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

NCT02077192 | Completed Phase 3 Results

• 2 other identifiers: C-935788-0492013-005454-30
• Study Type: interventional
• Target: 123
• Locations: 14 countries
• Sites: 54

Brief Summary

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

Conditions

Immune Thrombocytopenic Purpura

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (2)

• Duration of Platelet Response Based on Platelet Count and Rescue Medication

  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

  Up to 12 months

Study Arms (1)

Fostamatinib Disodium

EXPERIMENTAL

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

Drug: Fostamatinib Disodium

Interventions

Fostamatinib Disodium DRUG

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

Also known as: R935788, R788, Fostamatinib

Fostamatinib Disodium

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Rigel Pharmaceuticals: lead

Study Sites (55)

Arizona Oncology Associates

Tucson, Arizona, 85710, United States

Location

Bleeding & Clotting Disorders Institute

Peoria, Illinois, 61615, United States

Location

Horizon Oncology Research, Inc

Lafayette, Indiana, 47905, United States

Location

Center for Cancer and Blood Disorders

Bethesda, Maryland, 20817, United States

Location

Weill Cornell Medical College/New York Presbyterian Hospital

New York, New York, 10065, United States

Location

Weill Cornell Medicine

New York, New York, 10065, United States

Location

East Carolina University, Brody School of Medicine

Greenville, North Carolina, 27834, United States

Location

W.G. "Bill" Hefner VA Medical Center

Salisbury, North Carolina, 28144, United States

Location

Signal Point Clinical Research Center LLC

Middletown, Ohio, 45042, United States

Location

Concord Repatriation General Hospital

Concord, New South Wales, 2139, Australia

Location

Liverpool Hospital

Liverpool, New South Wales, 2170, Australia

Location

Prince of Wales Hospital

Randwick, New South Wales, 2031, Australia

Location

Westmead Hospital

Westmead, New South Wales, 2145, Australia

Location

Launceston General Hospital

Launceston, Tasmania, 7250, Australia

Location

The Alfred

Melbourne, Victoria, 3004, Australia

Location

Perth Blood Institute

Nedlands, Western Australia, 6009, Australia

Location

Hanusch-Krankenhaus Wiener Gebietskrankenkasse

Vienna, 1140, Austria

Location

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, 1756, Bulgaria

Location

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

Pleven, 5800, Bulgaria

Location

UMHAT Aleksandrovska, EAD

Sofia, 1431, Bulgaria

Location

MHAT Hristo Botev, AD, Vratsa, First Internal Department

Vratsa, 3000, Bulgaria

Location

Hamilton Health Sciences Corporation

Hamilton, Ontario, L8N 3Z5, Canada

Location

St. Michael's Hospital

Toronto, Ontario, M5B1W8, Canada

Location

Fakultni nemocnice Brno

Brno, 625 00, Czechia

Location

Fakultni nemocnice Ostrava

Ostrava-Poruba, 708 52, Czechia

Location

Herlev Hospital

Herlev, DK, 2730, Denmark

Location

Pecsi Tudomanyegyetem Klinikai Kozpont, I. sz. Belgyogyaszati Klinika

Pécs, H-7624, Hungary

Location

Istituto di Ematologia "Lorenzo e Ariosto Seràgnoli"

Bologna, BO, 40138, Italy

Location

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

Udine, 33100, Italy

Location

HAGA ziekenhuis

The Hague, NL, 2545 CH, Netherlands

Location

Haukeland universitetssykehus, Helse Bergen HF

Bergen, 5021, Norway

Location

Sykehuset Østfold Kalnes

Grålum, 1714, Norway

Location

Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wrocrlaw

Wroclaw, Dolnoslaski, 50-367, Poland

Location

Wojewodzki Szpital Specjalistyczny im. J. Korczaka

Słupsk, PO, 76-200, Poland

Location

Lkinika Hematologii I Transplantologii Uniwersyteckie Centrum Kliniczne

Gdansk, 80-952, Poland

Location

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

Krakow, 31-501, Poland

Location

Wojewódzki Szpital Specjalistyczny im. M. Kopernika w Łodzi

Lodz, 93-510, Poland

Location

Specjalistyczny Gabinet Lekarski

Lublin, 20-601, Poland

Location

Szpital Wojewodzki w Opolu

Opole, 45-061, Poland

Location

Instytut Hematologii I Transfuzjologii

Warsaw, 02-776, Poland

Location

Spitalul Clinic Colentina, Hematologie

Bucharest, 020125, Romania

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Hospital Universitari i Politécnic La Fe de Valencia

Valencia, 46026, Spain

Location

Colchester General Hospital

Colchester, Essex, CO4 5JL, United Kingdom

Location

Royal Victoria Infirmary

Newcastle upon Tyne, UK, NE1 4LP, United Kingdom

Location

Kent & Canterbury Hospital

Canterbury, CT1 3NG, United Kingdom

Location

James Paget University Hospital

Great Yarmouth, NR31 6LA, United Kingdom

Location

St. James's Hospital

Leeds, LS9 7TF, United Kingdom

Location

Leicester Royal Infirmary

Leicester, LE1 5WW, United Kingdom

Location

Royal Liverpool University Hospital

Liverpool, L78XP, United Kingdom

Location

Imperial College Healthcare NHS Trust

London, W12 0HS, United Kingdom

Location

University College Hospital

London, WC1E 6AG, United Kingdom

Location

Cancer and Haematology Centre

Oxford, OX3 7LE, United Kingdom

Location

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

Stoke-on-Trent, ST4 6QG, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 33995988 DERIVED

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

fostamatinib

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Results Point of Contact

• Title: Director of Clinical Operations
• Organization: Rigel Pharmaceuticals, Inc.

clinicaltrials@rigel.com

(650) 624-1100

Study Officials

• Rigel Pharmaceuticals, Inc.

  Rigel Pharmaceuticals,Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 27, 2014
• First Posted: March 4, 2014
• Study Start: October 1, 2014
• Primary Completion: June 2, 2020
• Study Completion: June 2, 2020
• Last Updated: December 11, 2023
• Results First Posted: December 11, 2023

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will not share

Locations

CA (2); US (9); BU (4); HU (1); IT (2); NO (2); ES (3); AU (7); CZ (2); PL (8); GB (11); NL (1); RO (1); DK (1)