Open Label Study of R788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)

A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura

The primary objective of this study was to assess the long term safety of fostamatinib in subjects with persistent/chronic ITP

Study Overview

• Status: Completed
• Conditions: Immune Thrombocytopenic Purpura
• Intervention / Treatment: Drug: Fostamatinib Disodium

• Study Type: Interventional
• Enrollment (Actual): 123
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Concord, New South Wales, Australia, 2139
      • Concord Repatriation General Hospital
    • Liverpool, New South Wales, Australia, 2170
      • Liverpool Hospital
    • Randwick, New South Wales, Australia, 2031
      • Prince of Wales Hospital
    • Westmead, New South Wales, Australia, 2145
      • Westmead Hospital
  • Tasmania
    • Launceston, Tasmania, Australia, 7250
      • Launceston General Hospital
  • Victoria
    • Melbourne, Victoria, Australia, 3004
      • The Alfred
  • Western Australia
    • Nedlands, Western Australia, Australia, 6009
      • Perth Blood Institute
• Austria
  • Vienna, Austria, 1140
    • Hanusch-Krankenhaus Wiener Gebietskrankenkasse
• Bulgaria
  • Pleven, Bulgaria, 5800
    • UMHAT Dr. Georgi Stranski, EAD, Pleven, Clinic of Hematology
  • Sofia, Bulgaria, 1431
    • UMHAT Aleksandrovska, EAD
  • Vratsa, Bulgaria, 3000
    • MHAT Hristo Botev, AD, Vratsa, First Internal Department
  • BG
    • Sofia, BG, Bulgaria, 1756
      • Specialized Hospital for Active Treatment of Hematology Diseases, EAD, Sofia, Department of Chemotherapy, Hemotherapy and Blood Inherited Diseases to Clinic of Clinical Hematology;
• Canada
  • Ontario
    • Hamilton, Ontario, Canada, L8N 3Z5
      • Hamilton Health Sciences Corporation
    • Toronto, Ontario, Canada, M5B1W8
      • St. Michael's Hospital
• Czechia
  • Brno, Czechia, 625 00
    • Fakultni Nemocnice Brno
  • Ostrava-Poruba, Czechia, 708 52
    • Fakultni nemocnice Ostrava
• Denmark
  • DK
    • Herlev, DK, Denmark, 2730
      • Herlev Hospital
• Hungary
  • Pecs, Hungary, H-7624
    • Pecsi Tudomanyegyetem Klinikai Kozpont, I. sz. Belgyogyaszati Klinika
• Italy
  • Udine, Italy, 33100
    • Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia" - Clinica Ematologica
  • BO
    • Bologna, BO, Italy, 40138
      • Istituto di Ematologia "Lorenzo e Ariosto Seràgnoli"
• Netherlands
  • NL
    • Den Haag, NL, Netherlands, 2545 CH
      • Haga Ziekenhuis
• Norway
  • Bergen, Norway, 5021
    • Haukeland universitetssykehus, Helse Bergen HF
  • Grålum, Norway, 1714
    • Sykehuset Østfold Kalnes
• Poland
  • Gdansk, Poland, 80-952
    • Lkinika Hematologii I Transplantologii Uniwersyteckie Centrum Kliniczne
  • Kraków, Poland, 31-501
    • SPZOZ Szpital Uniwersytecki w Krakowie Pracownia Separacji Krwinek i Bank Komórek Krwiotwórczych Klinika Hematologii
  • Lodz, Poland, 93-510
    • Wojewódzki Szpital Specjalistyczny im. M. Kopernika w Łodzi
  • Lublin, Poland, 20-601
    • Specjalistyczny Gabinet Lekarski
  • Opole, Poland, 45-061
    • Szpital Wojewodzki w Opolu
  • Warszawa, Poland, 02-776
    • Instytut Hematologii i Transfuzjologii
  • Dolnoslaski
    • Wroclaw, Dolnoslaski, Poland, 50-367
      • Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wrocrlaw
  • PO
    • Slupsk, PO, Poland, 76-200
      • Wojewodzki Szpital Specjalistyczny im. J. Korczaka
• Romania
  • Bucuresti, Romania, 020125
    • Spitalul Clinic Colentina, Hematologie
• Spain
  • Barcelona, Spain, 08035
    • Hospital Universitari Vall d'Hebron
  • Madrid, Spain, 28046
    • Hospital Universitario La Paz
  • Valencia, Spain, 46026
    • Hospital Universitari i Politecnic La Fe de Valencia
• United Kingdom
  • Canterbury, United Kingdom, CT1 3NG
    • Kent & Canterbury Hospital
  • Great Yarmouth, United Kingdom, NR31 6LA
    • James Paget University Hospital
  • Leeds, United Kingdom, LS9 7TF
    • St. James's Hospital
  • Leicester, United Kingdom, LE1 5WW
    • Leicester Royal Infirmary
  • Liverpool, United Kingdom, L78XP
    • Royal Liverpool University Hospital
  • London, United Kingdom, W12 0HS
    • Imperial College Healthcare NHS Trust
  • London, United Kingdom, WC1E 6AG
    • University College Hospital
  • Oxford, United Kingdom, OX3 7LE
    • Cancer and Haematology Centre
  • Stoke-on-Trent, United Kingdom, ST4 6QG
    • University Hospital of North Midlands NHS Trust, Royal Stoke University Hospital
  • Essex
    • Colchester, Essex, United Kingdom, CO4 5JL
      • Colchester General Hospital
  • UK
    • Newcastle-upon-Tyne, UK, United Kingdom, NE1 4LP
      • Royal Victoria Infirmary
• United States
  • Arizona
    • Tucson, Arizona, United States, 85710
      • Arizona Oncology Associates
  • Illinois
    • Peoria, Illinois, United States, 61615
      • Bleeding & Clotting Disorders Institute
  • Indiana
    • Lafayette, Indiana, United States, 47905
      • Horizon Oncology Research, Inc
  • Maryland
    • Bethesda, Maryland, United States, 20817
      • Center for Cancer and Blood Disorders
  • New York
    • New York, New York, United States, 10065
      • Weill Cornell Medicine
    • New York, New York, United States, 10065
      • Weill Cornell Medical College/New York Presbyterian Hospital
  • North Carolina
    • Greenville, North Carolina, United States, 27834
      • East Carolina University, Brody School of Medicine
    • Salisbury, North Carolina, United States, 28144
      • W.G. "Bill" Hefner VA Medical Center
  • Ohio
    • Middletown, Ohio, United States, 45042
      • Signal Point Clinical Research Center LLC

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Completed week 24 evaluation of Study C935788-047 or Study C935788-048 or discontinued early due to lack of response.
• Able and willing to give written informed consent

Exclusion Criteria:

• Discontinued participation in Study C935788-047 or Study C935788-048 for any reason other than lack of response
• Poorly controlled hypertension during Study C935788-047 or Study C935788-048
• Significant infection, an acute infection such as influenza, or known inflammatory process

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Fostamatinib Disodium; Fostamatinib Disodium tablet 100 mg or 150 mg by mouth twice a day	Drug: Fostamatinib Disodium; Fostamatinib Disodium tablet 100 mg or 150 mg by mouth twice a day; Other Names: R935788; Fostamatinib; R788

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Fostamatinib in 047/048 or 049):Version 1	Percentage of subjects who achieved platelet count of at least 50,000/µL within 12 Weeks of beginning treatment up to 12 months was analyzed among all subjects who received active treatment in one of the prior studies (C788-047 or C788-048), in the current extension study (C788-049), or in both prior and current studies. Treated Population was defined as all enrolled and treated subjects.	Up to 12 months
Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Placebo in 047/048 and Fostamatinib 049): Version 2	A within-subject, between-study comparison of platelet counts for subjects who were previously treated with placebo in one of the prior studies (C788-047 or C788-048) was prospectively defined in the protocol (version 2). Achievement of platelet response by 12 weeks and maintenance of platelet response for 12 weeks was analyzed among subjects who had been randomized to placebo in one of the prior studies (C788-047 or C788-048). Treated Population was defined as all enrolled and treated subjects.	Up to 12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Duration of Platelet Response Based on Platelet Count and Rescue Medication	The duration of platelet response was defined as the time from when the subject first achieved a platelet count of at least 50,000/µL, until the first of 2 visits with platelet counts < 50,000/µL that were at least 4 weeks apart without an intervening visit with a platelet count less than equal to (<=) 50,000/µL unrelated to rescue therapy. Duration of platelet response was analyzed using the Kaplan-Meier method. Treated Population was defined as all enrolled and treated subjects. Here, a number of subjects analyzed included all subjects evaluable for this endpoint.	Up to 12 months
Percentage of Subjects in Whom a Reduction in the Dose of Concomitant ITP Therapy Can be Achieved While Maintaining an Adequate Platelet Count	The percentage of subjects in whom a reduction in the dose of concomitant ITP therapy could be achieved while maintaining an adequate platelet count, the reduction event was clarified to apply only to reductions in the dose of concomitant ITP therapy occurring within a period of platelet response and the reduction event was not be prompted by an adverse event.	Up to 12 months

Collaborators and Investigators

• Sponsor: Rigel Pharmaceuticals
• Investigators: Study Director: Rigel Pharmaceuticals, Inc., Rigel Pharmaceuticals, Inc.

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start: October 1, 2014
• Primary Completion (Actual): June 2, 2020
• Study Completion (Actual): June 2, 2020

Study Registration Dates

• First Submitted: February 27, 2014
• First Submitted That Met QC Criteria: February 28, 2014
• First Posted (Estimated): March 4, 2014

Study Record Updates

• Last Update Posted (Estimated): December 11, 2023
• Last Update Submitted That Met QC Criteria: December 6, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Thrombocytopenia; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic
• Other Study ID Numbers: C-935788-049; 2013-005454-30 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO