Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy | Aplastic Anemia and MDS International Foundation (AAMDSIF) Return to top.

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

Trial Sponsor

NIH - National Heart, Lung, and Blood Institute

Contact Info

Ivana Darden

Start Date: December 19, 2016 End Date: August 31, 2023 Identifier: NCT02979873

If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.


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.

Bone Marrow Disease(s): 
Associated Drug(s): 
Phase 2
Age Group: 
2 years and older
Accepts Healthy Volunteers: 
Details on 
Inclusion Criteria: 
  • Age greater than or equal to 2 years old
  • Weight greater than 12 kg
  • Previous diagnosis of SAA by bone marrow biopsy and cytogenics, treated with lymphodepleting therapy ATG, cyclophosphamide or alemtuzumab that included cyclosporine. The lymphodepleting therapy must have been administered at least 12 months prior.
  • Continuous treatment with cyclosporine for the previous 6 months (excluding minor dose delays not exceeding more than 30 days).
  • Evidence of a hematologic response to an lymphodepletion-based regimen as evidence of at least two of the following:
    • Absolute neutrophil count greater than or equal to 500/uL
    • Platelet count greater than or equal to 20,000/uL (without transfusion support)
    • Absolute reticulocyte count greater than or equal to 60,000/uL (or hemoglobin 10 gm/dL without transfusion support)
Exclusion Criteria: 
  1. Evidence of relapse of aplastic anemia due to cyclosporine withdrawal during the previous 6 months
  2. Prior use of sirolimus or other mTOR inhibitor within 12 weeks of study entry
  3. 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)
  4. 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.
  5. Anaphylactic or hypersensitivity reaction to sirolimus
  6. 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.
  7. 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.
  8. Patients who have received live vaccines within the past 30 days
  9. 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.
  10. 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.
  11. 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.

Find Out More:


AAMDSIF does not recommend, endorse, or make any representation about the efficacy, appropriateness or suitability of any clinical trial listed on this website. Pharmaceutical company sponsored content is highlighted only to give additional information about the trial. All trials are listed on Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding a clinical trial, and never disregard professional medical advice or delay in seeking it because of something you have read on this website.