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

NCT03764618 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: C-935788-057
• Study Type: interventional
• Target: 90
• Locations: 20 countries
• Sites: 95

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

Keywords

wAIHA; Warm Antibody Autoimmune Hemolytic Anemia; Warm Autoimmune Hemolytic Anemia

Outcome Measures

Primary Outcomes (1)

• Durable Hemoglobin Response

  Proportion of subjects achieving a hemoglobin level ≥ 10 g/dL with an increase from Baseline in hemoglobin level of ≥ 2 g/dL on 3 consecutive available visits during the 24-week treatment period.

  24 Weeks

Secondary Outcomes (5)

• A Hemoglobin Response by Week 24

  24 weeks

• Change From Baseline in Hemoglobin Level of 2 g/dL or Greater

  24 weeks

• Change in Hemoglobin From Baseline to End of Treatment

  24 weeks

• Use of Rescue Antibody Autoimmune Hemolytic Anemia (AIHA) Regimens Use After Week 4

  24 weeks

• Change in Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-F)

  24 weeks

Study Arms (2)

Fostamatinib

EXPERIMENTAL

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.

Drug: Fostamatinib disodium

Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Interventions

Fostamatinib disodium 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.

Also known as: R935788, Fostamatinib

Fostamatinib

Placebo DRUG

Placebo

Placebo

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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).
• Have haptoglobin \<LLN or total bilirubin \>ULN or lactate dehydrogenase (LDH) \>ULN.
• 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).
• Karnofsky performance status (KPS) ≥70.
• 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

You may not qualify if:

• 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).
• 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.
• 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.
• 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.
• Has documented active hepatitis B or hepatitis C infection or HIV infection.

Sponsors & Collaborators

• Rigel Pharmaceuticals: lead

Study Sites (102)

Banner MD Anderson Cancer Center

Gilbert, Arizona, 85234, United States

Location

Mayo Clinic Hospital

Phoenix, Arizona, 85054, United States

Location

Arizona Oncology Associates, PC--HOPE Division

Tucson, Arizona, 85711, United States

Location

Moores UC San Diego Cancer Center

La Jolla, California, 92037, United States

Location

University of Southern California

Los Angeles, California, 90033, United States

Location

Harbor UCLA - Lundquist Institute

Torrance, California, 90502, United States

Location

The Oncology Institute of Hope and Innovation

Whittier, California, 90603, United States

Location

Banner MD Anderson Cancer Center at North Colorado Medical Center

Greeley, Colorado, 80538, United States

Location

Georgetown University - Lombardi Comprehensive Cancer Center

Washington D.C., District of Columbia, 20007, United States

Location

Cancer Specialists of North Florida

Jacksonville, Florida, 32256, United States

Location

Piedmont Cancer Institute

Atlanta, Georgia, 30318, United States

Location

Affiliated Oncologists

Chicago Ridge, Illinois, 60415, United States

Location

John Hopkins Bayview Medical Center

Baltimore, Maryland, 21205, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Comprehensive Cancer Centers of Nevada

Las Vegas, Nevada, 89169, United States

Location

Rutgers - Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States

Location

Montefiore Medical Center

The Bronx, New York, 10461, United States

Location

Duke Cancer Network

Clayton, North Carolina, 27520, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Texas Oncology - Baylor Research Institute

Dallas, Texas, 75246, United States

Location

Clear Lake Specialties, Research Dept.

Webster, Texas, 77598, United States

Location

American Oncology Network Vista Oncology Division

Olympia, Washington, 98506, United States

Location

Swedish Cancer Institute

Seattle, Washington, 98104, United States

Location

University of Washington

Seattle, Washington, 98109, United States

Location

Versiti Wisconsin, Inc.

Milwaukee, Wisconsin, 53226, United States

Location

Marshfield Clinic Cancer Center - Stevens Point

Stevens Point, Wisconsin, 54482, United States

Location

Concord Repatriation General Hospital

Sydney, New South Wales, 2139, Australia

Location

Princess Alexandra Hospital - Cancer Trials Unit

Brisbane, Queensland, 4201, Australia

Location

The Alfred Hospital

Melbourne, Victoria, 3004, Australia

Location

Royal Perth Hospital

Perth, Western Australia, 6000, Australia

Location

Universitätsklinik Innsbruck - Innere Medizin V

Innsbruck, 6020, Austria

Location

Universitätsklinikum Salzburg, 3.Medizin/Onkologie

Salzburg, 5020, Austria

Location

Medizinsche Universität Wien

Vienna, 1090, Austria

Location

Hanusch-Krankenhaus

Vienna, 1140, Austria

Location

Vitebsk Regional Clinical Hospital

Vitebsk, 210037, Belarus

Location

Vitebsk Regional Clinical Oncology Dispensary

Vitebsk, 210603, Belarus

Location

Ziekenhuis Network Antwerp, Stuivenberg

Antwerp, 2060, Belgium

Location

Universitair Ziekenhuis Antwerpen - Hematologie

Edegem, 2650, Belgium

Location

AZ Nikolaas

Sint-Niklaas, 9100, Belgium

Location

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

Pleven, 5800, Bulgaria

Location

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

Sofia, 1431, Bulgaria

Location

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

Sofia, 1431, Bulgaria

Location

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

Sofia, 1756, Bulgaria

Location

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

Varna, 9010, Bulgaria

Location

Hamilton Health Sciences- McMaster University Medical Centre

Hamilton, Ontario, L8S 4K1, Canada

Location

The Ottawa Hospital

Ottawa, Ontario, K1H8L6, Canada

Location

St. Michael's Hospital

Toronto, Ontario, M5B 1W8, Canada

Location

Fakultni nemocnice Brno Interni hematologicka a onkologicka klinika

Brno, 625 00, Czechia

Location

Fakultni nemocnice Ostrava Klinika hematoonkologie

Ostrava, 70852, Czechia

Location

Ustav Hematologie a Krevni Transfuze

Prague, 128 00, Czechia

Location

Aalborg University Hopital

Aalborg, 9000, Denmark

Location

Aarhus University Hospital - Dept of Hematology

Aarhus N, 8200, Denmark

Location

Herlev and Gentofte Hospital

Herlev, DK-2730, Denmark

Location

CHU Angers

Angers, 49100, France

Location

Institut d'hématologie de Basse Normandie, CHU Caen

Caen, 14033, France

Location

CHU Estaing - Chirurgie digestive et Médecine interne

Clermont-Ferrand, 63003, France

Location

CHU Henri Mondor

Créteil, 94000, France

Location

Hôpital Saint Antoine

Paris, 75012, France

Location

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

Pessac, 33603, France

Location

CHU Toulouse, IUCT Oncopôle

Toulouse, 31100, France

Location

M. Zodelava Hematology Centre, Tbilisi

Tbilisi, 0112, Georgia

Location

LTD Multiprofile Clinic Consilium Medulla

Tbilisi, 0186, Georgia

Location

Universitätsklinikum Essen

Essen, 45147, Germany

Location

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

Leipzig, 04103, Germany

Location

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

Budapest, 1083, Hungary

Location

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

Nyíregyháza, 4400, Hungary

Location

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

Pécs, 7624, Hungary

Location

ASST degli Spedali Civili di Brescia, Ematologia UOC

Brescia, 25123, Italy

Location

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

Milan, 20122, Italy

Location

SCDU Ematologia AOU "Maggiore della Carità"

Novara, 28100, Italy

Location

Ospedale Maggiore, SC Ematologia - Azienda Sanitaria Universitaria Giuliano Isontina

Trieste, 34125, Italy

Location

ULSS8 Berica Ospedale San Bortolo - Unità Operativa Complessa di Ematologia

Vicenza, 36100, Italy

Location

Academisch Medisch Centrum

Amsterdam, Netherlands

Location

Ålesund Hospital

Ålesund, 6017, Norway

Location

Haukeland University Hospital

Bergen, 5021, Norway

Location

Østfold Hopital

Grålum, 1714, Norway

Location

Coltea Clinical Hospital

Bucharest, 030171, Romania

Location

Emergency University Hospital Bucharest

Bucharest, 050098, Romania

Location

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

Cluj-Napoca, 400015, Romania

Location

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

Novosibirsk, Russian Federation, 630051, Russia

Location

National Research Center for Hematology

Moscow, 125167, Russia

Location

Botkin Moscow City Clinical Hospital

Moscow, 125284, Russia

Location

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

Moscow, 129301, Russia

Location

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

Sochi, 354057, Russia

Location

Regional State Autonomous Healthcare Institution "Tomsk Regional Clinical Hospital"

Tomsk, 634063, Russia

Location

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

Belgrade, 11080, Serbia

Location

Clinical Center Nis, Clinic for Hematology

Niš, 18000, Serbia

Location

Clinical Centre of Vojvodina, Clinic for Hematology

Novi Sad, 21000, Serbia

Location

Hospital Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital Clinic de Barcelona

Barcelona, 08036, Spain

Location

Hospital Universitario Gregorio Marañón

Madrid, 28009, Spain

Location

Hospital Universitario Puerta de Hierro de Majadahonda

Madrid, 28222, Spain

Location

Hospital Universitario Virgen del Rocio

Seville, 41013, Spain

Location

Hospital Universitario y Politecnico La Fe - Servicio de Hematología

Valencia, 46026, Spain

Location

Cherkasy Regional Oncology Dispensary, Hematology Center

Cherkasy, 18009, Ukraine

Location

City Clinical Hospital № 4, Hematology Center

Dnipro, 49102, Ukraine

Location

Kyiv City Clinical Hospital №9, hematology department №1

Kyiv, 04112, Ukraine

Location

East Kent Haemophilia Centre, Kent and Canterbury Hospital

Canterbury, Kent, CT1 3NG, United Kingdom

Location

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

Liverpool, L7 8XP, United Kingdom

Location

Hammersmith Hospital

London, United Kingdom

Location

The Royal London Hospital, Bart's Health NHS Trust

London, United Kingdom

Location

Sandwell and West Birmingham NHS Trust

West Bromwich, B71 4HJ, United Kingdom

Location

MeSH Terms

Interventions

fostamatinib

Results Point of Contact

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

clinicaltrials@rigel.com

(650) 624-1100

Publication Agreements

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

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: December 3, 2018
• First Posted: December 5, 2018
• Study Start: April 24, 2019
• Primary Completion: April 11, 2022
• Study Completion: April 11, 2022
• Last Updated: May 25, 2023
• Results First Posted: May 25, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

UA (3); GE (2); NL (1); SE (3); NO (3); GB (5); IT (5); RO (3); DK (3); HU (3); RU (6); ES (6); AU (4); BE (2); DE (2); CZ (3); FR (7); BU (5); CA (3); US (26)