NCT02979873

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

Active, enrolled Phase 2 Last updated 16 January 2026

What this trial tests

Phase 2 trial testing Sirolimus in Severe Aplastic Anemia in 84 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

19 December 2016

Primary endpoint
4 September 2025

30 June 2030

Quick facts

Lead sponsor	National Heart, Lung, and Blood Institute (NHLBI)
Phase	Phase 2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	84
Start date	19 December 2016
Primary completion	4 September 2025
Estimated completion	30 June 2030
Sites	1 location across United States

Drugs / interventions tested

• Sirolimus (sirolimus) — full drug profile →

Conditions studied

• Severe Aplastic Anemia — all drugs for Severe Aplastic Anemia →

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Who can join

Adults 2 to 99, any sex, with Severe Aplastic Anemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

1. Long-term outcomes in patients with severe aplastic anemia treated with immunosuppression and eltrombopag: a phase 2 study.
   Patel BA, Groarke EM, Lotter J, Shalhoub R, et al · · 2022 · cited 97× · PMID 34525188 · DOI 10.1182/blood.2021012130
2. Eltrombopag added to immunosuppression for children with treatment-naïve severe aplastic anaemia.
   Groarke EM, Patel BA, Gutierrez-Rodrigues F, Rios O, et al · · 2021 · cited 50× · PMID 33410523 · DOI 10.1111/bjh.17232
3. Activity of eltrombopag in severe aplastic anemia.
   Scheinberg P. · · 2018 · cited 31× · PMID 30425070 · DOI 10.1182/bloodadvances.2018020248
4. Activity of eltrombopag in severe aplastic anemia.
   Scheinberg P. · · 2018 · cited 21× · PMID 30504345 · DOI 10.1182/asheducation-2018.1.450
5. Clinical outcomes and immune responses to SARS-CoV-2 vaccination in severe aplastic anaemia.
   Rajput RV, Ma X, Boswell KL, Gaudinski M, et al · · 2022 · cited 6× · PMID 36128909 · DOI 10.1111/bjh.18456