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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03764618




Registration number
NCT03764618
Ethics application status
Date submitted
3/12/2018
Date registered
5/12/2018

Titles & IDs
Public title
A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHA
Scientific title
A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
Secondary ID [1] 0 0
C-935788-057
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Warm Antibody Autoimmune Hemolytic Anemia 0 0
Condition category
Condition code
Blood 0 0 0 0
Anaemia
Blood 0 0 0 0
Anaemia
Blood 0 0 0 0
Haematological diseases
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Fostamatinib disodium
Treatment: Drugs - Placebo

Experimental: 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.

Placebo comparator: 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.


Treatment: Drugs: 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.

Treatment: Drugs: Placebo
Placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Durable Hemoglobin Response
Timepoint [1] 0 0
24 Weeks
Secondary outcome [1] 0 0
A Hemoglobin Response by Week 24
Timepoint [1] 0 0
24 weeks
Secondary outcome [2] 0 0
Change From Baseline in Hemoglobin Level of 2 g/dL or Greater
Timepoint [2] 0 0
24 weeks
Secondary outcome [3] 0 0
Change in Hemoglobin From Baseline to End of Treatment
Timepoint [3] 0 0
24 weeks
Secondary outcome [4] 0 0
Use of Rescue Antibody Autoimmune Hemolytic Anemia (AIHA) Regimens Use After Week 4
Timepoint [4] 0 0
24 weeks
Secondary outcome [5] 0 0
Change in Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-F)
Timepoint [5] 0 0
24 weeks

Eligibility
Key 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
Minimum age
18 Years
Maximum age
100 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/04/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
11/04/2022
Sample size
Target
Accrual to date
Final
90
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
Concord Repatriation General Hospital - Sydney
Recruitment hospital [2] 0 0
Princess Alexandra Hospital - Cancer Trials Unit - Brisbane
Recruitment hospital [3] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [4] 0 0
Royal Perth Hospital - Perth
Recruitment postcode(s) [1] 0 0
2139 - Sydney
Recruitment postcode(s) [2] 0 0
4201 - Brisbane
Recruitment postcode(s) [3] 0 0
3004 - Melbourne
Recruitment postcode(s) [4] 0 0
6000 - Perth
Recruitment outside Australia
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United States of America
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Arizona
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California
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Colorado
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District of Columbia
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Florida
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Georgia
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Illinois
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Maryland
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Massachusetts
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Nevada
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New Jersey
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New York
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North Carolina
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South Carolina
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Texas
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Washington
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Wisconsin
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Austria
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Innsbruck
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Austria
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Salzburg
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Austria
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Vienna
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Austria
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Wien
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Belarus
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Vitebsk
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Belgium
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Antwerpen
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Belgium
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Edegem
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Belgium
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Sint-Niklaas
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Bulgaria
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Pleven
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Bulgaria
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Sofia
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Bulgaria
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Varna
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Canada
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Ontario
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Czechia
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Brno
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Czechia
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Ostrava
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Czechia
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Praha
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Denmark
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Aalborg
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Aarhus N
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Herlev
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Angers
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France
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Caen
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Paris
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Pessac
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France
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Toulouse
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Georgia
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Tbilisi
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Germany
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Essen
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Germany
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Leipzig
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Budapest
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Hungary
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Nyíregyháza
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Hungary
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Pécs
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Italy
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Brescia
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Milano
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Italy
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Novara
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Italy
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Trieste
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Italy
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Vicenza
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Netherlands
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Amsterdam
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Bergen
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Grålum
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Norway
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Ålesund
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Romania
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Bucharest
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Romania
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Cluj-Napoca
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Russian Federation
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Moscow
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Russian Federation
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Novosibirsk
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Russian Federation
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Sochi
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Russian Federation
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Tomsk
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Serbia
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Belgrade
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Serbia
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Niš
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Serbia
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Novi Sad
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Spain
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Barcelona
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Spain
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Madrid
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Spain
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Sevilla
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Spain
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Valencia
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Ukraine
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Cherkasy
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Ukraine
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Dnipro
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Ukraine
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Kyiv
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United Kingdom
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Kent
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United Kingdom
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Liverpool
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United Kingdom
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London
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United Kingdom
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West Bromwich

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Rigel Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03764618