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

Immune Thrombocytopenic Purpura Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02077192
• Other study ID #: C-935788-049
• Secondary ID: 2013-005454-30
• Status: Completed
• Phase: Phase 3
• Start date: October 2014
• Est. completion date: June 2, 2020

Study information

• Verified date: December 2023
• Source: Rigel Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 123
• Est. completion date: June 2, 2020
• Est. primary completion date: June 2, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

Drug:
• Fostamatinib Disodium
Fostamatinib Disodium tablet 100 mg or 150 mg by mouth twice a day

Locations

Country	Name	City	State
Australia	Concord Repatriation General Hospital	Concord	New South Wales
Australia	Launceston General Hospital	Launceston	Tasmania
Australia	Liverpool Hospital	Liverpool	New South Wales
Australia	The Alfred	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
Austria	Hanusch-Krankenhaus Wiener Gebietskrankenkasse	Vienna
Bulgaria	UMHAT Dr. Georgi Stranski, EAD, Pleven, Clinic of Hematology	Pleven
Bulgaria	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	UMHAT Aleksandrovska, EAD	Sofia
Bulgaria	MHAT Hristo Botev, AD, Vratsa, First Internal Department	Vratsa
Canada	Hamilton Health Sciences Corporation	Hamilton	Ontario
Canada	St. Michael's Hospital	Toronto	Ontario
Czechia	Fakultni nemocnice Brno	Brno
Czechia	Fakultni nemocnice Ostrava	Ostrava-Poruba
Denmark	Herlev Hospital	Herlev	DK
Hungary	Pecsi Tudomanyegyetem Klinikai Kozpont, I. sz. Belgyogyaszati Klinika	Pecs
Italy	Istituto di Ematologia "Lorenzo e Ariosto Seràgnoli"	Bologna	BO
Italy	Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia" - Clinica Ematologica	Udine
Netherlands	HAGA ziekenhuis	Den Haag	NL
Norway	Haukeland universitetssykehus, Helse Bergen HF	Bergen
Norway	Sykehuset Østfold Kalnes	Grålum
Poland	Lkinika Hematologii I Transplantologii Uniwersyteckie Centrum Kliniczne	Gdansk
Poland	SPZOZ Szpital Uniwersytecki w Krakowie Pracownia Separacji Krwinek i Bank Komórek Krwiotwórczych Klinika Hematologii	Kraków
Poland	Wojewódzki Szpital Specjalistyczny im. M. Kopernika w Lodzi	Lodz
Poland	Specjalistyczny Gabinet Lekarski	Lublin
Poland	Szpital Wojewodzki w Opolu	Opole
Poland	Wojewodzki Szpital Specjalistyczny im. J. Korczaka	Slupsk	PO
Poland	Instytut Hematologii I Transfuzjologii	Warszawa
Poland	Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wrocrlaw	Wroclaw	Dolnoslaski
Romania	Spitalul Clinic Colentina, Hematologie	Bucuresti
Spain	Hospital Universitari Vall d'Hebron	Barcelona
Spain	Hospital Universitario La Paz	Madrid
Spain	Hospital Universitari i Politécnic La Fe de Valencia	Valencia
United Kingdom	Kent & Canterbury Hospital	Canterbury
United Kingdom	Colchester General Hospital	Colchester	Essex
United Kingdom	James Paget University Hospital	Great Yarmouth
United Kingdom	St. James's Hospital	Leeds
United Kingdom	Leicester Royal Infirmary	Leicester
United Kingdom	Royal Liverpool University Hospital	Liverpool
United Kingdom	Imperial College Healthcare NHS Trust	London
United Kingdom	University College Hospital	London
United Kingdom	Royal Victoria Infirmary	Newcastle-upon-Tyne	UK
United Kingdom	Cancer and Haematology Centre	Oxford
United Kingdom	University Hospital of North Midlands NHS Trust, Royal Stoke University Hospital	Stoke-on-Trent
United States	Center for Cancer and Blood Disorders	Bethesda	Maryland
United States	East Carolina University, Brody School of Medicine	Greenville	North Carolina
United States	Horizon Oncology Research, Inc	Lafayette	Indiana
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	Weill Cornell Medicine	New York	New York
United States	Bleeding & Clotting Disorders Institute	Peoria	Illinois
United States	W.G. "Bill" Hefner VA Medical Center	Salisbury	North Carolina
United States	Arizona Oncology Associates	Tucson	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
Rigel Pharmaceuticals

Countries where clinical trial is conducted

United States,  Australia,  Austria,  Bulgaria,  Canada,  Czechia,  Denmark,  Hungary,  Italy,  Netherlands,  Norway,  Poland,  Romania,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	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
Secondary	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