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Trial record 1 of 1 for:    02076399 | thrombocytopenia
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A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP) (FIT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Immune Thrombocytopenic Purpura
Interventions Drug: Fostamatinib disodium
Drug: Placebo
Enrollment 76
Recruitment Details 76 patients were enrolled from July 2014 to April 2016
Pre-assignment Details  
Arm/Group Title Fostamatinib Recipient Placebo Recipient
Hide Arm/Group Description Fostamatinib (100 mg PO bid or 150 mg PO bid) Placebo
Period Title: Overall Study
Started 51 25
Completed 12 1
Not Completed 39 24
Arm/Group Title Fostamatinib Recipient Placebo Recipient Total
Hide Arm/Group Description Fostamatinib (100 mg PO bid or 150 mg PO bid) Placebo Total of all reporting groups
Overall Number of Baseline Participants 51 25 76
Hide Baseline Analysis Population Description
ITT Population
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 51 participants 25 participants 76 participants
57.3  (17.7) 53.2  (16.0) 56.0  (17.2)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 51 participants 25 participants 76 participants
Female
30
  58.8%
17
  68.0%
47
  61.8%
Male
21
  41.2%
8
  32.0%
29
  38.2%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 51 participants 25 participants 76 participants
Hispanic or Latino
3
   5.9%
1
   4.0%
4
   5.3%
Not Hispanic or Latino
48
  94.1%
24
  96.0%
72
  94.7%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 51 participants 25 participants 76 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
3
   5.9%
2
   8.0%
5
   6.6%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
2
   3.9%
2
   8.0%
4
   5.3%
White
44
  86.3%
21
  84.0%
65
  85.5%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
2
   3.9%
0
   0.0%
2
   2.6%
1.Primary Outcome
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)
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Fostamatinib Recipient Placebo Recipient
Hide Arm/Group Description:
Fostamatinib (100 mg PO bid or 150 mg PO bid)
Placebo
Overall Number of Participants Analyzed 51 25
Measure Type: Count of Participants
Unit of Measure: Participants
9
  17.6%
0
   0.0%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fostamatinib Recipient, Placebo Recipient
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0261
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value 17.6
Confidence Interval (2-Sided) 95%
7.2 to 28.1
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Number of Participants With Platelet Count ≥ 50,000/µL at Week 12
Hide Description Platelet Count ≥ 50,000/µL at Week 12
Time Frame Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Fostamatinib Recipient Placebo Recipient
Hide Arm/Group Description:
Fostamatinib (100 mg PO bid or 150 mg PO bid)
Placebo
Overall Number of Participants Analyzed 51 25
Measure Type: Count of Participants
Unit of Measure: Participants
11
  21.6%
0
   0.0%
3.Secondary Outcome
Title Number of Participants With Platelet Count ≥ 50,000/µL at Week 24
Hide Description Platelet Count ≥ 50,000/µL at Week 24
Time Frame Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Fostamatinib Recipient Placebo Recipient
Hide Arm/Group Description:
Fostamatinib (100 mg PO bid or 150 mg PO bid)
Placebo
Overall Number of Participants Analyzed 51 25
Measure Type: Count of Participants
Unit of Measure: Participants
8
  15.7%
0
   0.0%
4.Secondary Outcome
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.
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Fostamatinib Recipient Placebo Recipient
Hide Arm/Group Description:
Fostamatinib (100 mg PO bid or 150 mg PO bid)
Placebo
Overall Number of Participants Analyzed 25 12
Measure Type: Count of Participants
Unit of Measure: Participants
4
  16.0%
0
   0.0%
5.Secondary Outcome
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.
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Fostamatinib Recipient Placebo Recipient
Hide Arm/Group Description:
Fostamatinib (100 mg PO bid or 150 mg PO bid)
Placebo
Overall Number of Participants Analyzed 25 12
Measure Type: Count of Participants
Unit of Measure: Participants
4
  16.0%
0
   0.0%
6.Secondary Outcome
Title Mean of the ITP Bleeding Score (IBLS)
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Fostamatinib Recipient Placebo Recipient
Hide Arm/Group Description:
Fostamatinib (100 mg PO bid or 150 mg PO bid)
Placebo
Overall Number of Participants Analyzed 51 25
Mean (Standard Deviation)
Unit of Measure: scores on a scale
0.13  (0.12) 0.14  (0.10)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fostamatinib Recipient, Placebo Recipient
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6642
Comments P-value from a two-sided two-sample t-test, testing for a difference in means between fostamatinib and placebo.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value -0.01
Confidence Interval (2-Sided) 95%
-0.01 to 0.0
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Mean of World Health Organization (WHO) Bleeding Scale
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Fostamatinib Recipient Placebo Recipient
Hide Arm/Group Description:
Fostamatinib (100 mg PO bid or 150 mg PO bid)
Placebo
Overall Number of Participants Analyzed 51 25
Mean (Standard Deviation)
Unit of Measure: scores on a scale
0.61  (0.66) 0.46  (0.56)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fostamatinib Recipient, Placebo Recipient
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3365
Comments P-value from a two-sided two-sample t-test, testing for a difference in means between fostamatinib and placebo.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value 0.15
Confidence Interval (2-Sided) 95%
-0.2 to 0.5
Estimation Comments [Not Specified]
Time Frame 24 Weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Fostamatinib Recipient Placebo Recipient
Hide Arm/Group Description Fostamatinib (100 mg PO bid or 150 mg PO bid) Placebo
All-Cause Mortality
Fostamatinib Recipient Placebo Recipient
Affected / at Risk (%) Affected / at Risk (%)
Total   0/51 (0.00%)      1/25 (4.00%)    
Hide Serious Adverse Events
Fostamatinib Recipient Placebo Recipient
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   8/51 (15.69%)      5/25 (20.00%)    
Blood and lymphatic system disorders     
Anaemia  1  0/51 (0.00%)  0 1/25 (4.00%)  2
Febrile Neutropenia  1  1/51 (1.96%)  1 0/25 (0.00%)  0
Immune Thrombocytopenic Purpura  1  1/51 (1.96%)  1 0/25 (0.00%)  0
Thrombocytopenia  1  1/51 (1.96%)  2 0/25 (0.00%)  0
Cardiac disorders     
Cardiac Failure Congestive  1  0/51 (0.00%)  0 1/25 (4.00%)  1
Eye disorders     
Retinal Tear  1  1/51 (1.96%)  1 0/25 (0.00%)  0
Gastrointestinal disorders     
Diarrhoea  1  1/51 (1.96%)  1 0/25 (0.00%)  0
Gastrointestinal Haemorrhage  1  0/51 (0.00%)  0 1/25 (4.00%)  2
Infections and infestations     
Pneumonia  1  1/51 (1.96%)  1 0/25 (0.00%)  0
Sepsis  1  0/51 (0.00%)  0 1/25 (4.00%)  1
Nervous system disorders     
Syncope  1  1/51 (1.96%)  1 0/25 (0.00%)  0
Reproductive system and breast disorders     
Menorrhagia  1  0/51 (0.00%)  0 1/25 (4.00%)  1
Vaginal Haemorrhage  1  1/51 (1.96%)  1 0/25 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Chronic Obstructive Pulmonary Disease  1  0/51 (0.00%)  0 1/25 (4.00%)  1
Epistaxis  1  1/51 (1.96%)  2 1/25 (4.00%)  1
1
Term from vocabulary, MedDRA (18.1)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Fostamatinib Recipient Placebo Recipient
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   49/51 (96.08%)      19/25 (76.00%)    
Blood and lymphatic system disorders     
Anaemia  1  2/51 (3.92%)  2/25 (8.00%) 
Gastrointestinal disorders     
Diarrhoea  1  21/51 (41.18%)  4/25 (16.00%) 
Nausea  1  15/51 (29.41%)  1/25 (4.00%) 
Constipation  1  3/51 (5.88%)  1/25 (4.00%) 
Abdominal pain  1  3/51 (5.88%)  0/25 (0.00%) 
Flatulence  1  3/51 (5.88%)  0/25 (0.00%) 
Vomiting  1  2/51 (3.92%)  2/25 (8.00%) 
Rectal haemorrhage  1  0/51 (0.00%)  2/25 (8.00%) 
General disorders     
Fatigue  1  6/51 (11.76%)  1/25 (4.00%) 
Pyrexia  1  2/51 (3.92%)  2/25 (8.00%) 
Chest pain  1  4/51 (7.84%)  1/25 (4.00%) 
Infections and infestations     
Upper respiratory tract infection  1  5/51 (9.80%)  1/25 (4.00%) 
Urinary tract infection  1  3/51 (5.88%)  0/25 (0.00%) 
Injury, poisoning and procedural complications     
Contusion  1  3/51 (5.88%)  0/25 (0.00%) 
Investigations     
Alanine aminotransferase increased  1  9/51 (17.65%)  0/25 (0.00%) 
Aspartate aminotransferase increased  1  8/51 (15.69%)  0/25 (0.00%) 
Blood pressure increased  1  3/51 (5.88%)  1/25 (4.00%) 
Musculoskeletal and connective tissue disorders     
Musculoskeletal pain  1  0/51 (0.00%)  2/25 (8.00%) 
Nervous system disorders     
Headache  1  7/51 (13.73%)  6/25 (24.00%) 
Dizziness  1  9/51 (17.65%)  4/25 (16.00%) 
Dysgeusia  1  4/51 (7.84%)  0/25 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Epistaxis  1  9/51 (17.65%)  4/25 (16.00%) 
Dyspnoea  1  3/51 (5.88%)  3/25 (12.00%) 
Oropharyngeal pain  1  1/51 (1.96%)  2/25 (8.00%) 
Vascular disorders     
Hypertension  1  13/51 (25.49%)  1/25 (4.00%) 
Rash  1  4/51 (7.84%)  0/25 (0.00%) 
1
Term from vocabulary, MedDRA (18.1)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Anne-Marie Duliege, MD
Organization: Rigel
Phone: 650-624-1100
EMail: clinicaltrials@rigel.com
Layout table for additonal information
Responsible Party: Rigel Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02076399    
Other Study ID Numbers: C-935788-047
2013-005452-15 ( EudraCT Number )
First Submitted: February 26, 2014
First Posted: March 3, 2014
Results First Submitted: October 15, 2018
Results First Posted: January 11, 2019
Last Update Posted: February 12, 2019