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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04138927




Registration number
NCT04138927
Ethics application status
Date submitted
23/10/2019
Date registered
25/10/2019
Date last updated
28/09/2020

Titles & IDs
Public title
A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
Scientific title
A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
Secondary ID [1] 0 0
C-935788-058
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

Experimental: Fostamatinib - Subjects who at any time during the C-935788-057 study achieved a hemoglobin response in the absence of rescue in the previous 4 weeks or a steroid dose greater than baseline will continue at their current dose (100mg or 150 mg) and regimen in the extension study.
All other subjects who enter the extension study will initially receive fostamatinib 100 mg PO bid. Starting at Week 4, the initial fostamatinib dose of 100 mg PO bid will be increased to fostamatinib 150 mg PO bid if subjects have adequately tolerated the study drug, based on the investigator's judgment.


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.

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

Outcomes
Primary outcome [1] 0 0
Durable Response - A primary efficacy endpoint is the proportion of subjects who achieve a durable hemoglobin response.
Timepoint [1] 0 0
104 weeks
Primary outcome [2] 0 0
Durable Response and Hemoglobin Response - The proportion of any subjects who achieve a durable response and have a hemoglobin response at 48 weeks of exposure
Timepoint [2] 0 0
104 weeks
Secondary outcome [1] 0 0
Hemoglobin Response by Week 24 - Proportion of placebo crossover subjects who have achieved a hemoglobin response by week 24 of this study
Timepoint [1] 0 0
24 weeks
Secondary outcome [2] 0 0
Partial Hemoglobin Response - The proportion of any subjects who achieve a partial hemoglobin response
Timepoint [2] 0 0
24 Weeks
Secondary outcome [3] 0 0
Median Hemoglobin Value at Week 24 - Median hemoglobin value at week 24 of this study, for placebo crossover subjects
Timepoint [3] 0 0
24 weeks
Secondary outcome [4] 0 0
Median change from baseline in hemoglobin at week 24 - Median change from baseline in hemoglobin at week 24 of this study for placebo crossover subjects
Timepoint [4] 0 0
24 Weeks
Secondary outcome [5] 0 0
Durable Response and Maintain a Hemoglobin Response at 24 and 36 Weeks - The proportion of any subjects who achieve a durable response and maintain a hemoglobin response at 24 and 36 weeks of exposure
Timepoint [5] 0 0
36 Weeks

Eligibility
Key inclusion criteria
1. Subject must be willing and able to give written informed consent by signing an IRB
approved Informed Consent Form prior to undergoing any study-specific procedures.

2. Subject must have completed all 24 weeks of participation in the study C-935788-057.
Minimum age
18 Years
Maximum age
100 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Any subject who discontinued participation in Study C-935788-057 prior to Week 24.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Cancer Trials Unit - Brisbane
Recruitment hospital [2] 0 0
The Alfred Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
4201 - Brisbane
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Maryland
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Nevada
Country [5] 0 0
United States of America
State/province [5] 0 0
Washington
Country [6] 0 0
Austria
State/province [6] 0 0
Vienna
Country [7] 0 0
Belgium
State/province [7] 0 0
Sint-Niklaas
Country [8] 0 0
Czechia
State/province [8] 0 0
Brno
Country [9] 0 0
Czechia
State/province [9] 0 0
Ostrava
Country [10] 0 0
France
State/province [10] 0 0
Pessac
Country [11] 0 0
Georgia
State/province [11] 0 0
Tbilisi
Country [12] 0 0
Germany
State/province [12] 0 0
Essen
Country [13] 0 0
Italy
State/province [13] 0 0
Milano

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

Ethics approval
Ethics application status

Summary
Brief summary
The primary objectives of this study are:

- To establish the efficacy of fostamatinib in placebo crossover subjects with warm
antibody autoimmune hemolytic anemia (wAIHA)

- To determine the ability to maintain a response in subjects receiving fostamatinib for
wAIHA
Trial website
https://clinicaltrials.gov/show/NCT04138927
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

Summary results
Other publications