Trial Outcomes & Findings for A Efficacy and Safety Study of Fostamatinib in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP) (NCT NCT02076412)

NCT ID: NCT02076412

Last Updated: 2019-01-25

Results Overview

Stable platelet response by Week 24 defined as a platelet count of at least 50,000/µL on at least 4 of the 6 visits between Weeks 14-24

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 74 participants
• Primary outcome timeframe: Baseline to Week 24
• Results posted on: 2019-01-25

Participant Flow

74 patients were enrolled from January 2015 to August 2016

Participant milestones

Measure	Fostamatinib Recipient Fostamatinib (100 mg PO bid or 150 mg PO bid)	Placebo Recipient Placebo
Overall Study STARTED	50	24
Overall Study COMPLETED	13	2
Overall Study NOT COMPLETED	37	22

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Efficacy and Safety Study of Fostamatinib in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)

Baseline characteristics by cohort

Measure	Fostamatinib Recipient n=50 Participants Fostamatinib (100 mg PO bid or 150 mg PO bid)	Placebo Recipient n=24 Participants Placebo	Total n=74 Participants Total of all reporting groups
Age, Continuous	49.1 years STANDARD_DEVIATION 15.2 • n=5 Participants	49.5 years STANDARD_DEVIATION 16.5 • n=7 Participants	49.2 years STANDARD_DEVIATION 15.5 • n=5 Participants
Sex: Female, Male Female	31 Participants n=5 Participants	13 Participants n=7 Participants	44 Participants n=5 Participants
Sex: Female, Male Male	19 Participants n=5 Participants	11 Participants n=7 Participants	30 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	50 Participants n=5 Participants	24 Participants n=7 Participants	74 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	50 Participants n=5 Participants	24 Participants n=7 Participants	74 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to Week 24

Stable platelet response by Week 24 defined as a platelet count of at least 50,000/µL on at least 4 of the 6 visits between Weeks 14-24

Outcome measures

Measure	Fostamatinib Recipient n=50 Participants Fostamatinib (100 mg PO bid or 150 mg PO bid)	Placebo Recipient n=24 Participants Placebo
Number of Participants With Stable Platelet Response of at Least 50,000/µL	9 Participants	1 Participants

SECONDARY outcome

Timeframe: Baseline to Week 12

Platelet Count ≥ 50,000/µL at Week 12

Outcome measures

Measure	Fostamatinib Recipient n=50 Participants Fostamatinib (100 mg PO bid or 150 mg PO bid)	Placebo Recipient n=24 Participants Placebo
Number of Participants With Platelet Count ≥ 50,000/µL at Week 12	12 Participants	3 Participants

SECONDARY outcome

Timeframe: Baseline to Week 24

Platelet Count ≥ 50,000/µL at Week 24

Outcome measures

Measure	Fostamatinib Recipient n=50 Participants Fostamatinib (100 mg PO bid or 150 mg PO bid)	Placebo Recipient n=24 Participants Placebo
Number of Participants With Platelet Count ≥ 50,000/µL at Week 24	8 Participants	1 Participants

SECONDARY outcome

Timeframe: Baseline to Week 12

Among subjects with a baseline platelet count < 15,000/μL, achievement of a count ≥ 30,000/μL and at least 20,000/μL above baseline at Week 12.

Outcome measures

Measure	Fostamatinib Recipient n=22 Participants Fostamatinib (100 mg PO bid or 150 mg PO bid)	Placebo Recipient n=9 Participants Placebo
Number of Participants With Platelet Count ≥ 30,000/μL and at Least 20,000/μL Above Baseline at Week 12	6 Participants	1 Participants

SECONDARY outcome

Timeframe: Baseline to Week 24

Among subjects with a baseline platelet count < 15,000/μL, achievement of a count ≥ 30,000/μL and at least 20,000/μL above baseline at Week 24

Outcome measures

Measure	Fostamatinib Recipient n=22 Participants Fostamatinib (100 mg PO bid or 150 mg PO bid)	Placebo Recipient n=9 Participants Placebo
Number of Participants With Platelet Count ≥ 30,000/μL and at Least 20,000/μL Above Baseline at Week 24	3 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline to Week 24

The ITP Bleeding Scale (IBLS) is an immune thrombocytopenic purpura (ITP)-specific bleeding score used to analyze the correlation of clinical and laboratory platelet variables with bleeding. The IBLS comprises of 11 grades from 0 (none) to 2 (marked bleeding) by history over the previous week or by exam; 2 being worse. These 11 grades include: skin by physical exam, oral by physical exam, skin by history, oral by history, epistaxis, gastrointestinal, urinary, gynecological, pulmonary, intracranial hemorrhage, and subconjunctival hemorrhage. After each grade is scored, the mean value for all 11 grades is calculated (lowest score being 0 and highest score being 2) for each subject visit. LOCF method was used to impute any missing data.

The mean of the IBLS scores across visits during the 24-week treatment period was summarized by treatment group using descriptive statistics. A 2-sided, 2-sample t-test was used to test for a difference in means between treatments for this endpoint.

Outcome measures

Measure	Fostamatinib Recipient n=50 Participants Fostamatinib (100 mg PO bid or 150 mg PO bid)	Placebo Recipient n=24 Participants Placebo
Frequency and Severity of Bleeding According to the ITP Bleeding Score (IBLS)	0.04 scores on a scale Standard Deviation 0.08	0.06 scores on a scale Standard Deviation 0.07

SECONDARY outcome

Timeframe: Baseline to Week 24

The World Health Organization (WHO) bleeding scale is a standardized grading scale created to measure the severity of bleeding. The scale is a clinical investigator-assessed five-point scale with a score range starting at the lowest 0=No bleeding, 1 = Petechiae, 2=Mild blood loss, 3=Gross blood loss, to the worse 4=Debilitating blood loss. The WHO bleeding scale is scored by history over the previous-week or by exam. After each grade is scored, the mean value is calculated (lowest score being 0 [no bleeding] to the highest score being 4 [debilitating blood loss]) for each visit. LOCF method was used to impute any missing data.

The mean of the WHO bleeding scale across visits during the 24-week treatment period was summarized by treatment group using descriptive statistics. A 2-sided, 2-sample t-test was used to test for a difference in means between treatments for this endpoint.

Outcome measures

Measure	Fostamatinib Recipient n=50 Participants Fostamatinib (100 mg PO bid or 150 mg PO bid)	Placebo Recipient n=24 Participants Placebo
Frequency and Severity of Bleeding According to the World Health Organization (WHO) Bleeding Scale	0.26 scores on a scale Standard Deviation 0.38	0.38 scores on a scale Standard Deviation 0.47

Adverse Events

Fostamatinib Recipient

Serious events: 5 serious events

Other events: 36 other events

Deaths: 1 deaths

Placebo Recipient

Serious events: 6 serious events

Other events: 18 other events

Deaths: 0 deaths

Serious adverse events

Measure	Fostamatinib Recipient n=51 participants at risk Fostamatinib (100 mg PO bid or 150 mg PO bid)	Placebo Recipient n=23 participants at risk Placebo
Blood and lymphatic system disorders Thrombocytopenia	0.00% 0/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	13.0% 3/23 • Number of events 8 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Infections and infestations Bronchitis	2.0% 1/51 • Number of events 1 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	0.00% 0/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Infections and infestations Infection	0.00% 0/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	4.3% 1/23 • Number of events 1 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Injury, poisoning and procedural complications Contusion	2.0% 1/51 • Number of events 1 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	0.00% 0/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Injury, poisoning and procedural complications Muscle rupture	0.00% 0/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	4.3% 1/23 • Number of events 1 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Investigations Platelet count decreased	2.0% 1/51 • Number of events 1 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	0.00% 0/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Plasma cell myeloma	2.0% 1/51 • Number of events 1 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	0.00% 0/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Nervous system disorders Transient ischaemic attack	2.0% 1/51 • Number of events 1 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	0.00% 0/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Reproductive system and breast disorders Menorrhagia	0.00% 0/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	4.3% 1/23 • Number of events 1 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Respiratory, thoracic and mediastinal disorders Epistaxis	2.0% 1/51 • Number of events 1 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	0.00% 0/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Skin and subcutaneous tissue disorders Petechiae	0.00% 0/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	4.3% 1/23 • Number of events 2 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Vascular disorders Hypertensive crisis	2.0% 1/51 • Number of events 1 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	0.00% 0/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.

Other adverse events

Measure	Fostamatinib Recipient n=51 participants at risk Fostamatinib (100 mg PO bid or 150 mg PO bid)	Placebo Recipient n=23 participants at risk Placebo
Gastrointestinal disorders Diarrhoea	17.6% 9/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	13.0% 3/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Gastrointestinal disorders Nausea	7.8% 4/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	13.0% 3/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Vascular disorders Haematoma	2.0% 1/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	8.7% 2/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Vascular disorders Hypertension	13.7% 7/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	13.0% 3/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Infections and infestations Bronchitis	5.9% 3/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	0.00% 0/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Skin and subcutaneous tissue disorders Petechiae	3.9% 2/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	8.7% 2/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Skin and subcutaneous tissue disorders Rash	5.9% 3/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	4.3% 1/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Nervous system disorders Headache	5.9% 3/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	13.0% 3/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Respiratory, thoracic and mediastinal disorders Epistaxis	11.8% 6/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	4.3% 1/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Blood and lymphatic system disorders Thrombocytopenia	0.00% 0/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	13.0% 3/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.
Injury, poisoning and procedural complications Contusion	5.9% 3/51 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.	0.00% 0/23 • 24 weeks One patient randomized to the placebo group was given the wrong treatment kit, and was treated with fostamatinib for 2 weeks. This patient's efficacy data were analyzed with the placebo arm, but his safety data were analyzed with the fostamatinib arm.

Additional Information

Anne-Marie Duliege, MD

Rigel

Phone: 6506241100

Email: clinicaltrials@rigel.com

Results disclosure agreements

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