Clinical trial NCT02076399: A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Immune Thrombocytopenic Purpura
Keywords: Persistent Immune Thrombocytopenic Purpura
Chronic Immune Thrombocytopenic Purpura
Lead sponsor: Rigel Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2014-03-03
Enrollment: 76
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


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

Primary outcomes:
measure: 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)
timeFrame: From Week 14 to Week 24
Secondary outcomes:
measure: Number of Participants With Platelet Count ≥ 50,000/µL at Week 12
timeFrame: Week 12
measure: Number of Participants With Platelet Count ≥ 50,000/µL at Week 24
timeFrame: Week 24
measure: Platelet Count ≥ 30,000/μL and ≥ 20,000/μL Above Baseline in Subjects With Baseline Platelet Count of <15,000/μL at Week 12.
timeFrame: Baseline to Week 12
measure: Platelet Count ≥ 30,000/μL and ≥ 20,000/μL Above Baseline in Subjects With Baseline Platelet Count of <15,000/μL at Week 24.
timeFrame: Baseline to Week 24
measure: Mean of the ITP Bleeding Score (IBLS)
timeFrame: Assessed over the 24-week study period
measure: Mean of World Health Organization (WHO) Bleeding Scale
timeFrame: Assessed over the 24-week study period
Other outcomes:
Locations:
city: Tucson
country: United States
facility: Arizona Oncology Associates
state: Arizona
city: Los Angeles
country: United States
facility: University of Southern California
state: California
city: Lakeland
country: United States
facility: Lakeland Regional Cancer Center
state: Florida
city: Peoria
country: United States
facility: Bleeding & Clotting Disorders Institute
state: Illinois
city: Lafayette
country: United States
facility: Horizon Oncology Research, Inc
state: Indiana
city: Bethesda
country: United States
facility: Center for Cancer and Blood Disorders
state: Maryland
city: New York
country: United States
facility: Weill Cornell Medical College/New York Presbyterian Hospital
state: New York
city: Greenville
country: United States
facility: Pitt County Memorial Hospital
state: North Carolina
city: Salisbury
country: United States
facility: Bill Hefner VA Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Middletown
country: United States
facility: Signal Point Clinical Research Center LLC
state: Ohio
city: Salt Lake City
country: United States
facility: University of Utah Health Sciences Center
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
city: Concord
country: Australia
facility: Concord Repatriation General Hospital
state: New South Wales
city: Kogarah
country: Australia
facility: St George Hospital
state: New South Wales
city: Liverpool
country: Australia
facility: Liverpool Hospital
state: New South Wales
city: Randwick
country: Australia
facility: Prince of Wales Hospital
state: New South Wales
city: Westmead
country: Australia
facility: Westmead Hospital
state: New South Wales
city: Launceston
country: Australia
facility: Launceston General Hospital
state: Tasmania
city: Frankston
country: Australia
facility: Frankston Hospital
state: Victoria
city: Melbourne
country: Australia
facility: Dept of Haematology, The Alfred Hospital and Monash Medical Centre
state: Victoria
city: Nedlands
country: Australia
facility: Perth Blood Institute
state: Western Australia
city: Hamilton
country: Canada
facility: Hamilton Health Sciences Corporation
state: Ontario
city: Ottawa
country: Canada
facility: Ottawa Hospital Research Institute
state: Ontario
city: Toronto
country: Canada
facility: St. Michael's Hospital
state: Ontario
city: Herlev
country: Denmark
facility: Herlev Hospital University of Copenhagen, Department of Hematology L124
state: DK
city: Odense C
country: Denmark
facility: Dept. of Haematology, Odense University Hospital
state: DK
city: Roskilde
country: Denmark
facility: Hematological department, Roskilde Hospital
state: DK
city: Aalborg
country: Denmark
facility: Aarhus University Hospital
city: Budapest
country: Hungary
facility: Semmelweis University 1st
city: Debrecen
country: Hungary
facility: University of Debrecen
city: Pecs
country: Hungary
facility: Pecs University
city: Bologna
country: Italy
facility: Ematologia - Padigilione 8, Policinico S. Orsola Malpighi, Azienda Ospedaliero Universitaria di Bologna
city: Milano
country: Italy
facility: Ospedale San Raffaele S.r.l. Dipartimento di Oncoematologia
city: Napoli
country: Italy
facility: Universitã Federico II di Napoli
city: Udine
country: Italy
facility: OspedaleCivile-ClinicaEmatologica/PUGD
city: Vicenza
country: Italy
facility: ULSS 6 Vicenza-Ospedale San Bortolo di Vicenza
city: Haag
country: Netherlands
facility: HAGA ziekenhuis
state: NL
city: Kent
country: United Kingdom
facility: Kent & Canterbury Hospital
state: Canterbury
city: Colchester
country: United Kingdom
facility: Colchester General Hospital
state: Essex
city: Liverpool
country: United Kingdom
facility: Royal Liverpool University Hospital
state: UK
city: Leeds
country: United Kingdom
facility: St. James's Hospital
city: Leicester
country: United Kingdom
facility: Leicester Royal Infirmary
city: London
country: United Kingdom
facility: Royal London Hospital
city: London
country: United Kingdom
facility: Hammersmith Hospital, Catherine Lewis Centre
city: London
country: United Kingdom
facility: University College Hospital
city: Manchester
country: United Kingdom
facility: Manchester Royal Infirmary
city: Newcastle-upon-Tyne
country: United Kingdom
facility: Royal Victoria Infirmary
city: Norfolk
country: United Kingdom
facility: James Paget University Hospital
city: Oxford
country: United Kingdom
facility: Oxford University Hospital
city: Stoke-on-Trent
country: United Kingdom
facility: University Hospital of North Staffordshire
city: West Bromwich
country: United Kingdom
facility: Sandwell General Hospital
Study officials:
affiliation: Rigel Pharmaceuticals,Inc.
name: Rigel Pharmaceuticals, Inc.
role: STUDY_DIRECTOR
References:
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.