Trial Outcomes & Findings for Fostamatinib as a Single Agent or in Combination With Ruxolitinib for Treatment of Patients With Myelofibrosis With Severe Thrombocytopenia (NCT NCT04543279)
NCT ID: NCT04543279
Last Updated: 2023-12-15
Results Overview
-Defined as an increase in platelet count ≥ 50K/microL with at least one more confirmatory platelet count separated by at least 2 weeks (in the absence of platelet transfusion) within the first 12 weeks of fostamatinib treatment
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
3 participants
Primary outcome timeframe
Week 12
Results posted on
2023-12-15
Participant Flow
Participant milestones
| Measure |
Part A: Fostamatinib
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
0
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
3
|
0
|
Reasons for withdrawal
| Measure |
Part A: Fostamatinib
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Physician Decision
|
1
|
0
|
Baseline Characteristics
Fostamatinib as a Single Agent or in Combination With Ruxolitinib for Treatment of Patients With Myelofibrosis With Severe Thrombocytopenia
Baseline characteristics by cohort
| Measure |
Part A: Fostamatinib
n=3 Participants
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
Total
n=3 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
68 years
n=5 Participants
|
—
|
68 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
—
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
—
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
—
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
—
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
—
|
3 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Week 12Population: This outcome measure is only for Part A.
-Defined as an increase in platelet count ≥ 50K/microL with at least one more confirmatory platelet count separated by at least 2 weeks (in the absence of platelet transfusion) within the first 12 weeks of fostamatinib treatment
Outcome measures
| Measure |
Part A: Fostamatinib
n=3 Participants
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Number of Participants With a Platelet Response (Part A)
|
0 Participants
|
—
|
PRIMARY outcome
Timeframe: From start of treatment through 30 days after last day of study treatment (estimated to be approximately 40 weeks)Population: No participants from Part A went on to receive treatment in Part B.
-Measured by number of adverse events, serious adverse events, and laboratory abnormalities
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Through completion of fostamatinib treatment (12 weeks)Population: This outcome measure is for Part A only.
Outcome measures
| Measure |
Part A: Fostamatinib
n=3 Participants
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Number of Participants Eligible to Initiate Therapy With Ruxolitinib (Part A)
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: From start of treatment through 30 days after last day of study treatment (estimated to be approximately 16 weeks)Population: This outcome measure is for Part A only.
-Measured by number of adverse events, serious adverse events, and laboratory abnormalities
Outcome measures
| Measure |
Part A: Fostamatinib
n=3 Participants
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 3 neutrophil count decreased
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 3 white blood cell decreased
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 3 fatigue
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 1 chills
|
2 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 2 pain
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 1 fever
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 3 bacteremia
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 3 lung infection (pneumonia)
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 2 bruising
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 2 anorexia
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 3 joint pain secondary to arthritis
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 1 tendon cramps
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 3 neck pain
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 1 dyspnea
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 1 rash maculo-papular
|
1 Participants
|
—
|
|
Toxicity of Fostamatinib Treatment (Part A)
Grade 2 hypertension
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: Through 12 weeksPopulation: This outcome measure is for Part A only.
Outcome measures
| Measure |
Part A: Fostamatinib
n=3 Participants
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Number of Participants Who Permanently Discontinue Fostamatinib Due to Fostamatinib Related Adverse Events (Part A)
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Through 12 weeksPopulation: This outcome measure is for Part A only.
Outcome measures
| Measure |
Part A: Fostamatinib
n=3 Participants
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Number of Participants Who Require Treatment Interruption of Fostamatinib Due to Adverse Events (Part A)
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: Through 12 weeksPopulation: This outcome measure is for Part A only.
Outcome measures
| Measure |
Part A: Fostamatinib
n=3 Participants
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Number of Participants Who Was Dose Escalated and Tolerated Fostamatinib Dose Greater Than 100 mg BID (Part A)
|
2 Participants
|
—
|
SECONDARY outcome
Timeframe: Week 12Population: This outcome measure is for Part A only.
Outcome measures
| Measure |
Part A: Fostamatinib
n=3 Participants
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Number of Participants Who Achieve 35% or Greater Reduction in Spleen Volume as Determined by Ultrasound at Week 12 of Fostamatinib Treatment (Part A)
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Week 12Population: This outcome measure is for Part A only.
Outcome measures
| Measure |
Part A: Fostamatinib
n=3 Participants
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Change in Mean Spleen Volume as Determined by Ultrasound at Week 12 of Fostamatinib Treatment (Part A)
|
329.73 cm^3
Standard Deviation 169.04
|
—
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: This outcome measure is for Part A only.
-The Myeloproliferative Neoplasm - Symptom Assessment Form Total Symptom Score (MPN-SAF-TSS) has 10 questions for participants to rate their symptoms. The answers range from 0-absent to 10-worst imaginable. The total score for the questionnaire is 100. A higher score indicates worse symptoms.
Outcome measures
| Measure |
Part A: Fostamatinib
n=3 Participants
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Number of Participants With 50% or Greater Improvement in Myeloproliferative Neoplasm - Symptom Assessment Form Total Symptom Score (Part A) From Baseline to Week 12
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: Through week 12Population: This outcome measure is for Part A only.
Outcome measures
| Measure |
Part A: Fostamatinib
n=3 Participants
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Number of Participants Who Achieve Platelet Transfusion Independence (Part A)
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Through week 12Population: This outcome measure is for Part A only.
Outcome measures
| Measure |
Part A: Fostamatinib
n=3 Participants
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Number of Participants With Anemia Who Achieve RBC Transfusion Independence (Part A)
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Through week 12Population: This outcome measure is for Part A only.
Outcome measures
| Measure |
Part A: Fostamatinib
n=3 Participants
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Number of Participants With Change in Marrow Fibrosis by WHO Grading (Part A)
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: No participants from Part A went on to receive treatment in Part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At completion of combination treatment (estimated to be 36 weeks)Population: No participants from Part A went on to receive treatment in Part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 12 weeksPopulation: No participants from Part A went on to receive treatment in Part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At completion of combination treatment (estimated to be 36 weeks)Population: No participants from Part A went on to receive treatment in Part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 12 weeksPopulation: No participants from Part A went on to receive treatment in Part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At completion of combination treatment (estimated to be 36 weeks)Population: No participants from Part A went on to receive treatment in Part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 36 weeksPopulation: No participants from Part A went on to receive treatment in Part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At completion of combination treatment (estimated to be 36 weeks)Population: No participants from Part A went on to receive treatment in Part B.
Outcome measures
Outcome data not reported
Adverse Events
Part A: Fostamatinib
Serious events: 2 serious events
Other events: 3 other events
Deaths: 1 deaths
Part B: Fostamatinib + Ruxolitinib
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Part A: Fostamatinib
n=3 participants at risk
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
Infections and infestations
Lung infection (pneumonia)
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
Musculoskeletal and connective tissue disorders
Joint pain secondary to arthritis
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
Other adverse events
| Measure |
Part A: Fostamatinib
n=3 participants at risk
The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
|
Part B: Fostamatinib + Ruxolitinib
After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles.
Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.
|
|---|---|---|
|
General disorders
Chills
|
66.7%
2/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
General disorders
Fatigue
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
General disorders
Fever
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
General disorders
Pain
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
Infections and infestations
Bacteremia
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
Injury, poisoning and procedural complications
Bruising
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
Investigations
Neutrophil count decreased
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
Investigations
White blood cell count decreased
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
Metabolism and nutrition disorders
Anorexia
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
Musculoskeletal and connective tissue disorders
Tendon cramps
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
|
Vascular disorders
Hypertension
|
33.3%
1/3 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
—
0/0 • Adverse events were collected from start of treatment through 30 days after last dose of treatment (median follow-up time 102 days, full range 89-145 days).
No participants went on to receive Part B.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place