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
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 up to 6 courses in the absence
of disease progression or unacceptable toxicity.
Interventional
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Supportive Care
Best overall response rate as determined by International Working Group criteria
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.
Up to 2 years
No
Ruben Mesa
Principal Investigator
Mayo Clinic in Arizona
United States: Food and Drug Administration
MC1283
NCT01732445
April 2013
Name | Location |
---|---|
Mayo Clinic in Arizona | Scottsdale, Arizona 85259-5404 |