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Immunosuppressant

1 for Aplastic Anemia

Phase 1 & 2
Waitlist Available
Research Sponsored by Office of Rare Diseases (ORD)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up measured at months 3 and 6
Awards & highlights

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.

Eligible Conditions
  • Aplastic Anemia

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~measured at months 3 and 6
This trial's timeline: 3 weeks for screening, Varies for treatment, and measured at months 3 and 6 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Safety and tolerability of sirolimus and cyclosporine in each stratum of participants
Secondary outcome measures
Duration of hematologic response
Clonal Evolution
Response rate
+1 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: 1Experimental Treatment2 Interventions
Participants will be treated with sirolimus and cyclosporine. In phase I, each dose cohort will initially enroll three patients. If no dose-limiting toxicity (DLT) is observed by Day 28 in any patient of a cohort, then 3 patients will be treated with the next highest sirolimus dose. If 1 out of 3 patients in any cohort experiences a DLT, then 3 more patients will be enrolled in that cohort. If no more patients have a DLT by Day 28, then sirolimus dose escalation will proceed. If one or more patients experience a DLT then that dose level will be considered to be the maximum tolerated sirolimus dose, and Phase II patients will be treated at the next lowest level. Cyclosporine will be given as a twice daily oral dose.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sirolimus
FDA approved
Cyclosporine
FDA approved

Find a Location

Who is running the clinical trial?

Rare Diseases Clinical Research NetworkNETWORK
67 Previous Clinical Trials
19,053 Total Patients Enrolled
Office of Rare Diseases (ORD)Lead Sponsor
43 Previous Clinical Trials
12,277 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia
2006. "Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT00319878.
All > Aplastic Anemia > Restasis
~3 spots leftby Apr 2025
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