A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHA

NCT ID: NCT03764618

Last Updated: 2023-05-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 90 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-04-24
• Study Completion Date: 2022-04-11

Brief Summary

The primary objective of this study is to assess the efficacy of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA).

Conditions

Warm Antibody Autoimmune Hemolytic Anemia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Fostamatinib

Initial dose is 100 mg by mouth (PO) twice a day (bid). At week 4 dose will be increased to fostamatinib 150 mg PO bid if subjects have adequately tolerated the study drug in the opinion of the Investigator.

Group Type: EXPERIMENTAL

Fostamatinib disodium

Intervention Type: DRUG

Fostamatinib (100mg PO bid or 150 mg PO bid)

The dose may be reduced at any time to a dose as low as fostamatinib 100 mg PO qd or matching placebo if dose limiting adverse events are observed.

Placebo

Initial dose is 100 mg by mouth (PO) twice a day (bid). At week 4 dose will be increased to placebo 150 mg PO bid if subjects have adequately tolerated the study drug in the opinion of the Investigator.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo

Interventions

Fostamatinib disodium

Fostamatinib (100mg PO bid or 150 mg PO bid)

The dose may be reduced at any time to a dose as low as fostamatinib 100 mg PO qd or matching placebo if dose limiting adverse events are observed.

Intervention Type: DRUG

Placebo

Placebo

Intervention Type: DRUG

Other Intervention Names

R935788; Fostamatinib

Eligibility Criteria

Inclusion Criteria

1. Subject must have a diagnosis of primary or secondary warm Antibody Autoimmune Hemolytic Anemia (wAIHA) as documented by a positive direct antiglobulin test (DAT) specific for anti-IgG or anti-IgA.

2. Have failed or not tolerated at least one prior wAIHA treatment regimen, including steroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, MMF, danazol, vincristine, ESA or splenectomy (folate, iron or other supplements do not fulfill this criterion).

3. Have haptoglobin <LLN or total bilirubin >ULN or lactate dehydrogenase (LDH) >ULN.

4. At screening, subject's hemoglobin level must be ≤9 g/dL OR if hemoglobin value >9 g/dL and <10 g/dL, subject must be on an allowed wAIHA treatment AND the subject must have documented symptoms related to anemia (e.g., weakness, dizziness, fatigue, shortness of breath, chest pain).

5. Karnofsky performance status (KPS) ≥70.

6. Subject's concurrent treatment for wAIHA may consist of no more than two of any of the following agents: azathioprine, steroids, ESAs, mycophenolate mofetil, dapsone or danazol at a stable dose

Exclusion Criteria

1. Subject with other types of Antibody Autoimmune Hemolytic Anemia (AIHA) (e.g., cold antibody AIHA, cold agglutinin syndrome, mixed type AIHA, or paroxysmal cold hemoglobinuria).

2. Subject has AIHA secondary to autoimmune disease, including systemic lupus erythematosus (SLE), or lymphoid malignancy if the underlying disease is not stable or is not well-controlled on current therapy, per investigator medical judgement.

3. Subject has uncontrolled or poorly controlled hypertension, defined as systolic blood pressure ≥135 mmHg or diastolic blood pressure ≥85 mmHg, whether or not the subject is receiving anti-hypertensive treatment.

4. Subject has one or more of the following laboratory abnormalities at screening: neutrophil count of <1,000/μL or platelet count of <30,000/μL, unless due to Evans syndrome; transaminase levels (i.e., alanine aminotransferase [ALT] or aspartate aminotransferase [AST]) >1.5 x ULN.

5. Has documented active hepatitis B or hepatitis C infection or HIV infection.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 100 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Rigel Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Banner MD Anderson Cancer Center

Gilbert, Arizona, United States

Mayo Clinic Hospital

Phoenix, Arizona, United States

Arizona Oncology Associates, PC--HOPE Division

Tucson, Arizona, United States

Moores UC San Diego Cancer Center

La Jolla, California, United States

University of Southern California

Los Angeles, California, United States

Harbor UCLA - Lundquist Institute

Torrance, California, United States

The Oncology Institute of Hope and Innovation

Whittier, California, United States

Banner MD Anderson Cancer Center at North Colorado Medical Center

Greeley, Colorado, United States

Georgetown University - Lombardi Comprehensive Cancer Center

Washington D.C., District of Columbia, United States

Cancer Specialists of North Florida

Jacksonville, Florida, United States

Piedmont Cancer Institute

Atlanta, Georgia, United States

Affiliated Oncologists

Chicago Ridge, Illinois, United States

John Hopkins Bayview Medical Center

Baltimore, Maryland, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Comprehensive Cancer Centers of Nevada

Las Vegas, Nevada, United States

Rutgers - Robert Wood Johnson Medical School

New Brunswick, New Jersey, United States

Montefiore Medical Center

The Bronx, New York, United States

Duke Cancer Network

Clayton, North Carolina, United States

Medical University of South Carolina

Charleston, South Carolina, United States

Texas Oncology - Baylor Research Institute

Dallas, Texas, United States

Clear Lake Specialties, Research Dept.

Webster, Texas, United States

American Oncology Network Vista Oncology Division

Olympia, Washington, United States

Swedish Cancer Institute

Seattle, Washington, United States

University of Washington

Seattle, Washington, United States

Versiti Wisconsin, Inc.

Milwaukee, Wisconsin, United States

Marshfield Clinic Cancer Center - Stevens Point

Stevens Point, Wisconsin, United States

Concord Repatriation General Hospital

Sydney, New South Wales, Australia

Princess Alexandra Hospital - Cancer Trials Unit

Brisbane, Queensland, Australia

The Alfred Hospital

Melbourne, Victoria, Australia

Royal Perth Hospital

Perth, Western Australia, Australia

Universitätsklinik Innsbruck - Innere Medizin V

Innsbruck, , Austria

Universitätsklinikum Salzburg, 3.Medizin/Onkologie

Salzburg, , Austria

Medizinsche Universität Wien

Vienna, , Austria

Hanusch-Krankenhaus

Vienna, , Austria

Vitebsk Regional Clinical Hospital

Vitebsk, , Belarus

Vitebsk Regional Clinical Oncology Dispensary

Vitebsk, , Belarus

Ziekenhuis Network Antwerp, Stuivenberg

Antwerp, , Belgium

Universitair Ziekenhuis Antwerpen - Hematologie

Edegem, , Belgium

AZ Nikolaas

Sint-Niklaas, , Belgium

University Multiprofile Hospital for Active Treatment - Dr Georgi Stranski EAD, Pleven, Clinic of Clinical Haematology

Pleven, , Bulgaria

University Multiprofile Hospital for Active Treatment 'Alexandrovska' EAD, Clinic of Clinical Haematology

Sofia, , Bulgaria

University Multiprofile Hospital for Active Treatment 'Sv. Ivan Rilski' EAD, Clinic of Clinical Hematology, Department of Clinical Hematology

Sofia, , Bulgaria

Specialized Hospital for Active Treatment of Hematological Diseases EAD, Sofia, Clinic of Clinical Haematology

Sofia, , Bulgaria

University Multiprofile Hospital for Active Treatment 'Sveta Marina' EAD, Varna, Clinic of Clinical Haematology

Varna, , Bulgaria

Hamilton Health Sciences- McMaster University Medical Centre

Hamilton, Ontario, Canada

The Ottawa Hospital

Ottawa, Ontario, Canada

St. Michael's Hospital

Toronto, Ontario, Canada

Fakultni nemocnice Brno Interni hematologicka a onkologicka klinika

Brno, , Czechia

Fakultni nemocnice Ostrava Klinika hematoonkologie

Ostrava, , Czechia

Ustav Hematologie a Krevni Transfuze

Prague, , Czechia

Aalborg University Hopital

Aalborg, , Denmark

Aarhus University Hospital - Dept of Hematology

Aarhus N, , Denmark

Herlev and Gentofte Hospital

Herlev, , Denmark

CHU Angers

Angers, , France

Institut d'hématologie de Basse Normandie, CHU Caen

Caen, , France

CHU Estaing - Chirurgie digestive et Médecine interne

Clermont-Ferrand, , France

CHU Henri Mondor

Créteil, , France

Hôpital Saint Antoine

Paris, , France

CHU de Bordeaux - GH Sud- Hôpital Haut Lévêque Service Médecine Interne et Maladies Infectieuses

Pessac, , France

CHU Toulouse, IUCT Oncopôle

Toulouse, , France

M. Zodelava Hematology Centre, Tbilisi

Tbilisi, , Georgia

LTD Multiprofile Clinic Consilium Medulla

Tbilisi, , Georgia

Universitätsklinikum Essen

Essen, , Germany

University of Leipzig Medical Center, Medical Department I - Haematology and Cell Therapy, Medical Oncology, Hemophilia

Leipzig, , Germany

Semmelweis Egyetem, Általános Orvostudományi Kar, I.sz. Belgyógyászati Klinika, Haematológiai Osztály,

Budapest, , Hungary

Szabolcs-Szatmár- Bereg Megyei Kórházak és Egyetemi Oktatókórház, Jósa András Oktatókórház

Nyíregyháza, , Hungary

Pécsi Tudományegyetem, Klinikai Központ, I.számú Belgyógyászati Klinikai, Hematológiai Tanszék

Pécs, , Hungary

ASST degli Spedali Civili di Brescia, Ematologia UOC

Brescia, , Italy

Fondazione IRCCS Ca'Granda - Ospedale Maggiore Policlinico di Milano - UO Ematologia

Milan, , Italy

SCDU Ematologia AOU "Maggiore della Carità"

Novara, , Italy

Ospedale Maggiore, SC Ematologia - Azienda Sanitaria Universitaria Giuliano Isontina

Trieste, , Italy

ULSS8 Berica Ospedale San Bortolo - Unità Operativa Complessa di Ematologia

Vicenza, , Italy

Academisch Medisch Centrum

Amsterdam, , Netherlands

Ålesund Hospital

Ålesund, , Norway

Haukeland University Hospital

Bergen, , Norway

Østfold Hopital

Grålum, , Norway

Coltea Clinical Hospital

Bucharest, , Romania

Emergency University Hospital Bucharest

Bucharest, , Romania

"Prof. Dr. Ion Chiricuta" Institute of Oncology Cluj-Napoca

Cluj-Napoca, , Romania

State Budgetary Healthcare Institution of Novosibirsk Region "City Clinical Hospital №2"

Novosibirsk, Russian Federation, Russia

National Research Center for Hematology

Moscow, , Russia

Botkin Moscow City Clinical Hospital

Moscow, , Russia

State Budgetary Healthcare Institution of Moscow city "City Clinical Hospital No 40 Department of Healthcare of Moscow city"

Moscow, , Russia

State Budgetary Healthcare Institution Oncology Dispensary No. 2 of the Ministry of Health of the Krasnodar Territory

Sochi, , Russia

Regional State Autonomous Healthcare Institution "Tomsk Regional Clinical Hospital"

Tomsk, , Russia

University Hospital Medical Center "Bezanijska Kosa", Department of Hematology

Belgrade, , Serbia

Clinical Center Nis, Clinic for Hematology

Niš, , Serbia

Clinical Centre of Vojvodina, Clinic for Hematology

Novi Sad, , Serbia

Hospital Vall d'Hebron

Barcelona, , Spain

Hospital Clinic de Barcelona

Barcelona, , Spain

Hospital Universitario Gregorio Marañón

Madrid, , Spain

Hospital Universitario Puerta de Hierro de Majadahonda

Madrid, , Spain

Hospital Universitario Virgen del Rocio

Seville, , Spain

Hospital Universitario y Politecnico La Fe - Servicio de Hematología

Valencia, , Spain

Cherkasy Regional Oncology Dispensary, Hematology Center

Cherkasy, , Ukraine

City Clinical Hospital № 4, Hematology Center

Dnipro, , Ukraine

Kyiv City Clinical Hospital №9, hematology department №1

Kyiv, , Ukraine

East Kent Haemophilia Centre, Kent and Canterbury Hospital

Canterbury, Kent, United Kingdom

Roald Dahl Haemostasis and Thrombosis Centre, Royal Liverpool University Hospital, Liverpool University Hospitals NHS Foundation Trust

Liverpool, , United Kingdom

Hammersmith Hospital

London, , United Kingdom

The Royal London Hospital, Bart's Health NHS Trust

London, , United Kingdom

Sandwell and West Birmingham NHS Trust

West Bromwich, , United Kingdom

Countries

United States; Australia; Austria; Belarus; Belgium; Bulgaria; Canada; Czechia; Denmark; France; Georgia; Germany; Hungary; Italy; Netherlands; Norway; Romania; Russia; Serbia; Spain; Ukraine; United Kingdom

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

C-935788-057

Identifier Type: -

Identifier Source: org_study_id