Trial Outcomes & Findings for Study of SB939 in Subjects With Myelofibrosis (NCT NCT01200498)

Results Overview

Objective response defined as Complete, Partial response, and Clinical Improvement based on International Working Group (IWG) Criteria: Complete remission (CR): Absence transfusion \& growth factor support AND Complete resolution disease-related symptoms/signs; Peripheral blood count remission; Normal leukocyte differential; Bone marrow histological remission. Partial remission (PR): All CR except bone marrow histological remission. Clinical improvement (CI): No CR/PR, disease progression with one: ≥2 g/dL increase hemoglobin level or transfusion independent; Either ≥50% reduction in palpable splenomegaly of spleen ≥10 cm baseline or spleen palpable at \>5 cm baseline becomes not palpable; ≥100% increase in platelet count \& absolute platelet count ≥50,000 x 10\^9/L; or ≥100% increase in absolute neutrophil count (ANC) \& ANC ≥0.5 x 10\^9/L. Progressive disease: Progressive splenomegaly or Leukemic transformation confirmed by bone marrow blast of ≥20%; or Increase peripheral blood blast

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

23 participants

Primary outcome timeframe

Baseline to 3 Cycles (84 days)

Results posted on

2014-01-30

Participant Flow

Recruitment Period: 11/9/2010 through 11/12/2012. All participants recruited at The University of Texas MD Anderson Cancer Center.

Of the twenty-three participants enrolled, one participant withdrew consent and was excluded before starting the study.

Participant milestones

Participant milestones
Measure	SB939 SB939 starting dose 60 mg by mouth every other day, three times weekly for 3 weeks.
Overall Study STARTED	22
Overall Study COMPLETED	22
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of SB939 in Subjects With Myelofibrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	SB939 n=22 Participants SB939 starting dose 60 mg by mouth every other day, 3 times weekly for 3 weeks.
Age, Continuous	67 years n=5 Participants
Sex: Female, Male Female	17 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Region of Enrollment United States	22 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to 3 Cycles (84 days)

Objective response defined as Complete, Partial response, and Clinical Improvement based on International Working Group (IWG) Criteria: Complete remission (CR): Absence transfusion \& growth factor support AND Complete resolution disease-related symptoms/signs; Peripheral blood count remission; Normal leukocyte differential; Bone marrow histological remission. Partial remission (PR): All CR except bone marrow histological remission. Clinical improvement (CI): No CR/PR, disease progression with one: ≥2 g/dL increase hemoglobin level or transfusion independent; Either ≥50% reduction in palpable splenomegaly of spleen ≥10 cm baseline or spleen palpable at \>5 cm baseline becomes not palpable; ≥100% increase in platelet count \& absolute platelet count ≥50,000 x 10\^9/L; or ≥100% increase in absolute neutrophil count (ANC) \& ANC ≥0.5 x 10\^9/L. Progressive disease: Progressive splenomegaly or Leukemic transformation confirmed by bone marrow blast of ≥20%; or Increase peripheral blood blast

Outcome measures

Outcome measures
Measure	SB939 n=22 Participants SB939 starting dose 60 mg by mouth every other day, three times weekly for 3 weeks.
Participants With an Objective Response Complete Response (CR)	0 Participants
Participants With an Objective Response Partial Response (PR)	0 Participants
Participants With an Objective Response Clinical Improvement (CI)	2 Participants

Adverse Events

SB939

Serious events: 1 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	SB939 n=22 participants at risk SB939 starting dose 60 mg by mouth every other day, 3 times weekly for 3 weeks.
Nervous system disorders Cognitive Disturbance	4.5% 1/22 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug. The full study reporting period was two years.
Nervous system disorders Muscle Weakness	4.5% 1/22 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug. The full study reporting period was two years.

Other adverse events

Other adverse events
Measure	SB939 n=22 participants at risk SB939 starting dose 60 mg by mouth every other day, 3 times weekly for 3 weeks.
General disorders Fatigue	90.9% 20/22 • Number of events 20 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug. The full study reporting period was two years.
General disorders Peripheral Edema	18.2% 4/22 • Number of events 4 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug. The full study reporting period was two years.
Gastrointestinal disorders Diarrhea	13.6% 3/22 • Number of events 3 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug. The full study reporting period was two years.

Additional Information

Alfonso Quintas-Cardama, MD/Assistant Professor

The University of Texas MD Anderson Cancer Center

Phone: 713-745-4009

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place