factor treatment (e.g., erythropoietin), hormones (e.g., androgens, danazol) =\< 14 days prior to registration; note: patients who are on ruxolitinib may continue on without a 14 day wash out at the treating physician's discretion
* Major surgery =\< 28 days or radiation =\< 6 months prior to registration
* Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens
* Active acute infection requiring antibiotics
* Uncontrolled congestive heart failure (New York Heart Association classification 3 or 4), angina, myocardial infarction, cerebrovascular accident, coronary/peripheral artery bypass, graft surgery, transient ischemic attack, or pulmonary embolism within 3 months prior to registration
* Participation in any study of an investigational agent (drug, biologic, device) =\< 30 days, unless during non-treatment phase
* Any of the following:

* Pregnant women
* Nursing women
* Men or women of childbearing potential who are unwilling to employ adequate contraception
* Known human immunodeficiency virus or acquired immunodeficiency syndrome-related illness
* Clinically active hepatitis B or C
* Active malignancy other than MF, except adequately treated basal cell carcinoma and squamous cell carcinoma of the skin, cervical carcinoma in situ or other malignancies that have been stable and off therapy for 5 years
* Patient currently taking simvastatin, or lovastatin at a dose greater than 10 mg/day
* Men with prostate specific antigen (PSA) \> 4 ng/ml or with uncontrolled benign prostatic hypertrophy
* Patient received prior combination treatment with ruxolitinib and danazol together; note: previous treatment with ruxolitinib and/or danazol as single agent therapy is allowed
* Receiving any medications or substances that are strong or moderate inhibitors of cytochrome P450, family 3, subfamily A, polypeptide 4 (CYP3A4); use of the following strong or moderate inhibitors are prohibited =\< 7 days prior to registration

* Strong inhibitors of CYP3A4:

* Indinavir (Crixivan)
* Nelfinavir (Viracept)
* Atazanavir (Reyataz)
* Clarithromycin (Biaxin, Biaxin XL)
* Itraconazole (Sporanox)
* Ketoconazole (Nizoral)
* Nefazodone (Serzone)
* Saquinavir (Fortovase, Invirase)
* Telithromycin (Ketek)
* Moderate inhibitors of CYP3A4

* Erythromycin (Erythrocin, E.E.S., Ery-Tab, Eryc, EryPed, PCE)
* Fluconazole (Diflucan)
* Grapefruit juice
* Verapamil (Calan, Calan SR, Covera-HS, Isoptin SR, Verelan)
* Verelan PM
* Diltiazem (Cardizem, Cardizem CD, Cardizem LA, Cardizem SR, Cartia XT, Dilacor XR, Diltia XT, Taztia XT, Tiazac)
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

Mayo Clinic

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Ruben Mesa

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Locations

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Mayo Clinic in Arizona

Scottsdale, Arizona, United States

Site Status

Tisch Cancer Center

New York, New York, United States

Site Status

Countries

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United States

Other Identifiers

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NCI-2012-02201

Identifier Type: REGISTRY

Identifier Source: secondary_id

MC1283

Identifier Type: OTHER

Identifier Source: secondary_id

P30CA015083

Identifier Type: NIH

Identifier Source: secondary_id

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MC1283

Identifier Type: -

Identifier Source: org_study_id