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A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP) (FIT)

Primary Purpose

Immune Thrombocytopenic Purpura

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Fostamatinib disodium
Placebo
Sponsored by
Rigel Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Immune Thrombocytopenic Purpura focused on measuring Persistent Immune Thrombocytopenic Purpura, Chronic Immune Thrombocytopenic Purpura

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Exclusion Criteria:

  • Clinical diagnosis of autoimmune hemolytic anemia
  • Uncontrolled or poorly controlled hypertension
  • History of coagulopathy including prothrombotic conditions

Sites / Locations

  • Arizona Oncology Associates
  • University of Southern California
  • Lakeland Regional Cancer Center
  • Bleeding & Clotting Disorders Institute
  • Horizon Oncology Research, Inc
  • Center for Cancer and Blood Disorders
  • Weill Cornell Medical College/New York Presbyterian Hospital
  • Pitt County Memorial Hospital
  • Bill Hefner VA Medical Center
  • Cleveland Clinic
  • Signal Point Clinical Research Center LLC
  • University of Utah Health Sciences Center
  • University of Virginia
  • Concord Repatriation General Hospital
  • St George Hospital
  • Liverpool Hospital
  • Prince of Wales Hospital
  • Westmead Hospital
  • Launceston General Hospital
  • Frankston Hospital
  • Dept of Haematology, The Alfred Hospital and Monash Medical Centre
  • Perth Blood Institute
  • Hamilton Health Sciences Corporation
  • Ottawa Hospital Research Institute
  • St. Michael's Hospital
  • Herlev Hospital University of Copenhagen, Department of Hematology L124
  • Dept. of Haematology, Odense University Hospital
  • Hematological department, Roskilde Hospital
  • Aarhus University Hospital
  • Semmelweis University 1st
  • University of Debrecen
  • Pecs University
  • Ematologia - Padigilione 8, Policinico S. Orsola Malpighi, Azienda Ospedaliero Universitaria di Bologna
  • Ospedale San Raffaele S.r.l. Dipartimento di Oncoematologia
  • Universitã Federico II di Napoli
  • OspedaleCivile-ClinicaEmatologica/PUGD
  • ULSS 6 Vicenza-Ospedale San Bortolo di Vicenza
  • HAGA ziekenhuis
  • Kent & Canterbury Hospital
  • Colchester General Hospital
  • Royal Liverpool University Hospital
  • St. James's Hospital
  • Leicester Royal Infirmary
  • Royal London Hospital
  • Hammersmith Hospital, Catherine Lewis Centre
  • University College Hospital
  • Manchester Royal Infirmary
  • Royal Victoria Infirmary
  • James Paget University Hospital
  • Oxford University Hospital
  • University Hospital of North Staffordshire
  • Sandwell General Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Other

Arm Label

Fostamatinib Disodium

Placebo

Arm Description

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.

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Number of Participants With Platelet Count ≥ 50,000/µL at Week 12
Platelet Count ≥ 50,000/µL at Week 12
Number of Participants With Platelet Count ≥ 50,000/µL at Week 24
Platelet Count ≥ 50,000/µL at Week 24
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.
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.
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.
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.

Full Information

First Posted
February 26, 2014
Last Updated
January 24, 2019
Sponsor
Rigel Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT02076399
Brief Title
A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
Acronym
FIT
Official Title
A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura
Study Type
Interventional

2. Study Status

Record Verification Date
October 2018
Overall Recruitment Status
Completed
Study Start Date
July 14, 2014 (Actual)
Primary Completion Date
April 21, 2016 (Actual)
Study Completion Date
April 21, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Rigel Pharmaceuticals

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Immune Thrombocytopenic Purpura
Keywords
Persistent Immune Thrombocytopenic Purpura, Chronic Immune Thrombocytopenic Purpura

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
76 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Fostamatinib Disodium
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Other
Arm Description
Placebo tablet PO bid (morning and evening) over the course of 24 weeks
Intervention Type
Drug
Intervention Name(s)
Fostamatinib disodium
Other Intervention Name(s)
R935788, R788, Fostamatinib
Intervention Description
Fostamatinib (100 mg PO bid or 150 mg PO bid)
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo tablet PO bid (morning and evening) over the course of 24 weeks
Primary Outcome Measure Information:
Title
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)
Description
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
Time Frame
From Week 14 to Week 24
Secondary Outcome Measure Information:
Title
Number of Participants With Platelet Count ≥ 50,000/µL at Week 12
Description
Platelet Count ≥ 50,000/µL at Week 12
Time Frame
Week 12
Title
Number of Participants With Platelet Count ≥ 50,000/µL at Week 24
Description
Platelet Count ≥ 50,000/µL at Week 24
Time Frame
Week 24
Title
Platelet Count ≥ 30,000/μL and ≥ 20,000/μL Above Baseline in Subjects With Baseline Platelet Count of <15,000/μL at Week 12.
Description
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.
Time Frame
Baseline to Week 12
Title
Platelet Count ≥ 30,000/μL and ≥ 20,000/μL Above Baseline in Subjects With Baseline Platelet Count of <15,000/μL at Week 24.
Description
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.
Time Frame
Baseline to Week 24
Title
Mean of the ITP Bleeding Score (IBLS)
Description
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.
Time Frame
Assessed over the 24-week study period
Title
Mean of World Health Organization (WHO) Bleeding Scale
Description
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.
Time Frame
Assessed over the 24-week study period

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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 Exclusion Criteria: Clinical diagnosis of autoimmune hemolytic anemia Uncontrolled or poorly controlled hypertension History of coagulopathy including prothrombotic conditions
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Rigel Pharmaceuticals, Inc.
Organizational Affiliation
Rigel Pharmaceuticals, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Arizona Oncology Associates
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85710
Country
United States
Facility Name
University of Southern California
City
Los Angeles
State/Province
California
ZIP/Postal Code
90089
Country
United States
Facility Name
Lakeland Regional Cancer Center
City
Lakeland
State/Province
Florida
ZIP/Postal Code
33805-1965
Country
United States
Facility Name
Bleeding & Clotting Disorders Institute
City
Peoria
State/Province
Illinois
ZIP/Postal Code
61614
Country
United States
Facility Name
Horizon Oncology Research, Inc
City
Lafayette
State/Province
Indiana
ZIP/Postal Code
47905
Country
United States
Facility Name
Center for Cancer and Blood Disorders
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20817
Country
United States
Facility Name
Weill Cornell Medical College/New York Presbyterian Hospital
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Pitt County Memorial Hospital
City
Greenville
State/Province
North Carolina
ZIP/Postal Code
27858
Country
United States
Facility Name
Bill Hefner VA Medical Center
City
Salisbury
State/Province
North Carolina
ZIP/Postal Code
28144
Country
United States
Facility Name
Cleveland Clinic
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Signal Point Clinical Research Center LLC
City
Middletown
State/Province
Ohio
ZIP/Postal Code
45042
Country
United States
Facility Name
University of Utah Health Sciences Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
University of Virginia
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
Concord Repatriation General Hospital
City
Concord
State/Province
New South Wales
ZIP/Postal Code
2139
Country
Australia
Facility Name
St George Hospital
City
Kogarah
State/Province
New South Wales
ZIP/Postal Code
2217
Country
Australia
Facility Name
Liverpool Hospital
City
Liverpool
State/Province
New South Wales
ZIP/Postal Code
2170
Country
Australia
Facility Name
Prince of Wales Hospital
City
Randwick
State/Province
New South Wales
ZIP/Postal Code
2031
Country
Australia
Facility Name
Westmead Hospital
City
Westmead
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Facility Name
Launceston General Hospital
City
Launceston
State/Province
Tasmania
ZIP/Postal Code
7250
Country
Australia
Facility Name
Frankston Hospital
City
Frankston
State/Province
Victoria
ZIP/Postal Code
3199
Country
Australia
Facility Name
Dept of Haematology, The Alfred Hospital and Monash Medical Centre
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3004
Country
Australia
Facility Name
Perth Blood Institute
City
Nedlands
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Facility Name
Hamilton Health Sciences Corporation
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 3Z5
Country
Canada
Facility Name
Ottawa Hospital Research Institute
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1 H8L6
Country
Canada
Facility Name
St. Michael's Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5B1W8
Country
Canada
Facility Name
Herlev Hospital University of Copenhagen, Department of Hematology L124
City
Herlev
State/Province
DK
ZIP/Postal Code
2730
Country
Denmark
Facility Name
Dept. of Haematology, Odense University Hospital
City
Odense C
State/Province
DK
ZIP/Postal Code
DK-5000
Country
Denmark
Facility Name
Hematological department, Roskilde Hospital
City
Roskilde
State/Province
DK
ZIP/Postal Code
4000
Country
Denmark
Facility Name
Aarhus University Hospital
City
Aalborg
ZIP/Postal Code
9000
Country
Denmark
Facility Name
Semmelweis University 1st
City
Budapest
ZIP/Postal Code
H-1083
Country
Hungary
Facility Name
University of Debrecen
City
Debrecen
ZIP/Postal Code
H-1083
Country
Hungary
Facility Name
Pecs University
City
Pecs
ZIP/Postal Code
H-7624
Country
Hungary
Facility Name
Ematologia - Padigilione 8, Policinico S. Orsola Malpighi, Azienda Ospedaliero Universitaria di Bologna
City
Bologna
ZIP/Postal Code
40138
Country
Italy
Facility Name
Ospedale San Raffaele S.r.l. Dipartimento di Oncoematologia
City
Milano
ZIP/Postal Code
20132
Country
Italy
Facility Name
Universitã Federico II di Napoli
City
Napoli
ZIP/Postal Code
80131
Country
Italy
Facility Name
OspedaleCivile-ClinicaEmatologica/PUGD
City
Udine
ZIP/Postal Code
33100
Country
Italy
Facility Name
ULSS 6 Vicenza-Ospedale San Bortolo di Vicenza
City
Vicenza
Country
Italy
Facility Name
HAGA ziekenhuis
City
Haag
State/Province
NL
ZIP/Postal Code
2545 CH
Country
Netherlands
Facility Name
Kent & Canterbury Hospital
City
Kent
State/Province
Canterbury
ZIP/Postal Code
CT1 3NG
Country
United Kingdom
Facility Name
Colchester General Hospital
City
Colchester
State/Province
Essex
ZIP/Postal Code
CO4 5JL
Country
United Kingdom
Facility Name
Royal Liverpool University Hospital
City
Liverpool
State/Province
UK
ZIP/Postal Code
L78XP
Country
United Kingdom
Facility Name
St. James's Hospital
City
Leeds
ZIP/Postal Code
LS9 7TF
Country
United Kingdom
Facility Name
Leicester Royal Infirmary
City
Leicester
ZIP/Postal Code
LE1 5WW
Country
United Kingdom
Facility Name
Royal London Hospital
City
London
ZIP/Postal Code
E1 2ES
Country
United Kingdom
Facility Name
Hammersmith Hospital, Catherine Lewis Centre
City
London
ZIP/Postal Code
W12 0HS
Country
United Kingdom
Facility Name
University College Hospital
City
London
ZIP/Postal Code
WC1E 6HX
Country
United Kingdom
Facility Name
Manchester Royal Infirmary
City
Manchester
ZIP/Postal Code
M139WL
Country
United Kingdom
Facility Name
Royal Victoria Infirmary
City
Newcastle-upon-Tyne
ZIP/Postal Code
NE1 4LP
Country
United Kingdom
Facility Name
James Paget University Hospital
City
Norfolk
ZIP/Postal Code
NR31 6LA
Country
United Kingdom
Facility Name
Oxford University Hospital
City
Oxford
ZIP/Postal Code
OX3 9BQ
Country
United Kingdom
Facility Name
University Hospital of North Staffordshire
City
Stoke-on-Trent
ZIP/Postal Code
ST4 6QG
Country
United Kingdom
Facility Name
Sandwell General Hospital
City
West Bromwich
ZIP/Postal Code
B71 4HJ
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
33995988
Citation
Cooper N, Altomare I, Thomas MR, Nicolson PLR, Watson SP, Markovtsov V, Todd LK, Masuda E, Bussel JB. Assessment of thrombotic risk during long-term treatment of immune thrombocytopenia with fostamatinib. Ther Adv Hematol. 2021 Apr 30;12:20406207211010875. doi: 10.1177/20406207211010875. eCollection 2021.
Results Reference
derived

Learn more about this trial

A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)

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