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

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ClinicalTrials.gov Identifier: NCT02076399
Recruitment Status : Completed
First Posted : March 3, 2014
Results First Posted : January 11, 2019
Last Update Posted : February 12, 2019
Sponsor:
Information provided by (Responsible Party):
Rigel Pharmaceuticals

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).

Condition or disease Intervention/treatment Phase
Immune Thrombocytopenic Purpura Drug: Fostamatinib disodium Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 76 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : July 14, 2014
Actual Primary Completion Date : April 21, 2016
Actual Study Completion Date : April 21, 2016


Arm Intervention/treatment
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.
Drug: Fostamatinib disodium
Fostamatinib (100 mg PO bid or 150 mg PO bid)
Other Names:
  • R935788
  • R788
  • Fostamatinib

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




Primary Outcome Measures :
  1. 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) [ Time Frame: From Week 14 to Week 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 :
  1. Number of Participants With Platelet Count ≥ 50,000/µL at Week 12 [ Time Frame: Week 12 ]
    Platelet Count ≥ 50,000/µL at Week 12

  2. Number of Participants With Platelet Count ≥ 50,000/µL at Week 24 [ Time Frame: Week 24 ]
    Platelet Count ≥ 50,000/µL at Week 24

  3. Platelet Count ≥ 30,000/μL and ≥ 20,000/μL Above Baseline in Subjects With Baseline Platelet Count of <15,000/μL at Week 12. [ Time Frame: Baseline to 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.

  4. Platelet Count ≥ 30,000/μL and ≥ 20,000/μL Above Baseline in Subjects With Baseline Platelet Count of <15,000/μL at Week 24. [ Time Frame: Baseline to 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.

  5. Mean of the ITP Bleeding Score (IBLS) [ Time Frame: Assessed over the 24-week study period ]

    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.


  6. Mean of World Health Organization (WHO) Bleeding Scale [ Time Frame: Assessed over the 24-week study period ]

    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.




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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02076399


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Sponsors and Collaborators
Rigel Pharmaceuticals
Investigators
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Study Director: Rigel Pharmaceuticals, Inc. Rigel Pharmaceuticals, Inc.

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Responsible Party: Rigel Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02076399     History of Changes
Other Study ID Numbers: C-935788-047
2013-005452-15 ( EudraCT Number )
First Posted: March 3, 2014    Key Record Dates
Results First Posted: January 11, 2019
Last Update Posted: February 12, 2019
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Rigel Pharmaceuticals:
Persistent Immune Thrombocytopenic Purpura
Chronic Immune Thrombocytopenic Purpura

Additional relevant MeSH terms:
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Purpura
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases