Floatz Rating
B61/100
Confidence
Indicative
Sponsor
St. Jude Children's Research Hospital
Modality
Small molecule
Development Phase
Phase 2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
59High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT07616154CT.gov Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease | — | Not Yet Recruiting | — | — | — |
NCT04362293CT.gov Reduced Intensity Transplantation for Severe Sickle Cell Disease | — | Suspended | — | — | — |
NCT02757885CT.gov Transplantation for Patients With Sickle Cell Disease From Mismatched Family Donors of Bone Marrow | — | Completed | — | — | — |
NCT03653338CT.gov T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias | — | Recruiting | — | — | — |
NCT03513328CT.gov Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation | — | Completed | — | — | — |
NCT03263559CT.gov Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507) | — | Completed | — | — | — |
NCT03609840CT.gov Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients | — | Completed | — | — | — |
NCT03128996CT.gov Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders | — | Recruiting | — | — | — |
NCT02867800CT.gov Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease | — | Completed | — | — | — |
NCT01461837CT.gov Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease | — | Active Not Recruiting | — | — | — |
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NCT01049854CT.gov CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant | — | Completed | — | — | — |
NCT00152113CT.gov Haploidentical Stem Cell Transplant for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Ultrasound | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
65High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Sickle Cell Disease
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Thiotepa (this asset) | St. Jude Children's Research Hospital | P2 | B · 61 |
| Glutamine | Ain Shams University | P4 | BBB |
| Hydroxyurea | St. Jude Children's Research Hospital | P4 | BB |
| Nivestim | St. Jude Children's Research Hospital | P4 | BB |
| Magnesium Metallicum | Medical College of Wisconsin | P3 | BB |
| Crizanlizumab | Novartis Pharmaceuticals | P4 | BB |
| Nepenthe | Oman Medical Speciality Board | P4 | BB |
| ARG | Tanta University | P3 | BB |
| Fentanyl | University College Dublin | P4 | BB |
+42 more in the Sickle Cell Disease cohort
Other indications for Thiotepa
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Graft Versus Host Disease | — | P4 | BBB · 73 |
| Myelodysplastic/Myeloproliferative Neoplasm | — | P2/3 | BBB · 72 |
| Juvenile Myelomonocytic Leukemia | — | P3 | BBB · 72 |
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
Citation
Floatz Terminal. Thiotepa in Sickle Cell Disease. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/thiotepa-sickle-cell-disease
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