Sirolimus for Autoimmune Disease of Blood Cells

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00392951
• Date of registration: 24/10/2006
• Prospective Registration: Yes
• Primary sponsor: Children's Hospital of Philadelphia
• Public title: Sirolimus for Autoimmune Disease of Blood Cells
• Scientific title: Sirolimus for Patients With Chronic and/or Refractory Autoimmune Cytopenias: A Pilot Series
• Date of first enrolment: December 2006
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00392951
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: David T. Teachey, MD
• Affiliation: Children's Hospital of Philadelphia

Key inclusion & exclusion 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)



Age minimum: 1 Year
Age maximum: 30 Years
Gender: All

Health Condition(s) or Problem(s) studied

Lupus Erythematosus, Systemic

Idiopathic Thrombocytopenic Purpura

Inflammatory Bowel Disease

Evans Syndrome

Anemia, Hemolytic, Autoimmune

Rheumatoid Arthritis

Autoimmune Neutropenia

Autoimmune Lymphoproliferative Syndrome (ALPS)

Autoimmune Pancytopenia

Intervention(s)

Drug: sirolimus

Primary Outcome(s)

Number of Participants With Grade 3 and 4 Toxicities of Administration of Oral Sirolimus [Time Frame: 6 months]

Secondary Outcome(s)

Trough Levels Produced by Administration of Oral Sirolimus [Time Frame: Within first 5 days of starting sirolimus]

Effect of Sirolimus on Intracellular Targets [Time Frame: 6 months]

Number of Participants With Autoimmune Disease Response to Oral Sirolimus [Time Frame: 6 months]

Number of Participants With Lymphoproliferation Response to Oral Sirolimus [Time Frame: 6 months]

Secondary ID(s)

2006-7-4873

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 06/12/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00392951