Status:
COMPLETED
Sirolimus for Autoimmune Disease of Blood Cells
Lead Sponsor:
Children's Hospital of Philadelphia
Conditions:
Autoimmune Pancytopenia
Autoimmune Lymphoproliferative Syndrome (ALPS)
Eligibility:
All Genders
1-30 years
Phase:
PHASE1
PHASE2
Brief Summary
Treatment for patients with autoimmune destruction of blood cells is poor. The part of the body that fights infections is called the immune system and white blood cells (WBCs) are part of the immune s...
Detailed Description
Patients with autoimmune destruction of hematopoietic cells frequently have severe and debilitating disease requiring aggressive and frequent medical management. These patients are often treated with ...
Eligibility Criteria
Inclusion
- Age \> 12 months and \< 30 years at the time of study entry
- Diagnosis of autoimmune cytopenias requiring treatment with medications
- At least one of the following: Autoimmune Neutropenia, Autoimmune Hemolytic Anemia, and/or Autoimmune Thrombocytopenia
- Must be proven autoimmune by either a documented autoantibody (positive direct anti globulin test, positive anti-neutrophil, and/or anti-platelet antibody) and/or a documented clinical response to immunosuppression
- Autoimmune Cytopenias can be idiopathic (Idiopathic Thrombocytopenic Purpura (ITP), Autoimmune Hemolytic Anemia (AIHA), Autoimmune Neutropenia (AIN), or Evans syndrome) or secondary to one of following conditions: Lupus, Rheumatoid Arthritis (RA), ALPS (Autoimmune Lymphoproliferative Syndrome), or Inflammatory bowel disease (IBD)
- Patients must have chronic disease diagnosed by either a documented cytopenia syndrome (Lupus, ALPS, RA, or IBD), or by having Evans syndrome defined as idiopathic destruction of multiple blood cell types, and/or by having disease \>6 months
- Patients must be refractory to or unable to tolerate standard front-line therapies for autoimmune cytopenias (corticosteroids and/or IVIG)
- Patients may be taking second-line agents for autoimmune cytopenias (mycophenolate mofetil, cyclosporine, tacrolimus, mercaptopurine, and/or methotrexate) at time of study entry; however, attempts should be made to wean these agents. Patients may not stay on a combination of sirolimus and a calcineurin inhibitor for greater than 4 weeks
- Informed consent/assent must be obtained prior to initiating treatment
- Patient must be able to consume oral medication in the form of tablets or solution
Exclusion
- Pregnancy or breast feeding
- Uncontrolled infection
- Known allergy to Sirolimus or its components
- Patients with a documented malignancy on therapy or not in remission
- Patients who do not meet organ function requirements listed in protocol
- Patients with a documented history of severe combined immunodeficiency or human immunodeficiency virus infection (HIV)
Key Trial Info
Start Date :
December 1 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2016
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT00392951
Start Date
December 1 2006
End Date
February 1 2016
Last Update
November 19 2019
Active Locations (1)
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1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104