Trial Outcomes & Findings for Safety and Efficacy Study of Fostamatinib to Treat Immunoglobin A (IgA) Nephropathy (NCT NCT02112838)

Results Overview

Mean change from Baseline (Visit 2) of proteinuria as measured by the spot Protein-Creatinine Ratio (sPCR) at 24 weeks (Visit 9) for the ITT Population

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

76 participants

Primary outcome timeframe

Baseline to 24 weeks

Results posted on

2019-06-27

Participant Flow

Participant milestones

Participant milestones
Measure	Fostamatinib 150 mg Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks	Placebo Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks
Overall Study STARTED	25	26	25
Overall Study COMPLETED	20	24	24
Overall Study NOT COMPLETED	5	2	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety and Efficacy Study of Fostamatinib to Treat Immunoglobin A (IgA) Nephropathy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Fostamatinib 150 mg n=25 Participants Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 Participants Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks	Placebo n=25 Participants Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Total n=76 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Age, Categorical Between 18 and 65 years	22 Participants n=93 Participants	25 Participants n=4 Participants	25 Participants n=27 Participants	72 Participants n=483 Participants
Age, Categorical >=65 years	3 Participants n=93 Participants	1 Participants n=4 Participants	0 Participants n=27 Participants	4 Participants n=483 Participants
Age, Continuous	43.1 years STANDARD_DEVIATION 14.8 • n=93 Participants	42.3 years STANDARD_DEVIATION 14.1 • n=4 Participants	40.6 years STANDARD_DEVIATION 11.6 • n=27 Participants	42.0 years STANDARD_DEVIATION 40.5 • n=483 Participants
Sex: Female, Male Female	13 Participants n=93 Participants	14 Participants n=4 Participants	13 Participants n=27 Participants	40 Participants n=483 Participants
Sex: Female, Male Male	12 Participants n=93 Participants	12 Participants n=4 Participants	12 Participants n=27 Participants	36 Participants n=483 Participants
Race (NIH/OMB) American Indian or Alaska Native	NA Participants n=93 Participants	NA Participants n=4 Participants	NA Participants n=27 Participants	NA Participants n=483 Participants
Race (NIH/OMB) Asian	11 Participants n=93 Participants	6 Participants n=4 Participants	6 Participants n=27 Participants	23 Participants n=483 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	NA Participants n=93 Participants	NA Participants n=4 Participants	NA Participants n=27 Participants	NA Participants n=483 Participants
Race (NIH/OMB) Black or African American	1 Participants n=93 Participants	1 Participants n=4 Participants	0 Participants n=27 Participants	2 Participants n=483 Participants
Race (NIH/OMB) White	13 Participants n=93 Participants	19 Participants n=4 Participants	19 Participants n=27 Participants	51 Participants n=483 Participants
Race (NIH/OMB) More than one race	NA Participants n=93 Participants	NA Participants n=4 Participants	NA Participants n=27 Participants	NA Participants n=483 Participants
Race (NIH/OMB) Unknown or Not Reported	NA Participants n=93 Participants	NA Participants n=4 Participants	NA Participants n=27 Participants	NA Participants n=483 Participants

PRIMARY outcome

Timeframe: Baseline to 24 weeks

Mean change from Baseline (Visit 2) of proteinuria as measured by the spot Protein-Creatinine Ratio (sPCR) at 24 weeks (Visit 9) for the ITT Population

Outcome measures

Outcome measures
Measure	Placebo n=25 Participants Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=25 Participants Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 Participants Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Mean Change of Proteinuria as Measured by Spot Urine Protein/Creatinine Ratio (sPCR) at Week 24	-177.4 mg/g Standard Error 342.4	-157.5 mg/g Standard Error 345.6	-577.2 mg/g Standard Error 335.7

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: Analysis population includes only subjects with both pre-treatment and post-treatment biopsies collected.

Mean change from pre-treatment to post-treatment in mesangial hypercellularity on renal biopsies. Using the Oxford Classification of IgA Nepthropathy (IgAN), biopsy specimens with a minimum of 8 glomeruli, mesangial hypercellularity (M), endocapillary hypercellularity (E), segmental sclerosis (S), and interstitial fibrosis/tubular atrophy (T) lesions are scored for assessing histologic findings in IgAN. M = the mean score based on Oxford Classification system score is based on total count of mesangial cells for all glomeruli (count of \<4=0 score, 4 to 5=1, 6 to 7=2, ≥8=3). A decrease in score equates to improvement from IgAN disease.

Outcome measures

Outcome measures
Measure	Placebo n=22 renal biopsy samples Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=6 renal biopsy samples Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 renal biopsy samples Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Mean Change From Pre-treatment to Post-treatment in Mesangial Hypercellularity (M) on Renal Biopsies.	-0.1 mesangial hypercellularity score Standard Error 0.1	-0.3 mesangial hypercellularity score Standard Error 1.6	-0.2 mesangial hypercellularity score Standard Error 0.1

SECONDARY outcome

Timeframe: Baseline to Week 24

Percentage of subjects with ≥50% reduction in sPCR from Baseline (Visit 2) at Week 24 (Visit 9)

Outcome measures

Outcome measures
Measure	Placebo n=25 Participants Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=25 Participants Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 Participants Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Percentage of Subjects With ≥50% Reduction in sPCR From Baseline (Visit 2) at Week 24 (Visit 9).	4 Participants	5 Participants	8 Participants

SECONDARY outcome

Timeframe: Baseline to Week 24

Percentage of subjects with ≥ 30% reduction in proteinuria from Baseline (Visit 2) at 24 weeks (Visit 9).

Outcome measures

Outcome measures
Measure	Placebo n=25 Participants Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=25 Participants Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 Participants Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Percentage of Subjects With ≥ 30% Reduction in Proteinuria From Baseline (Visit 2) at 24 Weeks (Visit 9).	8 Participants	8 Participants	12 Participants

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: Analysis population includes only subjects with both pre-treatment and post-treatment biopsies collected.

Mean change from pre-treatment to post-treatment in mesangial hypercellularity on renal biopsies. Using the Oxford Classification of IgA Nepthropathy (IgAN), biopsy specimens with a minimum of 8 glomeruli, mesangial hypercellularity (M), endocapillary hypercellularity (E), segmental sclerosis (S), and interstitial fibrosis/tubular atrophy (T) lesions are scored for assessing histologic findings in IgAN. E = Percentage of glomeruli eypercellularity due to increased number of cells within glomerular capillary lumina causing narrowing of the lumina. A decrease in score equates to improvement from IgAN disease.

Outcome measures

Outcome measures
Measure	Placebo n=22 renal biopsy samples Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=6 renal biopsy samples Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 renal biopsy samples Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Mean Change From Pre-treatment to Post-treatment in Percentage of Glomeruli With Endocapillary Hypercellularity (E) on Renal Biopsies.	-5.4 Percentage of glomeruli Standard Error 1.7	-3.1 Percentage of glomeruli Standard Error 3.2	-4.0 Percentage of glomeruli Standard Error 1.5

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: Analysis population includes only subjects with both pre-treatment and post-treatment biopsies collected.

Mean change from pre-treatment to post-treatment in mesangial hypercellularity on renal biopsies. Using the Oxford Classification of IgA Nepthropathy (IgAN), biopsy specimens with a minimum of 8 glomeruli, mesangial hypercellularity (M), endocapillary hypercellularity (E), segmental sclerosis (S), and interstitial fibrosis/tubular atrophy (T) lesions are scored for assessing histologic findings in IgAN. S = Percentage of any amount of the tuft involved in sclerosis, but not involving the whole tuft or the presence of an adhesion in each glomeruli. A decrease in score equates to improvement from IgAN disease.

Outcome measures

Outcome measures
Measure	Placebo n=22 renal biopsy samples Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=6 renal biopsy samples Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 renal biopsy samples Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Mean Change From Pre-treatment to Post-treatment in Percentage of Glomeruli With Segmental Sclerosis/Adhesion (S) on Renal Biopsies.	-0.8 Percentage of glomeruli Standard Error 3.9	-4.4 Percentage of glomeruli Standard Error 7.8	-6.8 Percentage of glomeruli Standard Error 3.6

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: Analysis population includes only subjects with both pre-treatment and post-treatment biopsies collected.

Mean change from pre-treatment to post-treatment in mesangial hypercellularity on renal biopsies. Using the Oxford Classification of IgA Nepthropathy (IgAN), biopsy specimens with a minimum of 8 glomeruli, mesangial hypercellularity (M), endocapillary hypercellularity (E), segmental sclerosis (S), and interstitial fibrosis/tubular atrophy (T) lesions are scored for assessing histologic findings in IgAN. Percentage of any amount of the tuft involved in sclerosis, but not involving the whole tuft or the presence of an adhesion in each glomeruli. A decrease in score equates to improvement from IgAN disease.

Outcome measures

Outcome measures
Measure	Placebo n=22 renal biopsy samples Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=6 renal biopsy samples Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 renal biopsy samples Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Mean Change From Pre-treatment to Post-treatment in Percentage of Glomeruli With Global Glomerulosclerosis Score on Renal Biopsies.	0.5 Percentage of glomeruli Standard Error 5.2	-11.9 Percentage of glomeruli Standard Error 10.1	8.34 Percentage of glomeruli Standard Error 4.8

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: Analysis population includes only subjects with both pre-treatment and post-treatment biopsies collected.

Mean change from pre-treatment to post-treatment in mesangial hypercellularity on renal biopsies. Using the Oxford Classification of IgA Nepthropathy (IgAN), biopsy specimens with a minimum of 8 glomeruli, mesangial hypercellularity (M), endocapillary hypercellularity (E), segmental sclerosis (S), and interstitial fibrosis/tubular atrophy (T) lesions are scored for assessing histologic findings in IgAN. T= Percentage of cortical area involved by the tubular atrophy or interstitial fibrosis, whichever is greater. A decrease in score equates to improvement from IgAN disease.

Outcome measures

Outcome measures
Measure	Placebo n=22 renal biopsy samples Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=6 renal biopsy samples Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 renal biopsy samples Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Mean Change From Pre-treatment to Post-treatment in Percentage of Glomeruli With Tubulointerstitial Scarring (T) on Renal Biopsies.	-1.7 Percentage of glomeruli Standard Error 5.0	-7.4 Percentage of glomeruli Standard Error 9.7	4.4 Percentage of glomeruli Standard Error 4.6

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: Analysis population includes only subjects with both pre-treatment and post-treatment biopsies collected.

Mean change from pre-treatment to post-treatment in cellular/fibrocellular crescent score on renal biopsies. Biopsy specimens with a minimum of 8 glomeruli, mesangial hypercellularity (M), segmental sclerosis (S), and interstitial fibrosis/tubular atrophy (T) lesions are scored using the Oxford Classification of IgA nepthropathy (IgAN) system for assessing histologic findings in IgAN.

Outcome measures

Outcome measures
Measure	Placebo n=22 renal biopsy samples Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=6 renal biopsy samples Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 renal biopsy samples Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Mean Change From Pre-treatment to Post-treatment in Percentage of Glomeruli With Cellular/Fibrocellular Crescent Score on Renal Biopsies.	-0.4 Percentage of glomeruli Standard Error 0.8	2.3 Percentage of glomeruli Standard Error 1.6	-1.5 Percentage of glomeruli Standard Error 0.8

SECONDARY outcome

Timeframe: Baseline to Week 12

Population: Analysis population includes only subjects who completed visits through Week 12.

Mean change from Baseline (Visit 2) of eGFR at 12 weeks (Visit 7).

Outcome measures

Outcome measures
Measure	Placebo n=25 Participants Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=22 Participants Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=25 Participants Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Mean Change From Baseline (Visit 2) of eGFR at 12 Weeks (Visit 7).	1.4 eGFR (mL/min/1.73 m2) Standard Error 2.0	-2.0 eGFR (mL/min/1.73 m2) Standard Error 2.1	2.1 eGFR (mL/min/1.73 m2) Standard Error 1.9

SECONDARY outcome

Timeframe: Baseline to Week 24

Mean change from Baseline (Visit 2) of eGFR at 24 weeks (Visit 9).

Outcome measures

Outcome measures
Measure	Placebo n=24 Participants Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=21 Participants Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=24 Participants Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Mean Change From Baseline (Visit 2) of eGFR at 24 Weeks (Visit 9).	-0.2 eGFR (mL/min/1.73 m2) Standard Error 1.8	-0.9 eGFR (mL/min/1.73 m2) Standard Error 1.9	2.0 eGFR (mL/min/1.73 m2) Standard Error 1.8

SECONDARY outcome

Timeframe: Baseline to Week 12

Mean change from Baseline (Visit 2) of proteinuria at 12 weeks (Visit 7).

Outcome measures

Outcome measures
Measure	Placebo n=25 Participants Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=25 Participants Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 Participants Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Mean Change From Baseline (Visit 2) of Proteinuria at 12 Weeks (Visit 7).	-328.3 mg/g Standard Error 199.9	-293.1 mg/g Standard Error 202.0	-529.2 mg/g Standard Error 196.4

SECONDARY outcome

Timeframe: Baseline to Week 12

Percentage of subjects with sPCR \<50 mg/mmol (500 mg/g) at 12 weeks (Visit 7).

Outcome measures

Outcome measures
Measure	Placebo n=25 Participants Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=25 Participants Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 Participants Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Percentage of Subjects With sPCR <50 mg/mmol (500 mg/g) at 12 Weeks (Visit 7).	12 percentage of subjects	9 percentage of subjects	16 percentage of subjects

SECONDARY outcome

Timeframe: Baseline to Week 12

Shift in haematuria (dipstick test) from Baseline (Visit 2) at 12 weeks (Visit 7).

Outcome measures

Outcome measures
Measure	Placebo n=25 Participants Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=25 Participants Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 Participants Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Shift in Haematuria (Dipstick Test) From Baseline (Visit 2) at 12 Weeks (Visit 7). Same/Unchanged	21 subjects	13 subjects	21 subjects
Shift in Haematuria (Dipstick Test) From Baseline (Visit 2) at 12 Weeks (Visit 7). Decreased	4 subjects	8 subjects	4 subjects
Shift in Haematuria (Dipstick Test) From Baseline (Visit 2) at 12 Weeks (Visit 7). Increased	0 subjects	1 subjects	0 subjects

SECONDARY outcome

Timeframe: Baseline to Week 24

Shift in haematuria (dipstick test) from Baseline (Visit 2) at 24 weeks (Visit 9).

Outcome measures

Outcome measures
Measure	Placebo n=25 Participants Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 150 mg n=25 Participants Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 Participants Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks
Shift in Haematuria (Dipstick Test) From Baseline (Visit 2) at 24 Weeks (Visit 9). Decreased	5 subjects	6 subjects	8 subjects
Shift in Haematuria (Dipstick Test) From Baseline (Visit 2) at 24 Weeks (Visit 9). Increased	0 subjects	1 subjects	1 subjects
Shift in Haematuria (Dipstick Test) From Baseline (Visit 2) at 24 Weeks (Visit 9). Same/Unchanged	19 subjects	14 subjects	15 subjects

Adverse Events

Fostamatinib 150 mg

Serious events: 2 serious events

Other events: 24 other events

Deaths: 1 deaths

Fostamatinib 100 mg

Serious events: 2 serious events

Other events: 22 other events

Deaths: 0 deaths

Placebo

Serious events: 2 serious events

Other events: 21 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Fostamatinib 150 mg n=25 participants at risk Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 150 mg: Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks	Fostamatinib 100 mg n=26 participants at risk Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks Fostamatinib 100 mg: Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks	Placebo n=25 participants at risk Placebo tablet twice daily by mouth, over the course of 24 weeks Placebo: Placebo tablet twice daily by mouth, over the course of 24 weeks
Injury, poisoning and procedural complications Concussion	0.00% 0/25 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	3.8% 1/26 • Number of events 1 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	0.00% 0/25 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.
Injury, poisoning and procedural complications Post Procedural Haematuria	0.00% 0/25 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	0.00% 0/26 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	4.0% 1/25 • Number of events 1 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.
Injury, poisoning and procedural complications Procedural Pain	0.00% 0/25 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	0.00% 0/26 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	4.0% 1/25 • Number of events 1 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.
Gastrointestinal disorders Pancreatitis	4.0% 1/25 • Number of events 1 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	3.8% 1/26 • Number of events 1 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	0.00% 0/25 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.
Gastrointestinal disorders Peptic Ulcer Perforation	4.0% 1/25 • Number of events 1 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	0.00% 0/26 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	0.00% 0/25 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.
Infections and infestations Septic Shock	4.0% 1/25 • Number of events 1 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	0.00% 0/26 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	0.00% 0/25 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.
Investigations Hepatic Enzyme Abnormal	4.0% 1/25 • Number of events 1 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	0.00% 0/26 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	0.00% 0/25 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.
Vascular disorders Haematoma	4.0% 1/25 • Number of events 1 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	0.00% 0/26 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.	0.00% 0/25 • The AE reporting period begins with the first dose of study drug and ends with the final study (follow-up) visit. AEs that occur between the time of consent and first dose of study drug will be reported as Medical History. If an SAE is present at the follow-up visit (Week 26, Visit 10) or final visit for subjects receiving extended treatment it was followed to resolution or stabilization unless the subject is lost to follow-up.

Other adverse events

Additional Information

Sandra Tong

Rigel Pharmaceuticals

Phone: 1-650-624-1207

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place