Clinical Trials

Numerous Phase 1 through Phase 3 clinical trials, sponsored by pharmaceutical companies such as Rigel Pharmaceuticals and AstraZeneca alongside academic institutions and government agencies like the National Heart, Lung, and Blood Institute, have evaluated or are currently evaluating fostamatinib. Investigated indications include primary immune thrombocytopenia, immune-mediated anemia, chronic graft-versus-host disease, sickle cell disease, myelofibrosis, lung transplantation rejection, and severe viral pneumonias such as COVID-19. Trial statuses across these safety and efficacy evaluations range from active recruitment and enrollment by invitation to completed and terminated studies.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06233110 RECRUITING
Chronic Graft Versus Host Disease
Stefanie Sarantopoulos, MD, PhD.
 2025-10-29 PHASE1
NCT06948097 RECRUITING
Lung Transplantation
National Heart, Lung, and Blood Institute (NHLBI)
 2026-09-09 PHASE1
NCT06639724 RECRUITING
Pancreatic Ductal Adenocarcinoma (PDAC)
University of California, San Diego
 2024-12-05 PHASE1
NCT06564207 NOT_YET_RECRUITING
Acute Respiratory Distress Syndrome; ARDS
Inova Health Care Services
 2026-08-30 PHASE2
NCT05904093 RECRUITING
Sickle Cell Disease; Hb-SS Disease; Hemoglobin S; Disease Sickle Cell Anemia; Sickle Cell Disorders; Hemoglobin Beta Thalassemia Disease
National Heart, Lung, and Blood Institute (NHLBI)
 2024-12-18 PHASE1
NCT05509582 WITHDRAWN
Immune Mediated Anemia; Immune Mediated Thrombocytopenia; Chronic GVHD
National Heart, Lung, and Blood Institute (NHLBI)
 2025-11-07 PHASE2
NCT05030675 COMPLETED
Refractory Chronic Myelomonocytic Leukemia; Refractory Myelodysplastic Syndrome
M.D. Anderson Cancer Center
 2021-08-13 PHASE1
NCT05904093 Not yet recruiting
Sickle Cell Disease|Hb-SS Disease|Hemoglobin S|Disease Sickle Cell Anemia|Sickle Cell Disorders|Hemoglobin Beta Thalassemia Disease
National Heart Lung and Blood Institute (NHLBI)|National Institutes of Health Clinical Center (CC)
 2024-05-15 Phase 1
NCT05509582 Enrolling by invitation
Immune Mediated Anemia|Immune Mediated Thrombocytopenia|Chronic GVHD
National Heart Lung and Blood Institute (NHLBI)|National Institutes of Health Clinical Center (CC)
 2024-05-15 Phase 2
NCT04138927 ENROLLING_BY_INVITATION
Warm Antibody Autoimmune Hemolytic Anemia
Rigel Pharmaceuticals
 2019-10-30 PHASE3
NCT05593770 TERMINATED
COVID-19; SARS-CoV2 Infection; Coronavirus Infection
NEAT ID Foundation
 2022-10-27 PHASE2; PHASE3
NCT04924660 COMPLETED
COVID-19; SARS-CoV-2 Infection; Coronavirus Infection
Sean Collins
 2021-07-15 PHASE2; PHASE3
NCT05502783 TERMINATED
Immune Mediated Anemia; Immune Mediated Thrombocytopenia; Chronic GVHD
National Heart, Lung, and Blood Institute (NHLBI)
 2023-03-10 PHASE2
NCT03991780 ACTIVE_NOT_RECRUITING
Renal Transplant Rejection
Imperial College London
 2019-05-08 PHASE1; PHASE2
NCT06071520 Completed
Primary Immune Thrombocytopenia
Fundación Pública Andaluza para la gestión de la Investigación en Sevilla
 2023-03-01 --
NCT05613296 Not yet recruiting
ITP - Immune Thrombocytopenia|Chronic ITP|Refractory ITP
Gruppo Italiano Malattie EMatologiche dell''Adulto
 2023-02 --
NCT05040698 COMPLETED
Hidradenitis Suppurativa
Holdsworth House Medical Practice
 2021-10-01 PHASE2
NCT04581954 COMPLETED
Coronavirus; Covid19; Pneumonia
Imperial College London
 2020-10-02 PHASE1; PHASE2
NCT04629703 COMPLETED
Covid19; SARS (Severe Acute Respiratory Syndrome); SARS Pneumonia; SARS-Associated Coronavirus as Cause of Disease Classified Elsewhere; Pneumonia; Pneumonia, Viral
Rigel Pharmaceuticals
 2021-03-25 PHASE3
NCT03246074 COMPLETED
Ovarian Cancer
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
 2018-04-03 PHASE1
NCT04543279 TERMINATED
Myelofibrosis; Thrombocytopenia
Washington University School of Medicine
 2021-05-03 PHASE2
NCT03764618 COMPLETED
Warm Antibody Autoimmune Hemolytic Anemia
Rigel Pharmaceuticals
 2019-04-24 PHASE3
NCT04132050 COMPLETED
Idiopathic Thrombocytopenic Purpura
Kissei Pharmaceutical Co., Ltd.
 2019-12-24 PHASE3
NCT04543279 Terminated
Myelofibrosis|Thrombocytopenia
Washington University School of Medicine|Rigel Pharmaceuticals
 2021-05-03 Phase 2
NCT04904276 Terminated
ITP|Immune Thrombocytopenia
Rigel Pharmaceuticals
 2021-05-18 --
NCT04579393 COMPLETED
Coronavirus Disease 2019
National Heart, Lung, and Blood Institute (NHLBI)
 2020-10-08 PHASE2
NCT02611063 COMPLETED
Hematological Malignancies
Stefanie Sarantopoulos, MD, PhD.
 2016-01 PHASE1
NCT02077192 COMPLETED
Immune Thrombocytopenic Purpura
Rigel Pharmaceuticals
 2014-10 PHASE3
NCT02612558 COMPLETED
Warm Antibody Autoimmune Hemolytic Anemia
Rigel Pharmaceuticals
 2016-07 PHASE2
NCT02112838 COMPLETED
IGA Nephropathy
Rigel Pharmaceuticals
 2014-10 PHASE2
NCT02076412 COMPLETED
Immune Thrombocytopenic Purpura
Rigel Pharmaceuticals
 2015-01 PHASE3
NCT02076399 COMPLETED
Immune Thrombocytopenic Purpura
Rigel Pharmaceuticals
 2014-07-14 PHASE3
NCT02433236 WITHDRAWN
IGA Nephropathy
Rigel Pharmaceuticals
 2015-09 PHASE2
NCT01242514 TERMINATED
Rheumatoid Arthritis
AstraZeneca
 2011-01 PHASE3
NCT01499303 COMPLETED
Diffuse Large B-Cell Lymphoma
AstraZeneca
 2011-12 PHASE2
NCT00805467 TERMINATED
Rheumatoid Arthritis
AstraZeneca
 2008-08 PHASE2
NCT01569074 TERMINATED
Rheumatoid Arthritis
AstraZeneca
 2012-04 PHASE2
NCT01640054 TERMINATED
Rheumatoid Arthritis
AstraZeneca
 2012-07 PHASE2
NCT02092961 TERMINATED
Rheumatoid Arthritis
AstraZeneca
 2011-02 PHASE2
NCT01645085 COMPLETED
Healthy Volunteers
AstraZeneca
 2012-07 PHASE1
NCT01197534 COMPLETED
Rheumatoid Arthritis
AstraZeneca
 2010-09 PHASE3
NCT01197755 COMPLETED
Rheumatoid Arthritis
AstraZeneca
 2010-09 PHASE3
NCT01563978 COMPLETED
Rheumatoid Arthritis
AstraZeneca
 2012-04 PHASE2
NCT01725230 COMPLETED
Rheumatoid Arthritis
AstraZeneca
 2012-11 PHASE1
NCT01682408 COMPLETED
Pharmacokinetics
AstraZeneca
 2012-09 PHASE1
NCT01197521 COMPLETED
Rheumatoid Arthritis
AstraZeneca
 2010-09 PHASE3
NCT01264770 TERMINATED
Rheumatoid Arthritis
AstraZeneca
 2011-01 PHASE2
NCT01598571 COMPLETED
Healthy
AstraZeneca
 2012-05 PHASE1
NCT01608542 COMPLETED
Healthy Japanese Volunteers
AstraZeneca
 2012-06 PHASE1
NCT01276262 COMPLETED
Scientific Terminology Rheumatoid Arthritis, Healthy Female Volunteers, Pharmacokinetics, Oral Contraceptive, Drug-drug Interaction; Laymen Terminology Level of Oral Contraceptive in Blood, Oral Contraceptive, Rheumatoid Arthritis, Drug -Drug Interaction
AstraZeneca
 2011-03 PHASE1
NCT01387308 COMPLETED
Healthy
AstraZeneca
 2011-08 PHASE1
NCT01355354 COMPLETED
Healthy Volunteers; Rheumatoid Arthritis
AstraZeneca
 2011-06 PHASE1
NCT01336218 COMPLETED
Rheumatoid Arthritis; Healthy Volunteers
AstraZeneca
 2011-04 PHASE1
NCT01245790 COMPLETED
Rheumatoid Arthritis; Renal Impairment
AstraZeneca
 2010-11 PHASE1
NCT01222455 COMPLETED
Hepatic Impairment; Healthy Volunteers; Pharmacokinetics; Amount of R406 in Blood
AstraZeneca
 2010-10 PHASE1
NCT01167868 COMPLETED
Healthy
AstraZeneca
 2010-07 PHASE1
NCT01311622 COMPLETED
Rheumatoid Arthritis; Healthy Subjects
AstraZeneca
 2011-03 PHASE1
NCT01309854 COMPLETED
Rheumatoid Arthritis; Healthy Volunteers; Pharmacokinetics; Pioglitazone; Drug-drug Interaction; Amount of Pioglitazone in Blood
AstraZeneca
 2011-03 PHASE1
NCT01208155 COMPLETED
Bioavailability; Pharmacokinetics
AstraZeneca
 2010-09 PHASE1
NCT01197781 COMPLETED
Drug Drug Interactions; Healthy Volunteers
AstraZeneca
 2010-09 PHASE1
NCT00446095 COMPLETED
Lymphoma
Rigel Pharmaceuticals
 2007-04 PHASE1; PHASE2
NCT00798096 COMPLETED
T Cell Lymphoma
Rigel Pharmaceuticals
 2009-03 PHASE2
NCT00706342 COMPLETED
Purpura, Thrombocytopenic, Idiopathic
Rigel Pharmaceuticals
 2007-01 PHASE2
NCT00752999 WITHDRAWN
Systemic Lupus Erythematosus
Rigel Pharmaceuticals
 2008-11 PHASE2
NCT00665925 COMPLETED
Rheumatoid Arthritis
Rigel Pharmaceuticals
 2008-05 PHASE2
NCT00665626 COMPLETED
Rheumatoid Arthritis
Rigel Pharmaceuticals
 2008-05 PHASE2
NCT00923481 COMPLETED
Head and Neck Neoplasms; Pheochromocytoma; Colorectal Neoplasms; Carcinoma, Non-Small-Cell Lung; Carcinoma, Renal Cell
National Cancer Institute (NCI)
 2009-04 PHASE2
NCT00326339 COMPLETED
Rheumatoid Arthritis
Rigel Pharmaceuticals
 2006-08 PHASE2

(data from https://clinicaltrials.gov, updated on 2026-01-22)

Check the Fostamatinib (R788) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Fostamatinib, an oral prodrug converted to its active metabolite R406, selectively inhibits spleen tyrosine kinase (Syk), thereby blocking immunoreceptor signaling through IgG Fc gamma receptors and B-cell receptors to impair the downstream activation of mast cells, macrophages, and B lymphocytes. This suppression of inflammatory signaling and immune cell activation reduces tissue damage and pathologic autoimmune destruction, providing therapeutic relevance in clinical trial conditions such as immune thrombocytopenia, chronic graft-versus-host disease, and severe inflammatory respiratory disease.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.