A Efficacy and Safety Study of Fostamatinib in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)

NCT02076412 | Completed Phase 3 Results DMC

• 2 other identifiers: C-935788-0482013-005453-76
• Study Type: interventional
• Target: 74
• Locations: 9 countries
• Sites: 35

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).

Conditions

Immune Thrombocytopenic Purpura

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Stable Platelet Response of at Least 50,000/µL

  Stable platelet response by Week 24 defined as a platelet count of at least 50,000/µL on at least 4 of the 6 visits between Weeks 14-24

  Baseline to Week 24

Secondary Outcomes (6)

• Number of Participants With Platelet Count ≥ 50,000/µL at Week 12

  Baseline to Week 12

• Number of Participants With Platelet Count ≥ 50,000/µL at Week 24

  Baseline to Week 24

• Number of Participants With Platelet Count ≥ 30,000/μL and at Least 20,000/μL Above Baseline at Week 12

  Baseline to Week 12

• Number of Participants With Platelet Count ≥ 30,000/μL and at Least 20,000/μL Above Baseline at Week 24

  Baseline to Week 24

• Frequency and Severity of Bleeding According to the ITP Bleeding Score (IBLS)

  Baseline to Week 24

Study Arms (2)

Fostamatinib Disodium

EXPERIMENTAL

Fostamatinib Disodium tablet 100 mg or 150 mg PO bid (morning and evening) over the course of 24 weeks.

Drug: Fostamatinib Disodium

Placebo

OTHER

Placebo tablet PO bid (morning and evening) over the course of 24 weeks

Drug: Placebo

Interventions

Fostamatinib Disodium DRUG

Fostamatinib Disodium tablet 100 mg or 150 mg PO bid (morning and evening) over the course of 24 weeks.

Also known as: R935788, R788, Fostamatinib

Fostamatinib Disodium

Placebo DRUG

Placebo tablet PO bid (morning and evening)

Placebo

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• Clinical diagnosis of autoimmune hemolytic anemia
• Uncontrolled or poorly controlled hypertension
• History of coagulopathy including prothrombotic conditions

Sponsors & Collaborators

• Rigel Pharmaceuticals: lead

Study Sites (35)

Hematology Oncology Associates of Rockland Division of Highland Medical PC

Nyack, New York, 10960, United States

Location

Medizinische Universitaet Wien / AKH Wien

Vienna, AU, 1090, Austria

Location

Hanusch-Krankenhaus Wiener Gebietskrankenkasse

Vienna, AU, 1140, Austria

Location

LKH Feldkirch at LKH Rankweil

Rankweil, 6830, 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

MHAT Hristo Botev, AD, Vratsa, First Internal Department

Vratsa, BG, 3000, Bulgaria

Location

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

Pleven, 5800, Bulgaria

Location

UMHAT Aleksandrovska, EAD, Clinic of Clinical Hematology

Sofia, 1431, Bulgaria

Location

Fakultni nemocnice Brno

Brno, CZ, 625 00, Czechia

Location

Hospital Kyjov

Kyjov, CZ, 69733, Czechia

Location

Fakultni nemocnice Ostrava

Ostrava-Poruba, 708 52, Czechia

Location

Fakultni nemocnice Kralovske Vinohrady

Prague, 100 34, Czechia

Location

Vseobecna fakultni nemocnice, Linterní Klinika, Klinika hematologie

Prague, 128 08, Czechia

Location

Universitaetsklinikum Giessen und Marburg (UKGM)

Giessen, DE, 35392, Germany

Location

Werlhof Institut GmbH

Hanover, DE, 30159, Germany

Location

Charit Berlin - Campus Benjamin Franklin

Berlin, 12203, Germany

Location

Marien Hospital Duesseldorf

Düsseldorf, 40479, Germany

Location

Universittsklinikum Essen

Essen, 45147, Germany

Location

Haukeland University Hospital

Bergen, 5021, Norway

Location

Sykehuset Østfold Fredrikstad

Fredrikstad, 1606, Norway

Location

Uniwersyteckie Centrum Kliniczne

Gdansk, 80-952, Poland

Location

Szpital Uniwersytecki

Krakow, 31-501, Poland

Location

Wojewódzki Szpital Specjalistyczny im. M. Kopernika w Łodzi

Lodz, 93-510, Poland

Location

Specjalistyczny Gabinet Lekarski

Lublin, 20-081, Poland

Location

Szpital Wojewodzki w Opolu

Opole, 45-064, Poland

Location

Wojewodzki Szpital Specjalistyczny im. J. Korczaka

Słupsk, 76-200, Poland

Location

Instytut Hematologii I Transfuzjologii

Warsaw, 02-776, Poland

Location

Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wrocrlaw

Wroclaw, 50-367, Poland

Location

Spitalul Clinic Judetean de Urgenta Tirgu-Mures, Sectia Medicina Interna 1, Compartiment Hematologie

Târgu Mureş, Mureș County, 540136, Romania

Location

Spitalul Clinic Colentina, Hematologie

Bucharest, 020125, Romania

Location

Spitalul Clinic Coltea, Hematologie

Bucharest, 030171, Romania

Location

Hospital Universitari Vall D'Hebron

Barcelona, 08035, Spain

Location

Hospital Universitari Germans Trias i Pujol

Barcelona, 08916, Spain

Location

Hospital Universitariola Paz

Madrid, 28046, Spain

Location

Hospital Universitari i Politécnic La Fe de Valencia

Valencia, 46026, Spain

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: Anne-Marie Duliege, MD
• Organization: Rigel

clinicaltrials@rigel.com

6506241100

Study Officials

• Rigel Pharmaceuticals, Inc.

  Rigel Pharmaceuticals,Inc.

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 26, 2014
• First Posted: March 3, 2014
• Study Start: January 1, 2015
• Primary Completion: August 1, 2016
• Study Completion: August 1, 2016
• Last Updated: January 25, 2019
• Results First Posted: January 25, 2019

Record last verified: 2019-01

Data Sharing

• IPD Sharing: Will not share

Locations

AU (3); ES (4); CZ (5); US (1); NO (2); BU (4); PL (8); DE (5); RO (3)