Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 11:50 PM
Ignite Modification Date:
2025-12-24 @ 11:50 PM
NCT ID:
NCT00392951
Eligibility Criteria:
Inclusion Criteria:
* 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 Criteria:
* 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)
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
1 Year
Maximum Age:
30 Years
Study:
NCT00392951
Study Brief:
Protocol Section:
NCT00392951