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.

• Register: EUCTR
• Last refreshed on: 30 September 2024
• Main ID: EUCTR2019-001882-34-DK
• Date of registration: 20/01/2020
• 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: 03/06/2020
• Target sample size: 81
• Recruitment status: Not Recruiting
• 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, Czechia, Denmark, France, Georgia, Germany, Hungary, Italy, Netherlands, Norway, Romania, Russian Federation, Serbia, Spain, Ukraine, United Kingdom, United States

Contacts

• Name: Sandra Tong
• Affiliation: Rigel Pharmaceuticals, Inc.

• Name: Sandra Tong
• 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 14 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

Warm antibody autoimmune hemolytic anemia (wAIHA)
MedDRA version: 20.0 Level: LLT Classification code 10003825 Term: Autoimmune hemolytic anemia System Organ Class: 100000004851

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

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)

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

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)

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)

C-935788-058

2019-001882-34-ES

Source(s) of Monetary Support

Rigel Pharmaceuticals, Inc.

Ethics review

Status: Approved
Approval date: 03/06/2020
Contact:

Results

• Date Posted: 26/07/2023
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2019-001882-34/results