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NCT00392951 · ClinicalTrials.gov registry record · Phase 1
Sirolimus for Autoimmune Disease of Blood Cells
A Phase 1 study, sponsored by Children's Hospital of Philadelphia.
- Completed
- Registry status
- Phase 1
- Development phase
- 30
- Enrollment target
NCT00392951 is a Phase 1 study that has completed, run by Children's Hospital of Philadelphia. The registered enrollment target is 30 participants.
The verdict
NCT00392951, a Phase 1 study, has completed, sponsored by Children's Hospital of Philadelphia.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 30 participants
- Enrollment target
Study 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 system. Normally, a person's body creates WBCs to fight infections and eliminates WBCs which have stopped helping the body function. Patients with autoimmune destruction of blood cells have difficulty eliminating old WBCs. The abnormal WBCs build up and can damage other healthy cells, which can lead to anemia, fatigue, jaundice, internal bleeding, infection, and cancer. Few effective medications exist for treatment for patients with autoimmune cytopenias and those commonly used are fraught with side effects. Nevertheless, as scientific understanding of autoimmune diseases has improved, more directed and less toxic therapies are becoming available. A number of groups have been studying the efficacy of a medication called sirolimus in patients with autoimmune diseases. This medicine has been FDA-approved for over 20 years. Sirolimus is a medicine used in children with other diseases. Sirolimus works, in part, by eliminating old and abnormal WBCs. Our group and others have shown that sirolimus is effective in mice with autoimmunity and in children with a rare condition called Autoimmune Lymphoproliferative Syndrome (ALPS). We believe sirolimus will help children with autoimmune cytopenias. We believe it will improve their symptoms and make them less sick. We propose to study sirolimus in children with chronic and/or refractory autoimmune cytopenias.
Interventions
- DRUG sirolimus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2006-12 |
| Est. Completion | 2016-02 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00392951
The ClinicalTrials.gov registry entry for NCT00392951 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 30 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Children's Hospital of Philadelphia, which has 431 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which sirolimus is the first listed.
NCT00392951 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00392951 about?
NCT00392951 is a clinical study titled "Sirolimus for Autoimmune Disease of Blood Cells". 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 system. Normally, a person's body creates WBCs to fight infections and eliminates WBCs which have sto...
What is the current status of trial NCT00392951?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 30 participants. The study started on 2006-12. Estimated completion is 2016-02.
What interventions are being tested in trial NCT00392951?
The interventions under investigation include: sirolimus (DRUG).
Who is sponsoring clinical trial NCT00392951?
This trial is sponsored by Children's Hospital of Philadelphia, which has 431 total clinical trials registered on ClinicalTrials.gov.
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