Trial Review

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04138927




Registration number
NCT04138927
Ethics application status
Date submitted
23/10/2019
Date registered
25/10/2019
Date last updated
17/03/2022

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 will continue at their dose and regimen from the Week 22 visit in the C-935788-057 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 is supplied in two (2) dosage strengths: 100 mg and 150 mg.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Adverse Events
Timepoint [1] 0 0
104 weeks
Primary outcome [2] 0 0
Blood Pressure
Timepoint [2] 0 0
104 weeks
Primary outcome [3] 0 0
Absolute Neutrophil Count (ANC)
Timepoint [3] 0 0
104 weeks
Secondary outcome [1] 0 0
Achievement of Durable Hemoglobin Response
Timepoint [1] 0 0
24 weeks
Secondary outcome [2] 0 0
Total Duration of Response
Timepoint [2] 0 0
During the Intervention period up to 104 weeks
Secondary outcome [3] 0 0
Corticosteroid dose
Timepoint [3] 0 0
During the Intervention period up to 104 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
Sex
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
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/10/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/04/2024
Actual
Sample size
Target
90
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Princess Alexandra Hospital - 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
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Nevada
Country [6] 0 0
United States of America
State/province [6] 0 0
Washington
Country [7] 0 0
Austria
State/province [7] 0 0
Vienna
Country [8] 0 0
Austria
State/province [8] 0 0
Wien
Country [9] 0 0
Belarus
State/province [9] 0 0
Vitebsk
Country [10] 0 0
Belgium
State/province [10] 0 0
Sint-Niklaas
Country [11] 0 0
Bulgaria
State/province [11] 0 0
Pleven
Country [12] 0 0
Bulgaria
State/province [12] 0 0
Sofia
Country [13] 0 0
Czechia
State/province [13] 0 0
Brno
Country [14] 0 0
Czechia
State/province [14] 0 0
Ostrava
Country [15] 0 0
France
State/province [15] 0 0
Pessac
Country [16] 0 0
Georgia
State/province [16] 0 0
Tbilisi
Country [17] 0 0
Germany
State/province [17] 0 0
Essen
Country [18] 0 0
Italy
State/province [18] 0 0
Milano
Country [19] 0 0
Italy
State/province [19] 0 0
Milan
Country [20] 0 0
Italy
State/province [20] 0 0
Novara
Country [21] 0 0
Netherlands
State/province [21] 0 0
Amsterdam
Country [22] 0 0
Norway
State/province [22] 0 0
Bergen
Country [23] 0 0
Russian Federation
State/province [23] 0 0
Moscow
Country [24] 0 0
Russian Federation
State/province [24] 0 0
Sochi
Country [25] 0 0
Serbia
State/province [25] 0 0
Novi Sad
Country [26] 0 0
Spain
State/province [26] 0 0
Barcelona
Country [27] 0 0
Ukraine
State/province [27] 0 0
Dnipro
Country [28] 0 0
Ukraine
State/province [28] 0 0
Kyiv
Country [29] 0 0
United Kingdom
State/province [29] 0 0
Liverpool

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

Ethics approval
Ethics application status

Summary
Brief summary
The primary objective of this study is:

• To evaluate the long-term safety of fostamatinib in subjects with warm antibody autoimmune
hemolytic anemia (wAIHA).
Trial website
https://clinicaltrials.gov/ct2/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

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04138927