RecruitingPhase 1interventionalRules rewrite

Phase 1: testing Fostamatinib for Sickle Cell Disorders

Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ("sickled") red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves.

Official registry: https://clinicaltrials.gov/study/NCT05904093 · National Heart, Lung, and Blood Institute (NHLBI) · enrollment listed as 35

What this phase means

Phase 1: the first tests in people. The main question is safety and dose, not whether it cures the condition. Groups are small. Side effects are watched closely.

Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

What the registry says happens

If you join, the listed intervention(s) are Fostamatinib. The registry lists allocation as na. Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ("sickled") red blood cells.

They plan to enroll about 35 people at Bethesda. Taking part typically means taking a study drug (or dummy pill) plus check-ins.

Official title (unrewritten)

A Phase I Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

Interventions: Fostamatinib

Primary outcome (registry wording): Safety and tolerability of fostamatinib

Who it is for (from inclusion criteria)

Ages 18 Years to 65 Years
Age between 18-65 years
Unequivocal diagnosis of SCA (HbSS or HbSBeta\^0) confirmed by hemoglobin electrophoresis performed on patients at least 60 days after a blood transfusion if previously transfused
No transfusion in the 60 days prior to signing consent, or absence of Hb A on hemoglobin analysis (by high-performance liquid chromatography; HPLC)
Have adequate organ function, as defined by:
Serum aspartate aminotransferase (AST) \<=1.5 x Upper Limit of Normal (ULN) (unless the increased AST is assessed by the Investigator as due to hemolysis) and alanine aminotransferase (ALT) \<=1.5 x ULN
Absolute neutrophil count \>=1.5 x 10\^9/L
Hemoglobin \>= 7 g/dL

Who the registry excludes

An individual who meets any of the following criteria will be excluded from participation in this study:
Pain crisis requiring parenteral treatment within 14 days of signing consent
Have a significant medical condition that confers an unacceptable risk to participating in the study, and/or that could confound the interpretation of the study data. Such significant medical conditions include, but are not limited to the following:
History of neutropenia (benign ethnic neutropenia and/or acquired neutropenia related to drug suppression by hydroxyurea and/or cyclic hematopoiesis are permitted)
History of posterior reversible encephalopathy syndrome (PRES)
History of drug-induced cholestatic hepatitis
History of any primary malignancy

Public study contact (from the registry)

Jordan B Branch

(301) 221-3820

jordan.branch@nih.gov

Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.

Sites listed on ClinicalTrials.gov

1 listed

National Institutes of Health Clinical Center — Bethesda, Maryland (recruiting)

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FAQ about this NCT record

Is NCT05904093 still recruiting?

The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.

What phase is NCT05904093?

Phase 1. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

Can PlainTrials tell me if I qualify for NCT05904093?

No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.

How to read a ClinicalTrials.gov record · Questions to ask the coordinator · Glossary