Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Chronic Lymphocytic Leukaemia (CLL) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Chronic Lymphocytic Leukaemia (CLL). ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Chronic Lymphocytic Leukaemia (CLL): 1 is active or not yet recruiting; 6 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT01231412). The largest enrolment is 260 participants (NCT00544115). Registration activity spans 2001 to 2014. No published literature item is linked to Sirolimus and Chronic Lymphocytic Leukaemia (CLL) in the OSMF database yet, so the record rests on registry entries alone. Registry entries describe intent to study, not outcomes.
The RepurpOS disease-intelligence file for Chronic Lymphocytic Leukemia ranks 50 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: Early-stage (Rai 0-I): watch-and-wait appropriate in 50-70%; spontaneous remission extremely rare; median time to treatment in Rai Stage 0: 10+ years; Richter's transformation (5-10%) extremely poor prognosis.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 45.5 (trials 25.5, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 1 active / not yet recruiting, 6 completed, 1 other |
| Linked publications | 0 |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | ABL1 |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT01231412 | Graft-Versus-Host Disease Prophylaxis in Treating Patients With Hematologic Malignancies Undergoing Unrelated Donor Peripheral Blood Stem Cell Transplant 2010 | completed | Phase 3 | 174 |
| NCT00544115 | Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders 2001 | active, not recruiting | Phase 2 | 260 |
| NCT01244906 | Post-transplant Cyclophosphamide and Sirolimus Following Reduced Intensity Conditioning (RIC) Transplant 2010 | completed | Phase 2 | 26 |
| NCT00589563 | Sirolimus, Tacrolimus, and Antithymocyte Globulin in Preventing Graft-Versus-Host Disease in Patients Undergoing a Donor Stem Cell Transplant For Hematological Cancer 2007 | completed | Phase 2 | 32 |
| NCT00105001 | Tacrolimus and Mycophenolate Mofetil With or Without Sirolimus in Preventing Acute Graft-Versus-Host Disease in Patients Who Are Undergoing Donor Stem Cell Transplant for Hematologic Cancer 2004 | completed | Phase 2 | 210 |
| NCT02226861 | Ultra-Low Dose IL-2 Therapy as GVHD Prophylaxis in Haploidentical Allogeneic Stem Cell Transplantation 2014 | completed | Phase 1 | 24 |
| NCT00671112 | Everolimus and Bortezomib in Treating Patients With Relapsed or Refractory Lymphoma 2008 | terminated | Phase 1 | 30 |
| NCT00918333 | Panobinostat and Everolimus in Treating Patients With Recurrent Multiple Myeloma, Non-Hodgkin Lymphoma, or Hodgkin Lymphoma 2009 | completed | PHASE1, PHASE2 | 124 |
Published literature
No publication is linked to this pair yet.
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL, in this case via the biomarker or target ABL1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Chronic Lymphocytic Leukaemia (CLL)?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Chronic Lymphocytic Leukaemia (CLL) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Chronic Lymphocytic Leukaemia (CLL). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Chronic Lymphocytic Leukaemia (CLL)?
ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Chronic Lymphocytic Leukaemia (CLL): 1 is active or not yet recruiting; 6 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT01231412). The largest enrolment is 260 participants (NCT00544115). Registration activity spans 2001 to 2014.
What does the evidence show for Sirolimus in Chronic Lymphocytic Leukaemia (CLL)?
Sirolimus has 6 completed registered trials for Chronic Lymphocytic Leukaemia (CLL) but no linked publication, which usually means results are unpublished, pending, or not yet matched to this record. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL, in this case via the biomarker or target ABL1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-06. Page built 2026-10-07.