Trial Outcomes & Findings for Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia (NCT NCT00425477)

NCT ID: NCT00425477

Last Updated: 2018-10-05

Results Overview

Response to treatment was assessed after two cycles, according to International Working Group (IWG) criteria.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 26 participants
• Primary outcome timeframe: assessed after 2 cycles, up to 2 years
• Results posted on: 2018-10-05

Participant Flow

Participant milestones

Measure	Bexarotene + GM-CSF BEX and GM-CSF were administered in 4 week cycles. BEX was given orally with food daily for 28 days at the FDA-approved dose for treatment of CTCL of 300 mg/m2 and GM-CSF was given at a daily dose of 125 µg/m2 subcutaneously for 28 days.
Overall Study STARTED	26
Overall Study COMPLETED	13
Overall Study NOT COMPLETED	13

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia

Baseline characteristics by cohort

Measure	Bexarotene + GM-CSF n=26 Participants BEX and GM-CSF were administered in 4 week cycles. BEX was given orally with food daily for 28 days at the FDA-approved dose for treatment of CTCL of 300 mg/m2 and GM-CSF was given at a daily dose of 125 µg/m2 subcutaneously for 28 days.
Age, Continuous	72 years n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	19 Participants n=5 Participants
Region of Enrollment United States	26 participants n=5 Participants

PRIMARY outcome

Timeframe: assessed after 2 cycles, up to 2 years

Response to treatment was assessed after two cycles, according to International Working Group (IWG) criteria.

Outcome measures

Measure	Bexarotene + GM-CSF n=13 Participants BEX and GM-CSF were administered in 4 week cycles. BEX was given orally with food daily for 28 days at the FDA-approved dose for treatment of CTCL of 300 mg/m2 and GM-CSF was given at a daily dose of 125 µg/m2 subcutaneously for 28 days.
Clinical Response (Complete and Partial) PR (partial remission)	0 Participants
Clinical Response (Complete and Partial) HI (hematologic improvement)	4 Participants
Clinical Response (Complete and Partial) SD (stable disease)	4 Participants
Clinical Response (Complete and Partial) PD (progressive disease)	5 Participants

SECONDARY outcome

Timeframe: Baseline and after two cycles

ANC count at baseline and after two cycles were measured and compared. Due to the limited number of clinical responders, the changes in transfusion requirements were not measured.

Outcome measures

Measure	Bexarotene + GM-CSF n=13 Participants BEX and GM-CSF were administered in 4 week cycles. BEX was given orally with food daily for 28 days at the FDA-approved dose for treatment of CTCL of 300 mg/m2 and GM-CSF was given at a daily dose of 125 µg/m2 subcutaneously for 28 days.
Clinical Activity as Measured by Change in Peripheral Blood Counts and Changes in Transfusion Requirements ANC at baseline	524 neutrophils/mm^3 Standard Error 95
Clinical Activity as Measured by Change in Peripheral Blood Counts and Changes in Transfusion Requirements ANC after 2 cycles	931 neutrophils/mm^3 Standard Error 244

SECONDARY outcome

Timeframe: Baseline and 6, 12, 24, and 36 weeks

Population: Due to the limited number of clinical responders, this research assay was not done.

Outcome measures

Outcome data not reported

Adverse Events

Bexarotene + GM-CSF

Serious events: 7 serious events

Other events: 7 other events

Deaths: 3 deaths

Serious adverse events

Measure	Bexarotene + GM-CSF n=26 participants at risk BEX and GM-CSF were administered in 4 week cycles. BEX was given orally with food daily for 28 days at the FDA-approved dose for treatment of CTCL of 300 mg/m2 and GM-CSF was given at a daily dose of 125 µg/m2 subcutaneously for 28 days.
Infections and infestations Neutropenic infection	7.7% 2/26
Respiratory, thoracic and mediastinal disorders Dyspnea	15.4% 4/26
Cardiac disorders Atrial flutter	3.8% 1/26
Nervous system disorders acute subdural hemmorrhage	3.8% 1/26
Blood and lymphatic system disorders sepsis	7.7% 2/26

Other adverse events

Measure	Bexarotene + GM-CSF n=26 participants at risk BEX and GM-CSF were administered in 4 week cycles. BEX was given orally with food daily for 28 days at the FDA-approved dose for treatment of CTCL of 300 mg/m2 and GM-CSF was given at a daily dose of 125 µg/m2 subcutaneously for 28 days.
Hepatobiliary disorders Elevated ALT	11.5% 3/26
Metabolism and nutrition disorders Hypertriglyceridemia	7.7% 2/26
Musculoskeletal and connective tissue disorders Muscle weakness	7.7% 2/26

Additional Information

B. Douglas Smith

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Phone: 410-614-5068

Email: smithdo@jhmi.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place