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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02076399




Registration number
NCT02076399
Ethics application status
Date submitted
26/02/2014
Date registered
3/03/2014
Date last updated
12/02/2019

Titles & IDs
Public title
A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
Scientific title
A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura
Secondary ID [1] 0 0
2013-005452-15
Secondary ID [2] 0 0
C-935788-047
Universal Trial Number (UTN)
Trial acronym
FIT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Immune Thrombocytopenic Purpura 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Fostamatinib disodium
Treatment: Drugs - Placebo

Experimental: Fostamatinib Disodium - Subjects begin with Fostamatinib Disodium tablet 100 mg PO bid and increase to 150 mg big after week 4 based on platelet count and tolerability.

Other: Placebo - Placebo tablet PO bid (morning and evening) over the course of 24 weeks


Treatment: Drugs: Fostamatinib disodium
Fostamatinib (100 mg PO bid or 150 mg PO bid)

Treatment: Drugs: Placebo
Placebo tablet PO bid (morning and evening) over the course of 24 weeks

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Stable Platelet Response (Count of =50,000/µL on at Least 4 of the Last 6 Scheduled Visits Between Weeks 14 and 24) - A stable platelet response by Week 24 defined as a platelet count of at least 50,000/µL on at least 4 of the last 6 scheduled visits between Weeks 14 and 24
Timepoint [1] 0 0
From Week 14 to Week 24
Secondary outcome [1] 0 0
Number of Participants With Platelet Count = 50,000/µL at Week 12 - Platelet Count = 50,000/µL at Week 12
Timepoint [1] 0 0
Week 12
Secondary outcome [2] 0 0
Number of Participants With Platelet Count = 50,000/µL at Week 24 - Platelet Count = 50,000/µL at Week 24
Timepoint [2] 0 0
Week 24
Secondary outcome [3] 0 0
Platelet Count = 30,000/µL and = 20,000/µL Above Baseline in Subjects With Baseline Platelet Count of <15,000/µL at Week 12. - Number of subjects with baseline platelet count <15,000/µL who showed platelet count increase to =30,000/µL and =20,000/µL from baseline count at Week 12.
Timepoint [3] 0 0
Baseline to Week 12
Secondary outcome [4] 0 0
Platelet Count = 30,000/µL and = 20,000/µL Above Baseline in Subjects With Baseline Platelet Count of <15,000/µL at Week 24. - Number of subjects with baseline platelet count <15,000/µL who showed platelet count increase to =30,000/µL and =20,000/µL from baseline count at Week 24.
Timepoint [4] 0 0
Baseline to Week 24
Secondary outcome [5] 0 0
Mean of the ITP Bleeding Score (IBLS) - 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.
Timepoint [5] 0 0
Assessed over the 24-week study period
Secondary outcome [6] 0 0
Mean of World Health Organization (WHO) Bleeding Scale - 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.
Timepoint [6] 0 0
Assessed over the 24-week study period

Eligibility
Key inclusion criteria
- Clinical diagnosis of persistent/chronic ITP for at least 3 months.

- Average platelet count < 30,000/µL (and none > 35,000 unless as a result of rescue
therapy) from at least 3 qualifying counts
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Clinical diagnosis of autoimmune hemolytic anemia

- Uncontrolled or poorly controlled hypertension

- History of coagulopathy including prothrombotic conditions

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,TAS,VIC,WA
Recruitment hospital [1] 0 0
Concord Repatriation General Hospital - Concord
Recruitment hospital [2] 0 0
St George Hospital - Kogarah
Recruitment hospital [3] 0 0
Liverpool Hospital - Liverpool
Recruitment hospital [4] 0 0
Prince of Wales Hospital - Randwick
Recruitment hospital [5] 0 0
Westmead Hospital - Westmead
Recruitment hospital [6] 0 0
Launceston General Hospital - Launceston
Recruitment hospital [7] 0 0
Frankston Hospital - Frankston
Recruitment hospital [8] 0 0
Dept of Haematology, The Alfred Hospital and Monash Medical Centre - Melbourne
Recruitment hospital [9] 0 0
Perth Blood Institute - Nedlands
Recruitment postcode(s) [1] 0 0
2139 - Concord
Recruitment postcode(s) [2] 0 0
2217 - Kogarah
Recruitment postcode(s) [3] 0 0
2170 - Liverpool
Recruitment postcode(s) [4] 0 0
2031 - Randwick
Recruitment postcode(s) [5] 0 0
2145 - Westmead
Recruitment postcode(s) [6] 0 0
7250 - Launceston
Recruitment postcode(s) [7] 0 0
3199 - Frankston
Recruitment postcode(s) [8] 0 0
3004 - Melbourne
Recruitment postcode(s) [9] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
North Carolina
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
Country [10] 0 0
United States of America
State/province [10] 0 0
Utah
Country [11] 0 0
United States of America
State/province [11] 0 0
Virginia
Country [12] 0 0
Canada
State/province [12] 0 0
Ontario
Country [13] 0 0
Denmark
State/province [13] 0 0
DK
Country [14] 0 0
Denmark
State/province [14] 0 0
Aalborg
Country [15] 0 0
Hungary
State/province [15] 0 0
Budapest
Country [16] 0 0
Hungary
State/province [16] 0 0
Debrecen
Country [17] 0 0
Hungary
State/province [17] 0 0
Pecs
Country [18] 0 0
Italy
State/province [18] 0 0
Bologna
Country [19] 0 0
Italy
State/province [19] 0 0
Milano
Country [20] 0 0
Italy
State/province [20] 0 0
Napoli
Country [21] 0 0
Italy
State/province [21] 0 0
Udine
Country [22] 0 0
Italy
State/province [22] 0 0
Vicenza
Country [23] 0 0
Netherlands
State/province [23] 0 0
NL
Country [24] 0 0
United Kingdom
State/province [24] 0 0
Canterbury
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Essex
Country [26] 0 0
United Kingdom
State/province [26] 0 0
UK
Country [27] 0 0
United Kingdom
State/province [27] 0 0
Leeds
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Leicester
Country [29] 0 0
United Kingdom
State/province [29] 0 0
London
Country [30] 0 0
United Kingdom
State/province [30] 0 0
Manchester
Country [31] 0 0
United Kingdom
State/province [31] 0 0
Newcastle-upon-Tyne
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Norfolk
Country [33] 0 0
United Kingdom
State/province [33] 0 0
Oxford
Country [34] 0 0
United Kingdom
State/province [34] 0 0
Stoke-on-Trent
Country [35] 0 0
United Kingdom
State/province [35] 0 0
West Bromwich

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Rigel Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to determine whether fostamatinib is safe and effective in the
treatment of persistent/chronic Immune Thrombocytopenic Purpura (ITP).
Trial website
https://clinicaltrials.gov/show/NCT02076399
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Rigel Pharmaceuticals, Inc.
Address 0 0
Rigel Pharmaceuticals, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications