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.

A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2019-001882-34-ES

Enrollment

Registered

2019-12-02

Start date

2020-01-28

Completion date

Unknown

Last updated

2023-09-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Interventions

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

Eligibility

Sex/Gender

All

Inclusion 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

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

Design outcomes

Primary

Measure	Time frame
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.;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;Timepoint(s) of evaluation of this end point: 24 week evaluation	—

Measure

Time frame

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.;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;Timepoint(s) of evaluation of this end point: 24 week evaluation

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Secondary

Measure	Time frame
Secondary end point(s): None;Timepoint(s) of evaluation of this end point: None	—

Countries

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

Public ContactRegulatory Department

Pharm-Olam International (Spain) S.L.U

regulatory.spain@pharm-olam.com+34 91-145 91 10

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026