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

Phase 3
Completed
Conditions
Immune Thrombocytopenic Purpura
Interventions
Registration Number
NCT02077192
Lead Sponsor
Rigel Pharmaceuticals
Brief Summary

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

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
123
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 & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Fostamatinib DisodiumFostamatinib DisodiumFostamatinib Disodium tablet 100 mg or 150 mg by mouth twice a day
Primary Outcome Measures
NameTimeMethod
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 1Up 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 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.

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 2Up to 12 months

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.

Secondary Outcome Measures
NameTimeMethod
Duration of Platelet Response Based on Platelet Count and Rescue MedicationUp to 12 months

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.

Percentage of Subjects in Whom a Reduction in the Dose of Concomitant ITP Therapy Can be Achieved While Maintaining an Adequate Platelet CountUp to 12 months

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.

Trial Locations

Locations (55)

HAGA ziekenhuis

🇳🇱

Den Haag, NL, Netherlands

UMHAT Aleksandrovska, EAD

🇧🇬

Sofia, Bulgaria

East Carolina University, Brody School of Medicine

🇺🇸

Greenville, North Carolina, United States

Horizon Oncology Research, Inc

🇺🇸

Lafayette, Indiana, United States

St. Michael's Hospital

🇨🇦

Toronto, Ontario, Canada

Colchester General Hospital

🇬🇧

Colchester, Essex, United Kingdom

Fakultni nemocnice Ostrava

🇨🇿

Ostrava-Poruba, Czechia

Hospital Universitario La Paz

🇪🇸

Madrid, Spain

The Alfred

🇦🇺

Melbourne, Victoria, Australia

Fakultni nemocnice Brno

🇨🇿

Brno, Czechia

Hospital Universitari Vall d'Hebron

🇪🇸

Barcelona, Spain

Sykehuset Østfold Kalnes

🇳🇴

Grålum, Norway

Center for Cancer and Blood Disorders

🇺🇸

Bethesda, Maryland, United States

Signal Point Clinical Research Center LLC

🇺🇸

Middletown, Ohio, United States

Concord Repatriation General Hospital

🇦🇺

Concord, New South Wales, Australia

Wojewodzki Szpital Specjalistyczny im. J. Korczaka

🇵🇱

Slupsk, PO, Poland

Specjalistyczny Gabinet Lekarski

🇵🇱

Lublin, Poland

Prince of Wales Hospital

🇦🇺

Randwick, New South Wales, Australia

Westmead Hospital

🇦🇺

Westmead, New South Wales, Australia

Wojewódzki Szpital Specjalistyczny im. M. Kopernika w Łodzi

🇵🇱

Lodz, Poland

Hospital Universitari i Politécnic La Fe de Valencia

🇪🇸

Valencia, Spain

St. James's Hospital

🇬🇧

Leeds, United Kingdom

James Paget University Hospital

🇬🇧

Great Yarmouth, United Kingdom

Cancer and Haematology Centre

🇬🇧

Oxford, United Kingdom

Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia" - Clinica Ematologica

🇮🇹

Udine, Italy

Kent & Canterbury Hospital

🇬🇧

Canterbury, United Kingdom

University Hospital of North Midlands NHS Trust, Royal Stoke University Hospital

🇬🇧

Stoke-on-Trent, United Kingdom

Perth Blood Institute

🇦🇺

Nedlands, Western Australia, Australia

Pecsi Tudomanyegyetem Klinikai Kozpont, I. sz. Belgyogyaszati Klinika

🇭🇺

Pecs, Hungary

Instytut Hematologii I Transfuzjologii

🇵🇱

Warszawa, Poland

Spitalul Clinic Colentina, Hematologie

🇷🇴

Bucuresti, Romania

UMHAT Dr. Georgi Stranski, EAD, Pleven, Clinic of Hematology

🇧🇬

Pleven, Bulgaria

Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wrocrlaw

🇵🇱

Wroclaw, Dolnoslaski, Poland

Launceston General Hospital

🇦🇺

Launceston, Tasmania, Australia

Hanusch-Krankenhaus Wiener Gebietskrankenkasse

🇦🇹

Vienna, Austria

MHAT Hristo Botev, AD, Vratsa, First Internal Department

🇧🇬

Vratsa, Bulgaria

SPZOZ Szpital Uniwersytecki w Krakowie Pracownia Separacji Krwinek i Bank Komórek Krwiotwórczych Klinika Hematologii

🇵🇱

Kraków, Poland

Haukeland universitetssykehus, Helse Bergen HF

🇳🇴

Bergen, Norway

Herlev Hospital

🇩🇰

Herlev, DK, Denmark

Arizona Oncology Associates

🇺🇸

Tucson, Arizona, United States

Bleeding & Clotting Disorders Institute

🇺🇸

Peoria, Illinois, United States

Weill Cornell Medical College/New York Presbyterian Hospital

🇺🇸

New York, New York, United States

W.G. "Bill" Hefner VA Medical Center

🇺🇸

Salisbury, North Carolina, United States

Weill Cornell Medicine

🇺🇸

New York, New York, United States

Liverpool Hospital

🇦🇺

Liverpool, New South Wales, Australia

Specialized Hospital for Active Treatment of Hematology Diseases, EAD, Sofia, Department of Chemotherapy, Hemotherapy and Blood Inherited Diseases to Clinic of Clinical Hematology;

🇧🇬

Sofia, BG, Bulgaria

Istituto di Ematologia "Lorenzo e Ariosto Seràgnoli"

🇮🇹

Bologna, BO, Italy

Lkinika Hematologii I Transplantologii Uniwersyteckie Centrum Kliniczne

🇵🇱

Gdansk, Poland

Szpital Wojewodzki w Opolu

🇵🇱

Opole, Poland

Royal Victoria Infirmary

🇬🇧

Newcastle-upon-Tyne, UK, United Kingdom

Imperial College Healthcare NHS Trust

🇬🇧

London, United Kingdom

Leicester Royal Infirmary

🇬🇧

Leicester, United Kingdom

Hamilton Health Sciences Corporation

🇨🇦

Hamilton, Ontario, Canada

University College Hospital

🇬🇧

London, United Kingdom

Royal Liverpool University Hospital

🇬🇧

Liverpool, United Kingdom

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