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Fostamatinib disodium

Phase 3

Immune Thrombocytopenic Purpura | Small molecule | Hematology |Rigel Pharmaceuticals, Inc.|Last Updated: Dec 11, 2023

Target and mechanism

Molecular targetSYK
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials3
Total Enrollment273

FDA Designations

No designations recorded

Clinical trial landscape

Fostamatinib disodium · 8 trials · 4 indications

Phase 3 5Phase 2 3
NCT04138927A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic AnemiaWarm Antibody Autoimmune Hemolytic Anemia
ENROLLING BY_INVITATION90 Analytics
NCT03764618A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHAWarm Antibody Autoimmune Hemolytic Anemia
COMPLETED90 Analytics
NCT02076412A Efficacy and Safety Study of Fostamatinib in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)Immune Thrombocytopenic Purpura
COMPLETED74 Analytics
NCT02077192Open Label Study of R788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)Immune Thrombocytopenic Purpura
COMPLETED123 Analytics
NCT02076399A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)Immune Thrombocytopenic Purpura
COMPLETED76 Analytics
PHASE3ENROLLING BY_INVITATION
A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
Warm Antibody Autoimmune Hemolytic AnemiaUnlock trial analytics
PHASE3COMPLETED
A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHA
Warm Antibody Autoimmune Hemolytic AnemiaUnlock trial analytics
PHASE3COMPLETED
A Efficacy and Safety Study of Fostamatinib in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
Immune Thrombocytopenic PurpuraUnlock trial analytics
PHASE3COMPLETED
Open Label Study of R788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
Immune Thrombocytopenic PurpuraUnlock trial analytics
PHASE3COMPLETED
A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
Immune Thrombocytopenic PurpuraUnlock trial analytics

Study Endpoints

Primary Endpoints

Adverse Events
104 weeks

Incidence, frequency, seriousness, and severity of adverse events that occurred during the current study

Blood Pressure
104 weeks

Change from baseline in blood pressure over time

Absolute Neutrophil Count (ANC)
104 weeks

Change from baseline in absolute neutrophil count (ANC) over time

Durable Hemoglobin Response
24 Weeks

Proportion of subjects achieving a hemoglobin level ≥ 10 g/dL with an increase from Baseline in hemoglobin level of ≥ 2 g/dL on 3 consecutive available visits during the 24-week treatment period.

Number of Participants With Stable Platelet Response of at Least 50,000/µL
Baseline to Week 24

Stable platelet response by Week 24 defined as a platelet count of at least 50,000/µL on at least 4 of the 6 visits between Weeks 14-24

Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Fostamatinib in 047/048 or 049):Version 1
Up to 12 months

Percentage of subjects who achieved platelet count of at least 50,000/µL within 12 Weeks of beginning treatment up to 12 months was analyzed among all subjects who received active treatment in one of the prior studies (C788-047 or C788-048), in the current extension study (C788-049), or in both prior and current studies. Treated Population was defined as all enrolled and treated subjects.

Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Placebo in 047/048 and Fostamatinib 049): Version 2
Up to 12 months

A within-subject, between-study comparison of platelet counts for subjects who were previously treated with placebo in one of the prior studies (C788-047 or C788-048) was prospectively defined in the protocol (version 2). Achievement of platelet response by 12 weeks and maintenance of platelet response for 12 weeks was analyzed among subjects who had been randomized to placebo in one of the prior studies (C788-047 or C788-048). Treated Population was defined as all enrolled and treated subjects.

Number of Participants With Stable Platelet Response (Count of ≥50,000/µL on at Least 4 of the Last 6 Scheduled Visits Between Weeks 14 and 24)
From Week 14 to Week 24

A stable platelet response by Week 24 defined as a platelet count of at least 50,000/μL on at least 4 of the last 6 scheduled visits between Weeks 14 and 24

The Primary Efficacy Endpoint for This Study is Overall Regressive Response Rate (ORRR): Proportion of Patients With a Best Response of Complete Response (CR), Partial Response (PR), or Regressive Stable Disease (RSD).
Serial tumor assessments were taken at baseline (within 28 days of the start of treatment), and re-evaluated at Day 57, and every 8weeks therafter or to confirm response . (Maximum duration of treatment 182 days, Maximum duration of follow-up 217 days)

Overall regressive response rate (ORRR) is the proportion of patients with a best response of Complete Response (CR), Partial Response (PR) (per Cheson 2007), or Regressive Stable Disease (RSD) defined as regressive disease that does not meet the criteria for partial response.

American College of Rheumatology 20 (ACR20) Response at 3 Months
3 months

The number of participants with greater than or equal to 20% improvement in tender and swollen joint counts, AND in any 3 of the following: physician's assessment of disease activity, patient's assessment of disease activity, patient's assessment of pain, Health Assessment Questionnaire-Disability Index (HAQ-DI); and CRP or ESR, after 3 months

American College of Rheumatology 20 (ACR20) Response at 6 Months
6 months

The number of participants with greater than or equal to 20% improvement in tender and swollen joint counts, AND in any 3 of the following: physician's assessment of disease activity, patient's assessment of disease activity, patient's assessment of pain, HAQ-DI; and C-Reactive Protein (CRP) or erythrocyte sedimentation rate (ESR), after 6 months

Secondary Endpoints

Achievement of Durable Hemoglobin Response
24 weeks
Total Duration of Response
During the Intervention period up to 104 weeks
Corticosteroid dose
During the Intervention period up to 104 weeks
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
FostamatinibEXPERIMENTALSubjects 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.
PlaceboPLACEBO_COMPARATORInitial dose is 100 mg by mouth (PO) twice a day (bid). At week 4 dose will be increased to placebo 150 mg PO bid if subjects have adequately tolerated the study drug in the opinion of the Investigator.
Fostamatinib DisodiumEXPERIMENTALFostamatinib Disodium tablet 100 mg or 150 mg PO bid (morning and evening) over the course of 24 weeks.
1EXPERIMENTAL -
Arm 1EXPERIMENTALFostamatinib disodium (R935788) 100 mg tablet, orally, twice-a-day
Arm 2PLACEBO_COMPARATORPlacebo, orally, twice-a-day
2EXPERIMENTALR788, 150 mg tablet, orally, once a day
3PLACEBO_COMPARATORPlacebo, orally, either once a day, or twice a day

Interventions

NameTypeDescription
Fostamatinib disodiumDRUGFostamatinib is supplied in two (2) dosage strengths: 100 mg and 150 mg.
PlaceboDRUGPlacebo
Fostamatinib disodium (R935788)DRUGR935788 100 mg tablet, orally, twice-a-day
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Eligibility Criteria

Age Range18 Years to 100 Years
SexALL
Healthy VolunteersNo
Study Sites37

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. Exclusion Criteria: ...

Countries:United StatesAustraliaAustriaBelarusBelgiumBulgariaCzechiaFranceGeorgiaGermanyItalyNetherlandsNorwayRussiaSerbiaSpainUkraineUnited KingdomCanadaDenmarkHungaryRomaniaPolandColombiaPeruMexico
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Frequently asked questions about Fostamatinib disodium

What is Fostamatinib Disodium used for?

Fostamatinib Disodium is an investigational small molecule being studied for immune thrombocytopenic purpura, rheumatoid arthritis, T cell lymphoma, and warm antibody autoimmune hemolytic anemia. It is developed by Rigel Pharmaceuticals, Inc. (RIGL) and is currently in Phase 2 clinical development.

What does Fostamatinib Disodium target?

Fostamatinib Disodium is a kinase inhibitor, belonging to the -tinib class of drugs. It works by inhibiting spleen tyrosine kinase (Syk), which plays a role in immune signaling. This mechanism is being investigated for its potential effects in autoimmune and hematologic conditions.

Who makes Fostamatinib Disodium?

Fostamatinib Disodium is developed by Rigel Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol RIGL. The company is conducting clinical trials to evaluate the drug's safety and efficacy in several indications.

What phase is Fostamatinib Disodium in?

Fostamatinib Disodium is in Phase 2 clinical development. While some completed trials were Phase 3, the overall development program is currently at Phase 2 stage. It is investigational and not yet approved by regulatory authorities.

What clinical trials is Fostamatinib Disodium in?

Fostamatinib Disodium has been studied in several trials. NCT00798096 was a Phase 2 study in T cell lymphoma. NCT02076399 was a Phase 3 trial in immune thrombocytopenic purpura. NCT03764618 and NCT04138927 are Phase 3 studies in warm antibody autoimmune hemolytic anemia, with the latter currently enrolling by invitation.

Is Fostamatinib Disodium the same as R935788?

Yes, Fostamatinib Disodium is also known as R935788. In clinical trials, it has been referred to by this alternative name, such as in the study NCT02076399, which evaluated R935788 in the treatment of persistent or chronic immune thrombocytopenic purpura.