What side effects are associated with this type of intervention?

Common treatments for Sickle Cell Disease (SCD) include hydroxyurea, chronic transfusions, and hematopoietic stem cell transplantation. Hydroxyurea can cause cytopenias, mucocutaneous ulcers, diarrhea, peripheral neuropathy, and potential teratogenicity. Chronic transfusions carry risks of iron overload and alloimmunization. Hematopoietic stem cell transplantation may lead to graft-versus-host disease and infections. Investigational therapies like fostamatinib, a Spleen Tyrosine Kinase (SYK) inhibitor, are being studied for their safety and tolerability, with potential side effects including blood-related issues and other systemic effects.

What prior FDA approvals exist?

Hydroxyurea is the most established FDA-approved treatment for Sickle Cell Disease, known for its efficacy in reducing vaso-occlusive pain and other complications by increasing fetal hemoglobin (Hb F) levels. It is recommended for infants, children, adolescents, and adults with severe SCD genotypes such as homozygous Hb SS and sickle beta0 thalassemia. Other treatments include chronic transfusions and hematopoietic cell transplantation. While Fostamatinib, a Spleen Tyrosine Kinase (SYK) Inhibitor, is being studied for its potential benefits in SCD, it has not yet received FDA approval for this indication.

What other types of research exist?

Promising novel alternatives to Fostamatinib for Sickle Cell Disease patients include Hydroxyurea, which is recommended as the first-line therapy for reducing vaso-occlusive pain and other complications. Additionally, multitargeted kinase inhibitors like regorafenib, cabozantinib, sorafenib, and lenvatinib have shown some activity in advanced cases of related conditions and may be considered for further investigation in SCD.

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Tyrosine Kinase Inhibitor

Fostamatinib for Sickle Cell Disease

Phase 1

Waitlist Available

Led By Arun S Shet, M.D.

Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline (day 0) to end of study (day 70)

Awards & highlights

No Placebo-Only Group

Summary

This trial tests the safety and tolerability of fostamatinib, a drug taken by mouth, in people aged 18 to 65 with stable sickle cell disease. Fostamatinib aims to reduce inflammation and abnormal blood cell behavior by inhibiting a specific protein. Participants will take the drug for several weeks and have frequent check-ups to monitor side effects and effectiveness.

Who is the study for?

Adults aged 18-65 with stable Sickle Cell Anemia (SCA) who haven't had a blood transfusion in the last 12 weeks. Participants must not be pregnant, agree to use two forms of contraception, and have normal organ function. They can't join if they have certain medical conditions like uncontrolled hypertension, active infections, or history of malignancy.

What is being tested?

The trial is testing Fostamatinib's safety and how well people with SCA tolerate different doses. Over 12 weeks, participants will take the drug orally twice daily for up to six weeks and attend clinic visits every two weeks to monitor effects and possibly increase dosage based on tolerance.

What are the potential side effects?

Potential side effects include digestive issues, liver problems indicated by changes in specific blood tests (AST/ALT levels), lowered white blood cell counts which could raise infection risk, anemia symptoms worsening if hemoglobin drops too low, or bleeding complications.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline (day 0) to end of study (day 70)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline (day 0) to end of study (day 70)

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (day 0) to end of study (day 70) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

The number of type, incidence, severity and relationship to study treatment of adverse events and serious adverse events

Secondary study objectives

Number of participants that discontinued fostamatinib due to adverse events following CTCAE.

Side effects data

From 2013 Phase 2 trial • 101 Patients • NCT01499303

24%

BLOOD ALKALINE PHOSPHATASE INCREASED

24%

NEUTROPENIA

24%

THROMBOCYTOPENIA

19%

CONSTIPATION

19%

BACK PAIN

19%

NAUSEA

19%

PYREXIA

19%

COUGH

19%

ASPARTATE AMINOTRANSFERASE INCREASED

19%

FATIGUE

14%

DYSPNOEA

14%

BLOOD CREATININE INCREASED

14%

ALANINE AMINOTRANSFERASE INCREASED

14%

DIARRHOEA

14%

BLOOD BILIRUBIN INCREASED

10%

OEDEMA PERIPHERAL

10%

ABDOMINAL DISTENSION

10%

ANAEMIA

10%

HYPOTENSION

10%

ARTHRALGIA

10%

HEADACHE

10%

NEUTROPHIL COUNT DECREASED

10%

BLOOD UREA INCREASED

10%

ANXIETY

BLOOD LACTATE DEHYDROGENASE INCREASED

ABDOMINAL PAIN

HYPERTENSION

HYPONATRAEMIA

MYALGIA

NEUTROPENIC SEPSIS

WHITE BLOOD CELL COUNT DECREASED

100%

80%

60%

40%

20%

Study treatment Arm

100mg BID

200mg BID

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Fostamatinib in participants with Sickle Cell DiseaseExperimental Treatment1 Intervention

Participants with Sickle Cell Disease will receive Fostamatinib which will be administered orally, at a dose of 100 mg twice a day for 14 days and if tolerated, will be escalated to a dose of 150 mg, taken orally, twice a day for 28 days (total 42 days).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Fostamatinib

2021

Completed Phase 3

~1520

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Sickle Cell Disease (SCD) include hydroxyurea, which increases fetal hemoglobin (Hb F) to reduce red blood cell sickling and vaso-occlusive events, and chronic transfusions, which lower the proportion of sickled cells in the blood. Investigational therapies like Fostamatinib, a Spleen Tyrosine Kinase (SYK) inhibitor, aim to reduce inflammation and vascular occlusion by targeting the endothelium. These treatments are vital for SCD patients as they address the root causes of pain and organ damage, thereby improving quality of life and reducing complications.

Novel therapies targeting the endothelium in sickle cell disease.Advances in the management of sickle cell disease.