Ruxolitinib Phosphate and Danazol in Treating Anemia in Patients With Myelofibrosis
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
Drug: ruxolitinib phosphate
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Supportive Care
|Official Title:||Phase 2 Pilot Trial of Ruxolitinib Combined With Danazol for Patients With Primary Myelofibrosis (MF), Post Essential Thrombocythemia-Myelofibrosis (Post ET) and Post Polycythemia Vera Myelofibrosis (PV MF) Suffering From Anemia|
- Best overall response rate as determined by International Working Group criteria [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]The proportion of successes will be estimated by the number of successes (defined as complete response [CR], partial response [PR], or clinical improvement [CI]) divided by the total number of evaluable patients. Confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.
- Survival time [ Time Frame: From registration to death due to any cause, assessed up to 2 years ] [ Designated as safety issue: No ]The distribution of survival time will be estimated using the method of Kaplan-Meier.
- Adverse event rate(s), assessed using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 [ Time Frame: Up to 2 years ] [ Designated as safety issue: Yes ]The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.
|Study Start Date:||April 2013|
|Estimated Primary Completion Date:||November 2014 (Final data collection date for primary outcome measure)|
Experimental: 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 up to 6 courses in the absence of disease progression or unacceptable toxicity.
Drug: ruxolitinib phosphate
Other Names:Drug: danazol
I. To evaluate the efficacy (best overall response) of ruxolitinib (ruxolitinib phosphate) and danazol in patients with myelofibrosis suffering from anemia.
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.
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.
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 up to 6 courses in the absence of disease progression or unacceptable toxicity.
|United States, Arizona|
|Mayo Clinic in Arizona||Recruiting|
|Scottsdale, Arizona, United States, 85259|
|Contact: Mayo Clinic Clinical Trials Referral Office 507-538-7623|
|Principal Investigator: Ruben A. Mesa, M.D.|
|Principal Investigator:||Ruben Mesa||Mayo Clinic in Arizona|