Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy

Running, not enrolling · Phase 2

Conditions studied: Severe Aplastic Anemia

In brief

Background: People with severe aplastic anemia (SAA) do not make enough red and white blood cells, and/or platelets. Their body's immune system stops the bone marrow from making these cells. The treatment cyclosporine leads to better blood counts. But when this treatment is stopped, the disease may return in 1 in 3 people. The drug sirolimus may help by suppressing the immune system. Objective: To evaluate and compare the usefulness of sirolimus in preventing aplastic anemia from returning after cyclosporine is stopped, compared with stopping cyclosporine alone. Eligibility: People ages 2 and older with SAA who: Have responded to immunosuppressive therapy that includes cyclosporine, and continue to take cyclosporine Are not taking drugs with hematologic effects Design: Participants will be screened with: Medical history Physical exam Blood and urine tests Bone marrow biopsy: The area above the hipbone will be numbed. A thin needle will remove some bone marrow. Participants will be randomly assigned to a group. All will stop cyclosporine. Group 1 will take sirolimus by mouth at the same time each day for 3 months with close monitoring. Group 2 will not receive the study drug but will be monitored closely. Participants will have clinical tests for the first 3 months: Weekly blood test Monthly fasting blood test For group 1, measurements of sirolimus in the blood every 1 2 weeks Participants will have clinic visits at 3 months, 12 months, and annually for 5 years after the study starts. They may have another visit if their SAA returns. These will include: Blood and urine tests Bone marrow biopsy

Key facts

Study ID

NCT02979873

Run by

National Heart, Lung, and Blood Institute (NHLBI)

People needed

84

Starts

2016-12-19

Expected to finish

2030-06-30

Last updated by the study team

2026-01-16

Who can join

Age: 2 and older, up to 99. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Evidence of relapse of aplastic anemia due to cyclosporine withdrawal during the previous 6 months
• Prior use of sirolimus or other mTOR inhibitor within 12 weeks of study entry
• Myelodysplastic syndrome or acute myeloid leukemia, according to WHO diagnostic criteria (if baseline BM consistent with MDS after enrollment, patients will be considered ineligible and immediately exit the study, and the subject can be replaced with another subject)
• Patients that are on CYP3A4 inhibitors and cannot replace these medications with other equivalent medications for the period of study: protease inhibitors (ritonavir, indinavir, nelfinavir, saquinavir), some macrolide antibiotics (clarithromycin, telithromycin, erythromycin), azole anti-fungals (fluconazole, itraconazole, ketoconazole), metroclopramide, felodipine, nifedipine, carbamazepine, phenobarbital, grapefruit juice and St. John s Wort.
• Anaphylactic or hypersensitivity reaction to sirolimus
• Patients with infections not adequately responding to appropriate therapy as evidenced by persistence of a clear source of infection that, in the view of the investigator, would preclude safe treatment with sirolimus.
• Current pregnancy, or unwillingness to take oral contraceptives or use the barrier methods of birth control or practice abstinence to refrain from pregnancy if of childbearing potential during the course of the study 8. Lactating women, due to the potentially harmful effects on the nursing child.
• Patients who have received live vaccines within the past 30 days
• Patients with cancer who are actively receiving chemotherapeutic treatment or who take drugs with hematological effects such as thrombopoietin receptor agonists (such as eltrombopag), granulocyte-colony stimulating factor or erythroid stimulating agents.
• Moribund status such that death within 7 to 10 days is likely. Comorbidities of such severity that in the view of the Investigator it would likely preclude the patient's ability to tolerate sirolimus.
• Inability to understand the investigational nature of the study or to give informed consent or without a legally authorized representative or surrogate that can provide informed consent.

Where it is running

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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