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

Immune Thrombocytopenic Purpura Clinical Trial

— 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02076399
• Other study ID #: C-935788-047
• Secondary ID: 2013-005452-15
• Status: Completed
• Phase: Phase 3
• Start date: July 14, 2014
• Est. completion date: April 21, 2016

Study information

• Verified date: October 2018
• Source: Rigel Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 76
• Est. completion date: April 21, 2016
• Est. primary completion date: April 21, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Immune Thrombocytopenic Purpura
• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombocytopenic, Idiopathic

Intervention

Drug:
• Fostamatinib disodium
Fostamatinib (100 mg PO bid or 150 mg PO bid)
• Placebo
Placebo tablet PO bid (morning and evening) over the course of 24 weeks

Locations

Country	Name	City	State
Australia	Concord Repatriation General Hospital	Concord	New South Wales
Australia	Frankston Hospital	Frankston	Victoria
Australia	St George Hospital	Kogarah	New South Wales
Australia	Launceston General Hospital	Launceston	Tasmania
Australia	Liverpool Hospital	Liverpool	New South Wales
Australia	Dept of Haematology, The Alfred Hospital and Monash Medical Centre	Melbourne	Victoria
Australia	Perth Blood Institute	Nedlands	Western Australia
Australia	Prince of Wales Hospital	Randwick	New South Wales
Australia	Westmead Hospital	Westmead	New South Wales
Canada	Hamilton Health Sciences Corporation	Hamilton	Ontario
Canada	Ottawa Hospital Research Institute	Ottawa	Ontario
Canada	St. Michael's Hospital	Toronto	Ontario
Denmark	Aarhus University Hospital	Aalborg
Denmark	Herlev Hospital University of Copenhagen, Department of Hematology L124	Herlev	DK
Denmark	Dept. of Haematology, Odense University Hospital	Odense C	DK
Denmark	Hematological department, Roskilde Hospital	Roskilde	DK
Hungary	Semmelweis University 1st	Budapest
Hungary	University of Debrecen	Debrecen
Hungary	Pecs University	Pecs
Italy	Ematologia - Padigilione 8, Policinico S. Orsola Malpighi, Azienda Ospedaliero Universitaria di Bologna	Bologna
Italy	Ospedale San Raffaele S.r.l. Dipartimento di Oncoematologia	Milano
Italy	Universitã Federico II di Napoli	Napoli
Italy	OspedaleCivile-ClinicaEmatologica/PUGD	Udine
Italy	ULSS 6 Vicenza-Ospedale San Bortolo di Vicenza	Vicenza
Netherlands	HAGA ziekenhuis	Haag	NL
United Kingdom	Colchester General Hospital	Colchester	Essex
United Kingdom	Kent & Canterbury Hospital	Kent	Canterbury
United Kingdom	St. James's Hospital	Leeds
United Kingdom	Leicester Royal Infirmary	Leicester
United Kingdom	Royal Liverpool University Hospital	Liverpool	UK
United Kingdom	Hammersmith Hospital, Catherine Lewis Centre	London
United Kingdom	Royal London Hospital	London
United Kingdom	University College Hospital	London
United Kingdom	Manchester Royal Infirmary	Manchester
United Kingdom	Royal Victoria Infirmary	Newcastle-upon-Tyne
United Kingdom	James Paget University Hospital	Norfolk
United Kingdom	Oxford University Hospital	Oxford
United Kingdom	University Hospital of North Staffordshire	Stoke-on-Trent
United Kingdom	Sandwell General Hospital	West Bromwich
United States	Center for Cancer and Blood Disorders	Bethesda	Maryland
United States	University of Virginia	Charlottesville	Virginia
United States	Cleveland Clinic	Cleveland	Ohio
United States	Pitt County Memorial Hospital	Greenville	North Carolina
United States	Horizon Oncology Research, Inc	Lafayette	Indiana
United States	Lakeland Regional Cancer Center	Lakeland	Florida
United States	University of Southern California	Los Angeles	California
United States	Signal Point Clinical Research Center LLC	Middletown	Ohio
United States	Weill Cornell Medical College/New York Presbyterian Hospital	New York	New York
United States	Bleeding & Clotting Disorders Institute	Peoria	Illinois
United States	Bill Hefner VA Medical Center	Salisbury	North Carolina
United States	University of Utah Health Sciences Center	Salt Lake City	Utah
United States	Arizona Oncology Associates	Tucson	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
Rigel Pharmaceuticals

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Denmark,  Hungary,  Italy,  Netherlands,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	From Week 14 to Week 24
Secondary	Number of Participants With Platelet Count = 50,000/µL at Week 12	Platelet Count = 50,000/µL at Week 12	Week 12
Secondary	Number of Participants With Platelet Count = 50,000/µL at Week 24	Platelet Count = 50,000/µL at Week 24	Week 24
Secondary	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.	Baseline to Week 12
Secondary	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.	Baseline to Week 24
Secondary	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.	Assessed over the 24-week study period
Secondary	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.	Assessed over the 24-week study period