Clinical trial NCT03764618: A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHA
Official title: A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Warm Antibody Autoimmune Hemolytic Anemia
Keywords: wAIHA
Warm Antibody Autoimmune Hemolytic Anemia
Warm Autoimmune Hemolytic Anemia
Lead sponsor: Rigel Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2018-12-05
Enrollment: 90
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


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.

Primary outcomes:
measure: Durable Hemoglobin Response
timeFrame: 24 Weeks
Secondary outcomes:
measure: A Hemoglobin Response by Week 24
timeFrame: 24 weeks
measure: Change From Baseline in Hemoglobin Level of 2 g/dL or Greater
timeFrame: 24 weeks
measure: Change in Hemoglobin From Baseline to End of Treatment
timeFrame: 24 weeks
measure: Use of Rescue Antibody Autoimmune Hemolytic Anemia (AIHA) Regimens Use After Week 4
timeFrame: 24 weeks
measure: Change in Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-F)
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Gilbert
country: United States
facility: Banner MD Anderson Cancer Center
state: Arizona
city: Phoenix
country: United States
facility: Mayo Clinic Hospital
state: Arizona
city: Tucson
country: United States
facility: Arizona Oncology Associates, PC--HOPE Division
state: Arizona
city: La Jolla
country: United States
facility: Moores UC San Diego Cancer Center
state: California
city: Los Angeles
country: United States
facility: University of Southern California
state: California
city: Torrance
country: United States
facility: Harbor UCLA - Lundquist Institute
state: California
city: Whittier
country: United States
facility: The Oncology Institute of Hope and Innovation
state: California
city: Greeley
country: United States
facility: Banner MD Anderson Cancer Center at North Colorado Medical Center
state: Colorado
city: Washington
country: United States
facility: Georgetown University - Lombardi Comprehensive Cancer Center
state: District of Columbia
city: Jacksonville
country: United States
facility: Cancer Specialists of North Florida
state: Florida
city: Atlanta
country: United States
facility: Piedmont Cancer Institute
state: Georgia
city: Chicago Ridge
country: United States
facility: Affiliated Oncologists
state: Illinois
city: Baltimore
country: United States
facility: John Hopkins Bayview Medical Center
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Las Vegas
country: United States
facility: Comprehensive Cancer Centers of Nevada
state: Nevada
city: New Brunswick
country: United States
facility: Rutgers - Robert Wood Johnson Medical School
state: New Jersey
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Clayton
country: United States
facility: Duke Cancer Network
state: North Carolina
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Dallas
country: United States
facility: Texas Oncology - Baylor Research Institute
state: Texas
city: Webster
country: United States
facility: Clear Lake Specialties, Research Dept.
state: Texas
city: Olympia
country: United States
facility: American Oncology Network Vista Oncology Division
state: Washington
city: Seattle
country: United States
facility: Swedish Cancer Institute
state: Washington
city: Seattle
country: United States
facility: University of Washington
state: Washington
city: Milwaukee
country: United States
facility: Versiti Wisconsin, Inc.
state: Wisconsin
city: Stevens Point
country: United States
facility: Marshfield Clinic Cancer Center - Stevens Point
state: Wisconsin
city: Sydney
country: Australia
facility: Concord Repatriation General Hospital
state: New South Wales
city: Brisbane
country: Australia
facility: Princess Alexandra Hospital - Cancer Trials Unit
state: Queensland
city: Melbourne
country: Australia
facility: The Alfred Hospital
state: Victoria
city: Perth
country: Australia
facility: Royal Perth Hospital
state: Western Australia
city: Innsbruck
country: Austria
facility: Universitätsklinik Innsbruck - Innere Medizin V
city: Salzburg
country: Austria
facility: Universitätsklinikum Salzburg, 3.Medizin/Onkologie
city: Vienna
country: Austria
facility: Hanusch-Krankenhaus
city: Wien
country: Austria
facility: Medizinsche Universität Wien
city: Vitebsk
country: Belarus
facility: Vitebsk Regional Clinical Hospital
city: Vitebsk
country: Belarus
facility: Vitebsk Regional Clinical Oncology Dispensary
city: Antwerpen
country: Belgium
facility: Ziekenhuis Network Antwerp, Stuivenberg
city: Edegem
country: Belgium
facility: Universitair Ziekenhuis Antwerpen - Hematologie
city: Sint-Niklaas
country: Belgium
facility: AZ Nikolaas
city: Pleven
country: Bulgaria
facility: University Multiprofile Hospital for Active Treatment - Dr Georgi Stranski EAD, Pleven, Clinic of Clinical Haematology
city: Sofia
country: Bulgaria
facility: University Multiprofile Hospital for Active Treatment 'Alexandrovska' EAD, Clinic of Clinical Haematology
city: Sofia
country: Bulgaria
facility: University Multiprofile Hospital for Active Treatment 'Sv. Ivan Rilski' EAD, Clinic of Clinical Hematology, Department of Clinical Hematology
city: Sofia
country: Bulgaria
facility: Specialized Hospital for Active Treatment of Hematological Diseases EAD, Sofia, Clinic of Clinical Haematology
city: Varna
country: Bulgaria
facility: University Multiprofile Hospital for Active Treatment 'Sveta Marina' EAD, Varna, Clinic of Clinical Haematology
city: Hamilton
country: Canada
facility: Hamilton Health Sciences- McMaster University Medical Centre
state: Ontario
city: Ottawa
country: Canada
facility: The Ottawa Hospital
state: Ontario
city: Toronto
country: Canada
facility: St. Michael's Hospital
state: Ontario
city: Brno
country: Czechia
facility: Fakultni nemocnice Brno Interni hematologicka a onkologicka klinika
city: Ostrava
country: Czechia
facility: Fakultni nemocnice Ostrava Klinika hematoonkologie
city: Praha
country: Czechia
facility: Ustav Hematologie a Krevni Transfuze
city: Aalborg
country: Denmark
facility: Aalborg University Hopital
city: Aarhus N
country: Denmark
facility: Aarhus University Hospital - Dept of Hematology
city: Herlev
country: Denmark
facility: Herlev and Gentofte Hospital
city: Angers
country: France
facility: CHU Angers
city: Caen
country: France
facility: Institut d'hématologie de Basse Normandie, CHU Caen
city: Clermont-Ferrand
country: France
facility: CHU Estaing - Chirurgie digestive et Médecine interne
city: Créteil
country: France
facility: CHU Henri Mondor
city: Paris
country: France
facility: Hôpital Saint Antoine
city: Pessac
country: France
facility: CHU de Bordeaux - GH Sud- Hôpital Haut Lévêque Service Médecine Interne et Maladies Infectieuses
city: Toulouse
country: France
facility: CHU Toulouse, IUCT Oncopôle
city: Tbilisi
country: Georgia
facility: M. Zodelava Hematology Centre, Tbilisi
city: Tbilisi
country: Georgia
facility: LTD Multiprofile Clinic Consilium Medulla
city: Essen
country: Germany
facility: Universitätsklinikum Essen
city: Leipzig
country: Germany
facility: University of Leipzig Medical Center, Medical Department I - Haematology and Cell Therapy, Medical Oncology, Hemophilia
city: Budapest
country: Hungary
facility: Semmelweis Egyetem, Általános Orvostudományi Kar, I.sz. Belgyógyászati Klinika, Haematológiai Osztály,
city: Nyíregyháza
country: Hungary
facility: Szabolcs-Szatmár- Bereg Megyei Kórházak és Egyetemi Oktatókórház, Jósa András Oktatókórház
city: Pécs
country: Hungary
facility: Pécsi Tudományegyetem, Klinikai Központ, I.számú Belgyógyászati Klinikai, Hematológiai Tanszék
city: Brescia
country: Italy
facility: ASST degli Spedali Civili di Brescia, Ematologia UOC
city: Milano
country: Italy
facility: Fondazione IRCCS Ca'Granda - Ospedale Maggiore Policlinico di Milano - UO Ematologia
city: Novara
country: Italy
facility: SCDU Ematologia AOU "Maggiore della Carità"
city: Trieste
country: Italy
facility: Ospedale Maggiore, SC Ematologia - Azienda Sanitaria Universitaria Giuliano Isontina
city: Vicenza
country: Italy
facility: ULSS8 Berica Ospedale San Bortolo - Unità Operativa Complessa di Ematologia
city: Amsterdam
country: Netherlands
facility: Academisch Medisch Centrum
city: Bergen
country: Norway
facility: Haukeland University Hospital
city: Grålum
country: Norway
facility: Østfold Hopital
city: Ålesund
country: Norway
facility: Ålesund Hospital
city: Bucharest
country: Romania
facility: Coltea Clinical Hospital
city: Bucharest
country: Romania
facility: Emergency University Hospital Bucharest
city: Cluj-Napoca
country: Romania
facility: "Prof. Dr. Ion Chiricuta" Institute of Oncology Cluj-Napoca
city: Moscow
country: Russian Federation
facility: National Research Center for Hematology
city: Moscow
country: Russian Federation
facility: Botkin Moscow City Clinical Hospital
city: Moscow
country: Russian Federation
facility: State Budgetary Healthcare Institution of Moscow city "City Clinical Hospital No 40 Department of Healthcare of Moscow city"
city: Novosibirsk
country: Russian Federation
facility: State Budgetary Healthcare Institution of Novosibirsk Region "City Clinical Hospital №2"
city: Sochi
country: Russian Federation
facility: State Budgetary Healthcare Institution Oncology Dispensary No. 2 of the Ministry of Health of the Krasnodar Territory
city: Tomsk
country: Russian Federation
facility: Regional State Autonomous Healthcare Institution "Tomsk Regional Clinical Hospital"
city: Belgrade
country: Serbia
facility: University Hospital Medical Center "Bezanijska Kosa", Department of Hematology
city: Niš
country: Serbia
facility: Clinical Center Nis, Clinic for Hematology
city: Novi Sad
country: Serbia
facility: Clinical Centre of Vojvodina, Clinic for Hematology
city: Barcelona
country: Spain
facility: Hospital Vall d'Hebron
city: Barcelona
country: Spain
facility: Hospital Clinic de Barcelona
city: Madrid
country: Spain
facility: Hospital Universitario Gregorio Marañón
city: Madrid
country: Spain
facility: Hospital Universitario Puerta de Hierro de Majadahonda
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
city: Valencia
country: Spain
facility: Hospital Universitario y Politecnico La Fe - Servicio de Hematología
city: Cherkasy
country: Ukraine
facility: Cherkasy Regional Oncology Dispensary, Hematology Center
city: Dnipro
country: Ukraine
facility: City Clinical Hospital № 4, Hematology Center
city: Kyiv
country: Ukraine
facility: Kyiv City Clinical Hospital №9, hematology department №1
city: Canterbury
country: United Kingdom
facility: East Kent Haemophilia Centre, Kent and Canterbury Hospital
state: Kent
city: Liverpool
country: United Kingdom
facility: Roald Dahl Haemostasis and Thrombosis Centre, Royal Liverpool University Hospital, Liverpool University Hospitals NHS Foundation Trust
city: London
country: United Kingdom
facility: Hammersmith Hospital
city: London
country: United Kingdom
facility: The Royal London Hospital, Bart's Health NHS Trust
city: West Bromwich
country: United Kingdom
facility: Sandwell and West Birmingham NHS Trust
Study officials:
References: