Trial Outcomes & Findings for Azacitidine in Treating Patients With Myelofibrosis (NCT NCT00381693)

Last Updated: 2011-04-21

Results Overview

Response Definitions: * Completion Remission (CR):complete resolution of disease-related symptoms, ultrasound-documented resolution of hepastosplenomegaly, normalization of the peripheral blood count, white cell differential, and smear, normalization of bone marrow histology including disappearance of fibrosis and osteosclerosis. Residual cytogenetic abnormalities are allowed. * Partial Remission (PR): a major response in any baseline applicable criteria (except constitutional symptoms) without progression in any other category.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

10 participants

Primary outcome timeframe

4 months

Results posted on

2011-04-21

Participant Flow

Ten patients were recruited from August 2006 to December 2007 at Mayo Clinic. This trial was permanently closed in December 2007 due to lack of accrual and it demonstrated too little clinical benefit.

Participant milestones

Participant milestones
Measure	Azacitidine Azacitidine 75 mg/m\^2 subcutaneously
Overall Study STARTED	10
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	8

Reasons for withdrawal

Reasons for withdrawal
Measure	Azacitidine Azacitidine 75 mg/m\^2 subcutaneously
Overall Study Death	1
Overall Study Adverse Event	4
Overall Study Patient Choice	1
Overall Study Progression	2

Baseline Characteristics

Azacitidine in Treating Patients With Myelofibrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Azacitidine n=10 Participants Azacitidine 75 mg/m\^2 subcutaneously
Age Continuous	72 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants
Disease Primary Myelofibrosis	8 participants n=5 Participants
Disease Post-polycythemia Vera Myelofibrosis	1 participants n=5 Participants
Disease Post-essential Thrombocythemia Myelofibrosis	1 participants n=5 Participants
Lilie Score at Diagnosis (Myelofibrosis only) 0 (Low Risk)	2 participants n=5 Participants
Lilie Score at Diagnosis (Myelofibrosis only) 1 (Intermediate Risk)	5 participants n=5 Participants
Lilie Score at Diagnosis (Myelofibrosis only) 2 (High Risk)	3 participants n=5 Participants
Red Cell Transfusion-dependent Yes	7 participants n=5 Participants
Red Cell Transfusion-dependent No	3 participants n=5 Participants
Prior Thrombosis or Hemorrhage Yes	0 participants n=5 Participants
Prior Thrombosis or Hemorrhage No	10 participants n=5 Participants
Prior Therapy for Myelofibrosis Yes	7 participants n=5 Participants
Prior Therapy for Myelofibrosis No	3 participants n=5 Participants

PRIMARY outcome

Timeframe: 4 months

Outcome measures

Outcome measures
Measure	Azacitidine n=10 Participants Azacitidine 75 mg/m\^2 subcutaneously
Patients With Confirmed Response (Complete Remission or Partial Remission on 2 Consecutive Evaluation at Least 4 Weeks Apart) During the First 4 Months of Treatment	0 participants

SECONDARY outcome

Timeframe: From date of registration until death or 3 years after registration if patient is still alive

OS was defined as the time from registration to death due to any cause or time from registration to 3 years after registration if patient is still alive.

Outcome measures

Outcome measures
Measure	Azacitidine n=10 Participants Azacitidine 75 mg/m\^2 subcutaneously
Overall Survival (OS)	16.9 months Interval 10.7 to 21.2

SECONDARY outcome

Timeframe: up to 3 years

Population: Only 2 out of 10 patients had disease progression. One patient progressed at 0.5 months after registration and one patient progressed at 2.8 months after registration. Thus, median of time to progression and upper limit of 95% confidence interval are not attainable.

Time to progression was defined as the time from registration to progression of disease. Those who die without documentation of disease progression will be considered to have had disease progression at the time of their death unless documented evidence clearly indicates no progression has occured.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Every 4 weeks during treatment

Adverse events (AE) that are classified as either possibly, probably, or definitely related to study treatment according to the National Cancer Institute Common Toxicity Criteria for Adverse Events (NCI CTCAE version 3.0). The maximum grade for each type of AE will be recorded for each patient. Grade refers to the severity of the AE. Grade 1: Mild AE, Grade 2: Moderate AE, Grade 3: Severe AE, Grade 4: Life-threatening or disabling AE, Grade 5: Death related AE

Outcome measures

Outcome measures
Measure	Azacitidine n=10 Participants Azacitidine 75 mg/m\^2 subcutaneously
Number of Participants With Treatment Related Adverse Events Grade 1-2 Hematologic	1 Participants
Number of Participants With Treatment Related Adverse Events Grade 3-4 Hematologic	8 Participants
Number of Participants With Treatment Related Adverse Events Grade 1-2 Non-hematologic	23 Participants
Number of Participants With Treatment Related Adverse Events Grade 3-4 Non-hematologic	0 Participants

Adverse Events

Azacitidine

Serious events: 4 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Azacitidine n=10 participants at risk Azacitidine 75 mg/m\^2 subcutaneously
Blood and lymphatic system disorders Anemia	10.0% 1/10 • Number of events 1
Investigations Neutrophil count decreased	10.0% 1/10 • Number of events 2
Investigations Platelet count decreased	20.0% 2/10 • Number of events 3
Vascular disorders Hematoma	10.0% 1/10 • Number of events 1

Other adverse events

Other adverse events
Measure	Azacitidine n=10 participants at risk Azacitidine 75 mg/m\^2 subcutaneously
Blood and lymphatic system disorders Anemia	50.0% 5/10 • Number of events 8
Gastrointestinal disorders Diarrhea	20.0% 2/10 • Number of events 4
Gastrointestinal disorders Nausea	60.0% 6/10 • Number of events 8
Gastrointestinal disorders Vomiting	50.0% 5/10 • Number of events 6
General disorders Edema Limbs	10.0% 1/10 • Number of events 1
General disorders Fatigue	90.0% 9/10 • Number of events 24
Investigations Leukopenia	20.0% 2/10 • Number of events 4
Investigations Neutrophil count decreased	50.0% 5/10 • Number of events 18
Investigations Platelet count decreased	70.0% 7/10 • Number of events 24
Nervous system disorders Peripheral sensory neuropathy	20.0% 2/10 • Number of events 2
Respiratory, thoracic and mediastinal disorders Cough	10.0% 1/10 • Number of events 1
Respiratory, thoracic and mediastinal disorders Dyspnea	10.0% 1/10 • Number of events 1
Respiratory, thoracic and mediastinal disorders Pneumonitis	10.0% 1/10 • Number of events 1
Skin and subcutaneous tissue disorders Pruritus	60.0% 6/10 • Number of events 10

Additional Information

Dr. Ruben A. Mesa

Mayo Clinic

Phone: 507-284-3533

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place