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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 15 September 2020
Main ID:  EUCTR2019-001882-34-CZ
Date of registration: 20/12/2019
Prospective Registration: Yes
Primary sponsor: Rigel Pharmaceuticals, Inc.
Public title: This is a Phase 3 multi-center, open-label extension study to evaluate the long-term safety and efficacy of fostamatinib (R788) in subjects with wAIHA who have completed 24 weeks of participation in study C-935788-057.
Scientific title: A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
Date of first enrolment: 30/01/2020
Target sample size: 90
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-001882-34
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no
Randomised: no
Open: yes
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: no
Other: no
Number of treatment arms in the trial: 1
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Australia Austria Belarus Belgium Bulgaria Canada Czech Republic Denmark
France Georgia Germany Hungary Italy Netherlands Norway Romania
Russian Federation Serbia Spain Ukraine United Kingdom United States
Contacts
Name: Sandra Tong   
Address:  1180 Veterans Boulevard CA 94080 South San Francisco United States
Telephone: +1 650-624-1207
Email: stong@rigel.com
Affiliation:  Rigel Pharmaceuticals, Inc.
Name: Sandra Tong   
Address:  1180 Veterans Boulevard CA 94080 South San Francisco United States
Telephone: +1 650-624-1207
Email: stong@rigel.com
Affiliation:  Rigel Pharmaceuticals, Inc.
Key inclusion & exclusion criteria
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.
3. Female subjects must be either post-menopausal for at least 1 year or surgically sterile; or, if of childbearing potential, must not be pregnant or lactating and must agree to use a highly effective method of birth control throughout the duration of the trial and for 30 days following the last dose. Acceptable methods of birth control are defined as: hormonal contraception (pill, injection or implant) used consistently for at least 30 days prior to baseline an intrauterine device (IUD), or intrauterine hormone-releasing system (IUS), or true abstinence (i.e. abstinence is in line with the preferred and usual lifestyle of the subject).
4. In the investigator’s opinion, the subject has the ability to understand the nature of the study and any hazards of participation and to communicate satisfactorily with the investigator.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 81
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Any subject who discontinued participation in Study C-935788-057 prior to Week 24.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Warm antibody autoimmune hemolytic anemia (wAIHA)
MedDRA version: 20.0 Level: LLT Classification code 10003825 Term: Autoimmune hemolytic anemia System Organ Class: 100000004851
Intervention(s)

Product Name: Fostamatinib Disodium
Product Code: R935788
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Fostamatinib
CAS Number: 914295-16-2
Current Sponsor code: Fostamatinib Disodium R935788
Other descriptive name: R788, fostamatinib disodium hexahydrate (in USPI/FPI)
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-

Product Name: Fostamatinib Disodium
Product Code: R935788
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Fostamatinib
CAS Number: 914295-16-2
Current Sponsor code: Fostamatinib Disodium R935788
Other descriptive name: R788, fostamatinib disodium hexahydrate (in USPI/FPI)
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-

Primary Outcome(s)
Main Objective: The primary objective of this study is:
• To evaluate the long-term safety of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA)
Primary end point(s): Safety Endpoints:
• Adverse events
• Changes from baseline in selected laboratory values per CTCAE criteria V 5.0
• Changes from baseline in blood pressure over time

Efficacy Endpoints:
• Durable hemoglobin response (Yes/No) within 24 weeks of treatment
A hemoglobin response is defined as a hemoglobin level of >10 g/dL and =2 g/dL higher than the baseline (i.e, hemoglobin value prior to fostamatinib exposure), during the previous 4 weeks, the steroid dose is not higher than the baseline level, and rescue medication is not administered. A durable response is defined as a hemoglobin response on at least 3 scheduled visits during the first 24 weeks of this study.
• Hemoglobin response at 48 weeks (Yes/No) defined as subjects who achieve a durable response, as above, and have a hemoglobin response at 48 weeks
• Hemoglobin response by week 24
• Partial hemoglobin response, defined as a Hgb change from baseline of =2 g/dL
• Subjects who achieve steroid dose < 10 mg/day (prednisone equivalent)
• Change from baseline in hemoglobin values over time
• Change from baseline in steroid dose
• Subjects requiring any rescue during this study
• Overall duration of response and duration of first response
• Subjects who decrease any concomitant wAIHA therapy
Secondary Objective: The secondary objectives of this study are:
• To estimate the efficacy of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA)
• To estimate the durability of response in subjects receiving fostamatinib for wAIHA • To assess steroid use in subjects with warm antibody autoimmune hemolytic anemia (wAIHA) treated with fostamatinib..
Timepoint(s) of evaluation of this end point: 24 week evaluation
Secondary Outcome(s)
Secondary end point(s): None
Timepoint(s) of evaluation of this end point: None
Secondary ID(s)
2019-001882-34-ES
C-935788-058
Source(s) of Monetary Support
Rigel Pharmaceuticals, Inc.
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 30/01/2020
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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