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Ruxolitinib Phosphate and Danazol in Treating Anemia in Patients With Myelofibrosis

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ClinicalTrials.gov Identifier: NCT01732445
Recruitment Status : Completed
First Posted : November 22, 2012
Results First Posted : October 18, 2017
Last Update Posted : October 18, 2017
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Mayo Clinic

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Supportive Care
Conditions Anemia
Primary Myelofibrosis
Secondary Myelofibrosis
Interventions Drug: ruxolitinib phosphate
Drug: danazol
Other: quality-of-life assessment
Other: questionnaire administration
Enrollment 14
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Supportive Care (Ruxolitinib Phosphate and Danazol)
Hide Arm/Group Description Patients receive ruxolitinib phosphate PO 10 mg BID and danazol PO 200 mg 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.
Period Title: Overall Study
Started 14
Completed 14
Not Completed 0
Arm/Group Title Supportive Care (Ruxolitinib Phosphate and Danazol)
Hide Arm/Group Description Patients receive ruxolitinib phosphate PO 10 mg BID and danazol PO 200 mg 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.
Overall Number of Baseline Participants 14
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 14 participants
70.5
(43 to 78)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants
Female
5
  35.7%
Male
9
  64.3%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 14 participants
14
 100.0%
1.Primary Outcome
Title Best Overall Response Rate as Determined by International Working Group Criteria
Hide Description Best overall response rate as determined by International Working Group criteria: An evaluable patient will be classified as a responder for the primary endpoint if the patient’s best overall response is CR, PR or CI (Clinical Improvement) as determined by International Working Group Criteria over all cycles of study treatment. The percentage of successes will be estimated by the number of successes (defined as complete response, partial response, or clinical improvement) divided by the total number of evaluable patients times 100. Confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.
Time Frame Up to 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Supportive Care (Ruxolitinib Phosphate and Danazol)
Hide Arm/Group Description:
Patients receive ruxolitinib phosphate PO 10 mg BID and danazol PO 200 mg 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.
Overall Number of Participants Analyzed 14
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of patients with CR, PR or CI
28.6
(8.4 to 58.1)
2.Secondary Outcome
Title Survival Time
Hide Description Survival time is defined as the time from registration to death due to any cause. The distribution of survival time will be estimated using the method of Kaplan-Meier. The median and 95% confidence interval are reported below.
Time Frame From registration to death due to any cause, assessed up to 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Supportive Care (Ruxolitinib Phosphate and Danazol)
Hide Arm/Group Description:
Patients receive ruxolitinib phosphate PO 10 mg BID and danazol PO 200 mg 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.
Overall Number of Participants Analyzed 14
Median (95% Confidence Interval)
Unit of Measure: months
19.2
(4.1 to 28.8)
3.Secondary Outcome
Title Toxicity, Assessed Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 (v4)
Hide Description The maximum grade for each type of toxicity will be recorded for each patient, and frequency tables will be reviewed to determine toxicity patterns within patient groups. In addition, we will review all adverse event data that is graded as 3, 4, or 5 and classified as either "unrelated" or "unlikely to be related" to study treatment in the event of an actual relationship developing. The overall toxicity rates (percentages) for grade 3 or higher adverse events considered at least possibly related to treatment are reported below.
Time Frame Up to 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Supportive Care (Ruxolitinib Phosphate and Danazol)
Hide Arm/Group Description:
Patients receive ruxolitinib phosphate PO 10 mg BID and danazol PO 200 mg 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.
Overall Number of Participants Analyzed 14
Measure Type: Number
Unit of Measure: percentage of patients
Platelet count decreased 7.1
Anemia 64.3
White blood cell decreased 7.1
Neutrophil count decreased 14.3
Hypertension 7.1
Premature menopause 7.1
4.Other Pre-specified Outcome
Title Patient-reported Symptoms Assessed Using the MPN-SAF, as Measured by the Percentage of Patients With a Decrease in MPN-SAF TSS Greater Than 50% From Baseline
Hide Description Patient-reported symptoms will be described at each time point using the mean, confidence interval, median, and range. The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) will be analyzed using published scoring algorithms. MPN-SAF includes 27 items scored on a scale of 0 to 10. The MPN-SAF Total Symptom Score (TSS) (range 0-100) was computed according to the published scoring algorithm. Higher scores represent worse symptom burden. The percentage of patients with a decrease in MPN-SAF TSS greater than 50% from baseline and 95% confidence interval are reported below.
Time Frame Baseline to up to 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Supportive Care (Ruxolitinib Phosphate and Danazol)
Hide Arm/Group Description:
Patients receive ruxolitinib phosphate PO 10 mg BID and danazol PO 200 mg 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.
Overall Number of Participants Analyzed 14
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of patients
28.6
(8.4 to 58.1)
Time Frame Up to 2 years
Adverse Event Reporting Description CTCAE term (AE description) and grade: The descriptions and grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 will be utilized for AE reporting. Each CTCAE term in the current version is a unique representation of a specific event used for medical documentation and scientific analysis and is a single MedDRA Lowest Level Term (LLT).
 
Arm/Group Title Supportive Care (Ruxolitinib Phosphate and Danazol)
Hide Arm/Group Description Patients receive ruxolitinib phosphate PO 10 mg BID and danazol PO 200 mg 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.
All-Cause Mortality
Supportive Care (Ruxolitinib Phosphate and Danazol)
Affected / at Risk (%)
Total   2/14 (14.29%)    
Show Serious Adverse Events Hide Serious Adverse Events
Supportive Care (Ruxolitinib Phosphate and Danazol)
Affected / at Risk (%) # Events
Total   7/14 (50.00%)    
Blood and lymphatic system disorders   
Anemia  1  1/14 (7.14%)  1
Cardiac disorders   
Myocardial infarction  1  1/14 (7.14%)  1
General disorders   
General disorders and administration site conditions - Other, specify  1  1/14 (7.14%)  1
Sudden death NOS  1  1/14 (7.14%)  1
Infections and infestations   
Lung infection  1  1/14 (7.14%)  1
Investigations   
Platelet count decreased  1  1/14 (7.14%)  1
Metabolism and nutrition disorders   
Hyperkalemia  1  2/14 (14.29%)  2
Hyponatremia  1  2/14 (14.29%)  2
Nervous system disorders   
Intracranial hemorrhage  1  1/14 (7.14%)  1
Transient ischemic attacks  1  1/14 (7.14%)  1
1
Term from vocabulary, MedDRA 12
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Supportive Care (Ruxolitinib Phosphate and Danazol)
Affected / at Risk (%) # Events
Total   14/14 (100.00%)    
Blood and lymphatic system disorders   
Anemia  1  9/14 (64.29%)  24
Gastrointestinal disorders   
Diarrhea  1  3/14 (21.43%)  6
Nausea  1  3/14 (21.43%)  6
General disorders   
Edema limbs  1  2/14 (14.29%)  2
Infections and infestations   
Upper respiratory infection  1  1/14 (7.14%)  1
Urinary tract infection  1  1/14 (7.14%)  1
Investigations   
Alanine aminotransferase increased  1  2/14 (14.29%)  2
Neutrophil count decreased  1  4/14 (28.57%)  5
Platelet count decreased  1  12/14 (85.71%)  29
White blood cell decreased  1  5/14 (35.71%)  9
Metabolism and nutrition disorders   
Anorexia  1  1/14 (7.14%)  1
Hyperkalemia  1  1/14 (7.14%)  1
Musculoskeletal and connective tissue disorders   
Pain in extremity  1  2/14 (14.29%)  2
Reproductive system and breast disorders   
Irregular menstruation  1  1/14 (7.14%)  1
Premature menopause  1  1/14 (7.14%)  1
Skin and subcutaneous tissue disorders   
Periorbital edema  1  1/14 (7.14%)  1
Photosensitivity  1  1/14 (7.14%)  1
Vascular disorders   
Hypertension  1  1/14 (7.14%)  1
1
Term from vocabulary, MedDRA 12
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Ruben A. Mesa, M.D.
Organization: Mayo Clinic
Phone: 480/301-8335
Responsible Party: Mayo Clinic
ClinicalTrials.gov Identifier: NCT01732445     History of Changes
Other Study ID Numbers: MC1283
NCI-2012-02201 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
MC1283 ( Other Identifier: Mayo Clinic in Arizona )
P30CA015083 ( U.S. NIH Grant/Contract )
First Submitted: November 19, 2012
First Posted: November 22, 2012
Results First Submitted: August 4, 2017
Results First Posted: October 18, 2017
Last Update Posted: October 18, 2017