Trial Outcomes & Findings for SB1518 for Patients With Myelodysplastic Syndrome (MDS) (NCT NCT01436084)

Last Updated: 2016-07-13

Results Overview

Overall response based on hematologic improvement defined by International Working Group (IWG) response criteria in myelodysplasia. Complete remission (CR): Bone marrow of 5% myeloblasts with normal maturation of all cell lines, noted persistent dysplasia; Partial Remission: CR criteria if abnormal before treatment except Bone marrow blasts decreased by 50% over pretreatment but still \> 5%; Marrow CR: Bone marrow 5% myeloblasts and decrease by 50% over pretreatment. Bone marrow aspirate pre-therapy (Day 0) and on Day 28 of first cycle then every 3 cycles. Responses must last at least 4 weeks.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

8 participants

Primary outcome timeframe

28 days to one year

Results posted on

2016-07-13

Participant Flow

Recruitment Period: December 22, 2011 to January 24, 2012. All recruitment done at The University of Texas (UT) MD Anderson Cancer Center.

Four participants of the eight enrolled failed to meet the screening criteria and were not treated on the study. The study was closed to new patient entry 2 months after activation due to sponsor/collaborator issues; the only patient remaining was taken off study at that time.

Participant milestones

Participant milestones
Measure	SB1518 400 mg orally a day for 28 day cycle.
Overall Study STARTED	4
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	SB1518 400 mg orally a day for 28 day cycle.
Overall Study Disease Progression	2
Overall Study Withdrawal by Subject	1
Overall Study Early Closure of Study	1

Baseline Characteristics

SB1518 for Patients With Myelodysplastic Syndrome (MDS)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	SB1518 n=4 Participants 400 mg orally a day for 28 day cycle.
Age, Continuous	69 years n=93 Participants
Sex: Female, Male Female	1 Participants n=93 Participants
Sex: Female, Male Male	3 Participants n=93 Participants
Region of Enrollment United States	4 participants n=93 Participants

PRIMARY outcome

Timeframe: 28 days to one year

Population: Study was halted prior to completion of treatment and assessment for any participant(s).

Outcome measures

Outcome data not reported

Adverse Events

SB1518

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	SB1518 n=4 participants at risk 400 mg orally a day for 28 day cycle.
Gastrointestinal disorders Gastrointestinal (GI) Other	25.0% 1/4 • Number of events 1 • Adverse event data collected during 28 day drug cycles to thirty days following the last dose of study drug. In the absence of treatment delays due to adverse events, treatment may continue indefinitely. Study period December 2011 to January 2012.
General disorders Abdominal pain	25.0% 1/4 • Number of events 1 • Adverse event data collected during 28 day drug cycles to thirty days following the last dose of study drug. In the absence of treatment delays due to adverse events, treatment may continue indefinitely. Study period December 2011 to January 2012.
General disorders Nausea	50.0% 2/4 • Number of events 2 • Adverse event data collected during 28 day drug cycles to thirty days following the last dose of study drug. In the absence of treatment delays due to adverse events, treatment may continue indefinitely. Study period December 2011 to January 2012.
General disorders Fatigue	50.0% 2/4 • Number of events 2 • Adverse event data collected during 28 day drug cycles to thirty days following the last dose of study drug. In the absence of treatment delays due to adverse events, treatment may continue indefinitely. Study period December 2011 to January 2012.
Skin and subcutaneous tissue disorders Sweating	25.0% 1/4 • Number of events 1 • Adverse event data collected during 28 day drug cycles to thirty days following the last dose of study drug. In the absence of treatment delays due to adverse events, treatment may continue indefinitely. Study period December 2011 to January 2012.
Gastrointestinal disorders Diarrhea	50.0% 2/4 • Number of events 2 • Adverse event data collected during 28 day drug cycles to thirty days following the last dose of study drug. In the absence of treatment delays due to adverse events, treatment may continue indefinitely. Study period December 2011 to January 2012.
General disorders Pain Other	25.0% 1/4 • Number of events 1 • Adverse event data collected during 28 day drug cycles to thirty days following the last dose of study drug. In the absence of treatment delays due to adverse events, treatment may continue indefinitely. Study period December 2011 to January 2012.
General disorders Vomiting	25.0% 1/4 • Number of events 1 • Adverse event data collected during 28 day drug cycles to thirty days following the last dose of study drug. In the absence of treatment delays due to adverse events, treatment may continue indefinitely. Study period December 2011 to January 2012.
Gastrointestinal disorders Heartburn	25.0% 1/4 • Number of events 1 • Adverse event data collected during 28 day drug cycles to thirty days following the last dose of study drug. In the absence of treatment delays due to adverse events, treatment may continue indefinitely. Study period December 2011 to January 2012.

Additional Information

Guillermo Garcia-Manero, MD / Professor, Leukemia

University of Texas (UT) MD Anderson Cancer Center

Phone: 1-877-MDA-6789

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place