Summary
Abatacept 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 7 registered trials linking Abatacept to Sickle Cell Disease: 2 are currently recruiting; 3 are active or not yet recruiting; 2 have completed. The most advanced is Phase 2 (NCT07616154). The largest enrolment is 90 participants (NCT07599176). Registration activity spans 2014 to 2026. The literature layer holds 1 publication for this pair: 1 clinical trial publication. The dated items are from 2020. 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 | 44.0 (trials 22.0, literature 2.0, tier 15, approved bonus 5.0) |
| Registered trials | 7 total: 2 recruiting, 3 active / not yet recruiting, 2 completed, 0 other |
| Linked publications | 1 (1 clinical trial publication) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | inhibitor |
| Data sources | DGIdb |
| Linked via biomarker / target | CD80 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT03924401 | Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant 2019 | active, not recruiting | Phase 2 | 30 |
| NCT07616154 | Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease 2026 | not yet recruiting | Phase 2 | 45 |
| NCT02867800 | Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease 2016 | completed | Phase 1 | 24 |
| NCT01917708 | Bone Marrow Transplant With Abatacept for Non-Malignant Diseases 2014 | completed | Phase 1 | 10 |
| NCT06145282 | Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function 2023 | active, not recruiting | PHASE1, PHASE2 | 6 |
| NCT03128996 | Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders 2017 | recruiting | PHASE1, PHASE2 | 29 |
| NCT07599176 | Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors 2026 | recruiting | PHASE1, PHASE2 | 90 |
Published literature
- 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: 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 CD80. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Abatacept approved for Sickle Cell Disease?
Abatacept 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 Abatacept in clinical trials for Sickle Cell Disease?
ClinicalTrials.gov lists 7 registered trials linking Abatacept to Sickle Cell Disease: 2 are currently recruiting; 3 are active or not yet recruiting; 2 have completed. The most advanced is Phase 2 (NCT07616154). The largest enrolment is 90 participants (NCT07599176). Registration activity spans 2014 to 2026.
What does the evidence show for Abatacept in Sickle Cell Disease?
Abatacept has 2 completed trials and 1 linked publication 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 CD80. 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.