Summary
Alemtuzumab 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 Alemtuzumab to Sickle Cell Disease: 3 are currently recruiting; 2 are active or not yet recruiting; 2 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT01962415). It plans or enrolled 100 participants. Registration activity spans 2002 to 2018. The literature layer holds 5 publications for this pair: 5 clinical trial publications. Publication years run from 2020 to 2026. These are primary trial reports rather than syntheses, so results have not yet been pooled or graded independently.
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 | 54.0 (trials 24.0, literature 10.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 3 recruiting, 2 active / not yet recruiting, 2 completed, 1 other |
| Linked publications | 5 (5 clinical trial publications) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | antibody; antibody; inhibitor |
| Data sources | DGIdb |
| Linked via biomarker / target | CD52 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT02105766 | Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure 2014 | active, not recruiting | Phase 2 | 56 |
| NCT01962415 | Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT 2014 | recruiting | Phase 2 | 100 |
| NCT00408447 | Stem Cell Transplant in Sickle Cell Disease and Thalassemia 2004 | status unknown | Phase 2 | 53 |
| NCT02061800 | CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant 2013 | active, not recruiting | PHASE1, PHASE2 | 14 |
| NCT00176852 | Stem Cell Transplant for Hemoglobinopathy 2002 | completed | PHASE2, PHASE3 | 22 |
| NCT05249452 | Adding Azathioprine/Hydroxyurea Preconditioning to Alemtuzumab/TBI to Reduce Risk of Graft Failure in MSD HSCT in Adult SCD Patients 2018 | completed | Not applicable | 20 |
| NCT03128996 | Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders 2017 | recruiting | PHASE1, PHASE2 | 29 |
| NCT03653338 | T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias 2018 | recruiting | PHASE1, PHASE2 | 5 |
Published literature
- Clinical trial publication Nonmyeloablative HLA-identical sibling transplant for sickle cell disease in children: a multicenter prospective studyNickel RS, Abraham A, Chiang KY et al. · Blood advances · 2026 · PMID 41945757
- Clinical trial publication Alemtuzumab clearance, lymphocyte count, and T-cell chimerism after hematopoietic stem cell transplant in sickle cell diseaseFurstenau D, Peer CJ, Hughes TE et al. · Pharmacotherapy · 2022 · PMID 34669981
- Clinical trial publication Non-myeloablative human leukocyte antigen-matched related donor transplantation in sickle cell disease: outcomes from three independent centresAlzahrani M, Damlaj M, Jeffries N et al. · British journal of haematology · 2021 · PMID 33534948
- Clinical trial publication Timing of Alemtuzumab With Respect to Day of Bone Marrow Infusion and its Effects Upon Engraftment and Graft-Versus-Host Disease in Patients With Sickle Cell Disease: A Single-Institutional StudySahdev I, Brochstein J, Werther N et al. · Journal of pediatric hematology/oncology · 2020 · PMID 32890079
- Clinical trial publication Abatacept is effective as GVHD prophylaxis in unrelated donor stem cell transplantation for children with severe sickle cell diseaseNgwube A, Shah N, Godder K et al. · Blood advances · 2020 · PMID 32813873
Mechanism and notes
Recorded mechanism or class: antibody; antibody; inhibitor.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target CD52. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Alemtuzumab approved for Sickle Cell Disease?
Alemtuzumab 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 Alemtuzumab in clinical trials for Sickle Cell Disease?
ClinicalTrials.gov lists 8 registered trials linking Alemtuzumab to Sickle Cell Disease: 3 are currently recruiting; 2 are active or not yet recruiting; 2 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT01962415). It plans or enrolled 100 participants. Registration activity spans 2002 to 2018.
What does the evidence show for Alemtuzumab in Sickle Cell Disease?
Alemtuzumab has 2 completed trials and 5 linked publications for Sickle Cell Disease. Completed trials may or may not have posted results; follow the NCT links to check. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target CD52. 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.