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Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia

NCT00319878 · Status: UNKNOWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 52

Last updated 2008-10-07

No results posted yet for this study

Summary

Aplastic anemia is a rare autoimmune disorder in which the bone marrow production of blood cells is greatly decreased or absent. Symptoms include fatigue, weakness, tiny reddish-purple marks on the skin, abnormal bruising, and bleeding from the gums, nose, or intestine. While some cases of aplastic anemia are caused by medications, toxic exposures, or inherited genes, most often the cause remains unknown. The purpose of this study is to determine the safety and efficacy of combining two drugs, sirolimus and cyclosporine, for treating individuals with aplastic anemia that has not responded to other treatments.

Conditions

  • Anemia, Aplastic

Interventions

DRUG

Sirolimus

Oral loading dose followed by a once daily dose: * Cohort 1: Loading Dose - 1.2 mg; Daily Dose - 0.4 mg * Cohort 2: % Dose Increase - 100%; Loading Dose - 2.4 mg; Daily Dose - 0.8 mg * Cohort 3: % Dose Increase - 67%; Loading Dose - 3.9 mg; Daily Dose - 1.3 mg * Cohort 4: % Dose Increase - 50%; Loading Dose - 6.0 mg; Daily Dose - 2.0 mg

DRUG

Cyclosporine

Dose of 5 mg/kg divided as a twice daily oral dose

Sponsors & Collaborators

  • Rare Diseases Clinical Research Network

    collaborator NETWORK

  • Office of Rare Diseases (ORD)

    lead NIH

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2006-05-31
Primary Completion
2009-07-31
Completion
2009-12-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00319878 on ClinicalTrials.gov