Trial Outcomes & Findings for Low-Dose Decitabine in Treating Patients With Symptomatic Myelofibrosis (NCT NCT00630994)

Last Updated: 2015-12-30

Results Overview

Confirmed response: objective status of CR, PR, or CI on 2 consecutive evaluations \>=4 weeks apart. CR:Complete resolution of disease-related symptoms and signs; peripheral blood count remission; normal leukocyte differential; bone marrow histologic remission. PR: All criteria for CR except the bone marrow histologic remission. CI: one of the following in the absence of both disease progression and CR/PR: minimum (MI) 20-g/L increase (INC) in hemoglobin level; MI 50% reduction in palpable splenomegaly (\>=10cm); MI 100% INC in platelet count(\>=50000x10\^9/L) or ANC (\>=0.5x10\^9/L)

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

Every 4 weeks during treatment (up to 16 weeks)

Results posted on

2015-12-30

Participant Flow

Four (4) patient was recruited from March 2008 to May 2009 at Mayo Clinic. This trial was permanently closed in September 2009 due to slow accrual.

Participant milestones

Participant milestones
Measure	Decitabine 20 mg/m\^2/day intravenous over one hour on days 1-5 out of 28 days of treatment cycle
Overall Study STARTED	4
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Decitabine 20 mg/m\^2/day intravenous over one hour on days 1-5 out of 28 days of treatment cycle
Overall Study Adverse Event	1
Overall Study Disease Progression	1
Overall Study Lack of Efficacy	1
Overall Study Other	1

Baseline Characteristics

Low-Dose Decitabine in Treating Patients With Symptomatic Myelofibrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Decitabine n=4 Participants 20 mg/m\^2/day intravenous over one hour on days 1-5 out of 28 days of treatment cycle
Age, Continuous	64.8 years STANDARD_DEVIATION 6.6 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants
Prior disease specific therapy (including drugs, stem cell transplant, or splenectomy) Yes	2 participants n=5 Participants
Prior disease specific therapy (including drugs, stem cell transplant, or splenectomy) No	2 participants n=5 Participants
Prior bleeding events felt to be related to underlying disease Yes	0 participants n=5 Participants
Prior bleeding events felt to be related to underlying disease No	4 participants n=5 Participants
Prior thrombosis Yes	2 participants n=5 Participants
Prior thrombosis No	2 participants n=5 Participants
Category of Primary myelofibrosis Primary Myelofibrosis	2 participants n=5 Participants
Category of Primary myelofibrosis Post Essential Thrombocythemia (ET) Myelofibrosis	1 participants n=5 Participants
Category of Primary myelofibrosis Post Polycythemia Vera (PV) Myelofibrosis	1 participants n=5 Participants

PRIMARY outcome

Timeframe: Every 4 weeks during treatment (up to 16 weeks)

Population: All participants who met the eligibility criteria that have signed a consent form and went on treatment were evaluable for response. The study was terminated early due to slow enrollment. The primary outcome measure should be assessed with caution.

Outcome measures

Outcome measures
Measure	Decitabine n=4 Participants 20 mg/m\^2/day intravenous over one hour on days 1-5 out of 28 days of treatment cycle
Number of Participants Who Achieve a Confirmed Response (Complete Remission (CR), Partial Remission (PR), or Clinical Improvement (CI)), According to International Working Group (IWG) Consensus Criteria.	1 participants

SECONDARY outcome

Timeframe: up to 3 years

Population: Study terminated prematurely. Analysis not performed.

OS was defined as the time from registration to death of any cause.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 3 years

Population: Study terminated prematurely. Analysis not performed.

Time to disease progression is defined as the time from registration to progression of disease or death due to any cause. Progression was defined as any one or more of the following: 1)progressive splenomegaly; 2) leukemic transformation confirmed by a bone marrow blast count of \>= 20%; 3) an increase in peripheral blood blast percentage of \>=20% that lasts for \>= 8 weeks.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 48 weeks

Population: Study terminated prematurely. Analysis not performed.

Constitutional symptoms including the presence of one or more of the following felt to be attributed to the disease: severe night sweats, fevers, weight loss and bone pain. Symptoms were assessed every cycle during treatment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 48 weeks

Severe adverse events were defined as grade 3 or higher, regardless of attribution to study drugs. Adverse events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 3. Adverse events were assessed every cycle during treatment.

Outcome measures

Outcome measures
Measure	Decitabine n=4 Participants 20 mg/m\^2/day intravenous over one hour on days 1-5 out of 28 days of treatment cycle
Number of Participants With Severe Adverse Events Anemia	3 participants
Number of Participants With Severe Adverse Events Platelet count decreased	1 participants
Number of Participants With Severe Adverse Events Neutrophil count decreased	2 participants
Number of Participants With Severe Adverse Events Leukopenia	1 participants
Number of Participants With Severe Adverse Events Alkaline Phosphatase Increased	1 participants
Number of Participants With Severe Adverse Events Ascites	1 participants
Number of Participants With Severe Adverse Events Clostridial Infection	1 participants
Number of Participants With Severe Adverse Events Hyperglycemia	1 participants

Adverse Events

Decitabine

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Decitabine n=4 participants at risk 20 mg/m\^2/day intravenous over one hour on days 1-5 out of 28 days of treatment cycle
Gastrointestinal disorders Ascites	25.0% 1/4 • Number of events 1
Infections and infestations Clostridial infection	25.0% 1/4 • Number of events 1

Other adverse events

Other adverse events
Measure	Decitabine n=4 participants at risk 20 mg/m\^2/day intravenous over one hour on days 1-5 out of 28 days of treatment cycle
Blood and lymphatic system disorders Anemia	100.0% 4/4 • Number of events 17
Gastrointestinal disorders Diarrhea	25.0% 1/4 • Number of events 1
Gastrointestinal disorders Nausea	75.0% 3/4 • Number of events 4
General disorders Fatigue	100.0% 4/4 • Number of events 16
Investigations Alkaline phosphatase increased	25.0% 1/4 • Number of events 1
Investigations Bilirubin	25.0% 1/4 • Number of events 1
Investigations Leukopenia	50.0% 2/4 • Number of events 5
Investigations Neutrophil count decreased	75.0% 3/4 • Number of events 7
Investigations Platelet count decreased	100.0% 4/4 • Number of events 12
Metabolism and nutrition disorders Hyperglycemia	25.0% 1/4 • Number of events 2
Skin and subcutaneous tissue disorders Pruritus	50.0% 2/4 • Number of events 3

Additional Information

Dr. Ruben A. Mesa

Mayo Clinic

Phone: 507-284-2511

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place