Summary
Tacrolimus Anhydrous has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Sickle Cell Disease would be drug repurposing rather than first-in-human development. This does not mean it is approved for Sickle Cell Disease. ClinicalTrials.gov lists 8 registered trials linking Tacrolimus Anhydrous to Sickle Cell Disease: 2 are currently recruiting; 1 is active or not yet recruiting; 2 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT05736419). The largest enrolment is 29 participants (NCT03128996). Registration activity spans 2001 to 2023. No published literature item is linked to Tacrolimus Anhydrous and Sickle Cell Disease 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 Sickle Cell Disease 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: No spontaneous remission; progressive organ damage; median survival historically 40-60 years but improving; vaso-occlusive crises (VOC) in 1-4 per year average.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 40.0 (trials 20.0, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 2 recruiting, 1 active / not yet recruiting, 2 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 | ADA |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT01565616 | Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease 2012 | completed | Phase 2 | 22 |
| NCT05736419 | A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT) 2023 | recruiting | Phase 2 | 24 |
| NCT00034528 | Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia 2001 | terminated | Phase 2 | 2 |
| NCT01279616 | A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease 2010 | terminated | Phase 2 | 8 |
| NCT04776850 | Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies 2020 | withdrawn | Early Phase 1 | — |
| NCT02061800 | CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant 2013 | active, not recruiting | PHASE1, PHASE2 | 14 |
| NCT02435901 | HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity 2008 | completed | PHASE1, PHASE2 | 29 |
| NCT03128996 | Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders 2017 | recruiting | PHASE1, PHASE2 | 29 |
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 ADA. 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 Sickle Cell Disease?
Tacrolimus Anhydrous has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Sickle Cell Disease would be drug repurposing rather than first-in-human development. This does not mean it is approved for Sickle Cell Disease. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Tacrolimus Anhydrous in clinical trials for Sickle Cell Disease?
ClinicalTrials.gov lists 8 registered trials linking Tacrolimus Anhydrous to Sickle Cell Disease: 2 are currently recruiting; 1 is active or not yet recruiting; 2 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT05736419). The largest enrolment is 29 participants (NCT03128996). Registration activity spans 2001 to 2023.
What does the evidence show for Tacrolimus Anhydrous in Sickle Cell Disease?
Tacrolimus Anhydrous has 2 completed registered trials for Sickle Cell Disease 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 ADA. 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.