Sirolimus for Autoimmune Disease of Blood Cells

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

30

Participants

Timeline

Start Date

December 31, 2006

Primary Completion Date

February 29, 2016

Study Completion Date

February 29, 2016

Conditions
Autoimmune PancytopeniaAutoimmune Lymphoproliferative Syndrome (ALPS)Evans SyndromeIdiopathic Thrombocytopenic PurpuraAnemia, Hemolytic, AutoimmuneAutoimmune NeutropeniaLupus Erythematosus, SystemicInflammatory Bowel DiseaseRheumatoid Arthritis
Interventions
DRUG

sirolimus

Tablet or liquid; taken once or twice daily; dosage is based on establishing a serum trough of 5-15 ng/ml by high-performance liquid chromatography (initial loading dose of 3 mg/m2 then 2.5 mg/m2 with adjustment based on serum trough)

Trial Locations (1)

19104

Children's Hospital of Philadelphia, Philadelphia

All Listed Sponsors
lead

Children's Hospital of Philadelphia

OTHER

NCT00392951 - Sirolimus for Autoimmune Disease of Blood Cells | Biotech Hunter | Biotech Hunter