Summary
Tacrolimus Anhydrous has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Myelodysplastic Syndromes (MDS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Myelodysplastic Syndromes (MDS). ClinicalTrials.gov lists 8 registered trials linking Tacrolimus Anhydrous to Myelodysplastic Syndromes (MDS): 1 is currently recruiting; 1 is active or not yet recruiting; 3 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT05316701). It plans or enrolled 187 participants. Registration activity spans 2005 to 2022. No published literature item is linked to Tacrolimus Anhydrous and Myelodysplastic Syndromes (MDS) 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 Myelodysplastic Syndrome 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: Essentially zero; progressive cytopaenia and transformation to AML in 30-40% of higher-risk MDS over 2-5 years; median survival low-risk MDS ~5 years, high-risk ~1 year without treatment.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 43.5 (trials 23.5, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 1 recruiting, 1 active / not yet recruiting, 3 completed, 3 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 | BCL2 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT05316701 | Precision-T: A Randomized Study of Orca-T in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies 2022 | active, not recruiting | Phase 3 | 187 |
| NCT00322101 | Low-Dose or High-Dose Conditioning Followed by Peripheral Blood Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia 2006 | completed | Phase 3 | 25 |
| NCT02566304 | Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies 2015 | completed | Phase 2 | 35 |
| NCT04644016 | Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders 2020 | recruiting | Phase 2 | 31 |
| NCT01518153 | Planned Donor Lymphocyte Infusion (DLI) After Allogeneic Stem Cell Transplantation (SCT) 2012 | terminated | Phase 2 | 16 |
| NCT00818961 | Donor Stem Cell Transplant in Treating Patients With High-Risk Hematologic Cancer 2005 | terminated | Phase 2 | 36 |
| NCT00402558 | Alloreactive NK Cells for Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) 2006 | completed | Phase 1 | 15 |
| NCT01875237 | Donor Lymphocyte Infusion (DLI) of T-cells Genetically Modified With iCasp9 Suicide Gene 2013 | terminated | PHASE1, PHASE2 | 3 |
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 BCL2. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Tacrolimus Anhydrous approved for Myelodysplastic Syndromes (MDS)?
Tacrolimus Anhydrous has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Myelodysplastic Syndromes (MDS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Myelodysplastic Syndromes (MDS). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Tacrolimus Anhydrous in clinical trials for Myelodysplastic Syndromes (MDS)?
ClinicalTrials.gov lists 8 registered trials linking Tacrolimus Anhydrous to Myelodysplastic Syndromes (MDS): 1 is currently recruiting; 1 is active or not yet recruiting; 3 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT05316701). It plans or enrolled 187 participants. Registration activity spans 2005 to 2022.
What does the evidence show for Tacrolimus Anhydrous in Myelodysplastic Syndromes (MDS)?
Tacrolimus Anhydrous has 3 completed registered trials for Myelodysplastic Syndromes (MDS) 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 BCL2. 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.