Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication TAVALISSE is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. TAVALISSE is a kinase inhibitor indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. FDA label
Also known as tamatinib fosdium, Fostamatinib disodium, R935788, Fostamatinib, Tavalisse, Fostamatinib Disodium (R935788)
R788 · 17 trials · 17 indications
Incidence, frequency, seriousness, and severity of adverse events that occurred during the current study
Change from baseline in blood pressure over time
Change from baseline in absolute neutrophil count (ANC) over time
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.
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 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.
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.
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 cumulative incidence of serious adverse events (SAEs) occurring through day 30 following the intervention. An SAE is defined by the International Conference on Harmonization (ICH) guidelines as any adverse event fulfilling at least one of the following criteria: Results in death; Is life threatening; Requires in-subject hospitalization or prolongation of an existing hospitalization; Results in persistent or significant disability/incapacity; Is considered an important medical event (or medically significant)
Alterations in Gene Expression Profiling, cell counts (CD3+, CD11c+, Neutrophil Elastase+, CD20+, CD138+) at Week 4 compared to
Alterations in Gene Expression Profiling, cell counts (CD3+, CD11c+, Neutrophil Elastase+, CD20+, CD138+) at Week 12 compared to Baseline
Hemoglobin level of \> 10 g/dL and 2 g/dL higher than the baseline hemoglobin
Mean change from Baseline (Visit 2) of proteinuria as measured by the spot Protein-Creatinine Ratio (sPCR) at 24 weeks (Visit 9) for the ITT Population
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.
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
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
Number and percentage of participants who experience surgical delay, as measured by the proportion of enrolled participants for whom pancreatic resection cannot be performed within 6 weeks of the last pre-operative treatment cycle.
Proportion of patients with Complete Response (CR) or Partial Response (PR). Revised Response Criteria for Malignant Lymphoma categorises the response of the treatment of a patient's tumour to; CR: the disappearance of all evidence of disease; PR: ≥ 50% decrease in the sum of the perpendicular diameters (SPD) of the six largest dominant nodes plus no increase in the size of other nodes and no new sites of disease; Stable Disease (SD): less than a PR but not progressive disease; Relapsed Disease or PD: Any new lesion or increase by ≥ 50% of previously involved sites from nadir. Primary efficacy is based on Phase II patients only.
Proportion of patients with Complete Response (CR), Partial Response (PR), or Stable Disease (SD)
| Arm | Type | Description |
|---|---|---|
| Fostamatinib (150 mg twice daily for 14 days) + Standard of Care | ACTIVE_COMPARATOR | Fostamatinib (150 mg twice daily for 14 days) + Standard of Care |
| Placebo (twice daily for 14 days) + Standard of Care | PLACEBO_COMPARATOR | Placebo (twice daily for 14 days) + Standard of Care |
| Fostamatinib | EXPERIMENTAL | 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. |
| Placebo | PLACEBO_COMPARATOR | Initial 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 Disodium | EXPERIMENTAL | Fostamatinib Disodium tablet 100 mg or 150 mg PO bid (morning and evening) over the course of 24 weeks. |
| Standard of Care (SOC) + Placebo (BID for 14 Days) | PLACEBO_COMPARATOR | Participants randomized to this arm will receive standard of care (SOC) treatment plus placebo given twice daily for 14 days |
| Standard of Care (SOC) + Fostamatinib 150mg (BID for 14 Days) | EXPERIMENTAL | Participants randomized to this arm will receive standard of care (SOC) treatment plus fostamatinib 150mg given twice daily for 14 days |
| Open Label Fostamatinib | EXPERIMENTAL | Open label Fostamatinib 100mg dose adjusted by the Principal Investigator after week 1 |
| Fostamatinib 150 mg | EXPERIMENTAL | Fostamatinib 150 mg bid (morning and evening) over the course of 24 weeks |
| Fostamatinib 100 mg | ACTIVE_COMPARATOR | Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks |
| 1 | EXPERIMENTAL | - |
| Arm 1 | EXPERIMENTAL | Fostamatinib disodium (R935788) 100 mg tablet, orally, twice-a-day |
| Arm 2 | PLACEBO_COMPARATOR | Placebo, orally, twice-a-day |
| 2 | EXPERIMENTAL | R788, 150 mg tablet, orally, once a day |
| 3 | PLACEBO_COMPARATOR | Placebo, orally, either once a day, or twice a day |
| 4 | PLACEBO_COMPARATOR | Placebo PO bid |
| Fostamatinib in combination with gemcitabine/nab-paclitaxel | EXPERIMENTAL | Fostamatinib 100 mg will be taken by the study participants orally twice a day for 7 days prior to, and then during chemotherapy with gemcitabine/nab-paclitaxel. These 2 agents will be administered intravenously on days 1, 8, and 15 of each 28-day cycle. |
| Name | Type | Description |
|---|---|---|
| Fostamatinib | DRUG | Fostamatinib (150 mg twice daily) for 14 days and Standard of Care |
| Placebo | DRUG | Placebo (twice daily) for 14 days and Standard of Care |
| Fostamatinib disodium | DRUG | Fostamatinib is supplied in two (2) dosage strengths: 100 mg and 150 mg. |
| Fostamatinib 150 mg bid | DRUG | Fostamatinib 150 mg bid. The dose of Fostamatinib may be reduced at any time to as low as 100 mg PO once daily (qd) if dose limiting adverse events are observed. |
| Fostamatinib 150 mg | DRUG | Fostamatinib 150 milligram (mg) tablet twice daily by mouth, over the course of 24 weeks |
| Fostamatinib 100 mg | DRUG | Fostamatinib 100 mg tablet twice daily by mouth, over the course of 24 weeks |
| Fostamatinib disodium (R935788) | DRUG | R935788 100 mg tablet, orally, twice-a-day |
| Fostamatinib Disodium / R935788 | DRUG | R935788 tablets |
| R788 | DRUG | R788 50 mg, 100 mg, or 150 mg PO bid |
| Fostamatinib in combination with chemotherapy (gemcitabine and nab-paclitaxel) | COMBINATION_PRODUCT | Fostamatinib is a Syk kinase inhibitor currently FDA-approved for chronic idiopathic thrombocytopenia purpura but it has not been studied in PDAC. The investigators hypothesize that Syk inhibition reprograms macrophages to an immunostimulatory phenotype in the tumor microenvironment. Thus, Syk inhibition with fostamatinib in combination with chemotherapy could improve outcomes for patients with PDAC while having a favorable safety profile. |
Inclusion Criteria: * ≥18 years of age at screening. * The subject or a legally authorized representative has provided written informed consent. * Hospitalized COVID-19 subjects without respiratory failure who are either not receiving any oxygen therapy or are receiving supplemental oxygen via mask...
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R788 is an investigational small molecule being studied for the treatment of rheumatoid arthritis, an inflammatory joint disease. It is being developed by Rigel Pharmaceuticals, Inc. as an oral therapy in the immunology therapeutic area. R788 has been evaluated in a Phase 2 clinical trial in adults with rheumatoid arthritis.
R788 targets SYK, which stands for spleen tyrosine kinase. SYK is a kinase enzyme involved in signaling pathways within immune cells. By inhibiting SYK, R788 is designed to interfere with the signaling that contributes to inflammation in rheumatoid arthritis. R788 is a small molecule kinase inhibitor.
R788 is being developed by Rigel Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker symbol RIGL. Rigel is the company responsible for the clinical development of R788 in rheumatoid arthritis.
R788 is in Phase 2 clinical development for rheumatoid arthritis. It is an investigational drug and has not been approved by the FDA. The Phase 2 program includes one completed trial, and no active trials are currently listed for R788.
R788 has been studied in the Phase 2 trial NCT00326339, titled Treatment of Arthritis With Syk Kinase Inhibition, also known as TASKI-1. The trial enrolled 189 adults with rheumatoid arthritis across the United States and Mexico. It was randomized, double-blind, and placebo-controlled, and it has been completed.