Disease · MONDO_0044348
Trials15
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.
1 to 15 of 15
Download this page as JSON| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT00580034 | Allo Non-Myeloablative SCT Utilizing Mis-Matched Family Member Stem Cells Purged Using Campath | Phase 2 | 176 | 2014-06-03 | Lymphoma, Myeloma, Leukemia, Myelodysplasia, Solid Tumors | ALEMTUZUMAB | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT00919503 | Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders | Phase 2 | 98 | 2021-08-13 | Non-Neoplastic Hematologic and Lymphocytic Disorder | FLUDARABINE PHOSPHATE, TREOSULFAN | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT00301834 | Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders | Phase 2 | 35 | 2013-05-16 | Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia | ALEMTUZUMAB, BUSULFAN, FLUDARABINE PHOSPHATE | Its record names the disease | ClinicalTrials.gov |
| NCT00176852 | Stem Cell Transplant for Hemoglobinopathy | Phase 2/3 | 22 | 2017-05-09 | Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome | ALEMTUZUMAB, BUSULFAN, CYCLOPHOSPHAMIDE, FLUDARABINE | Its record names the disease | ClinicalTrials.gov |
| NCT00744692 | Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders | Phase 1 | 22 | 2014-08-13 | Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease | ALEMTUZUMAB, FLUDARABINE, HYDROXYUREA, MELPHALAN, THIOTEPA | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT00578344 | Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP | Not applicable | 8 | 2020-07-31 | Sickle Cell Disease, Hemoglobin SC | ALEMTUZUMAB, BUSULFAN, CYCLOPHOSPHAMIDE, MESNA | Its record names the disease | ClinicalTrials.gov |
| NCT03513328 | Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation | Phase 1/2 | 6 | 2023-09-15 | Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy | BUSULFAN, FLUDARABINE, THIOTEPA | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT00579111 | Reduced Intensity Preparative Regimen Followed by Stem Cell Transplant (FAB) | Phase 1/2 | 4 | 2012-08-09 | Myelodysplastic and Myeloproliferative Disorders, Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, Multiple Myeloma, Plasma Cell Dyscrasia, Lymphoproliferative Disorders, Hematologic Diseases | ALEMTUZUMAB, BUSULFAN, FLUDARABINE | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT01534676 | Effects of Transfusion of Older Stored Red Cells | Not applicable | 3 | 2016-10-05 | Sickle Cell Disease, Thalassemia | BLOOD CELLS, RED | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT00004143 | Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes | Phase 2 | 2 | 2013-11-11 | Sickle Cell Anemia, Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria (PNH), Pure Red Cell Aplasia | ALEMTUZUMAB, CYCLOPHOSPHAMIDE, FLUDARABINE | Its record names the disease | ClinicalTrials.gov |
| NCT00153985 | Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies | Phase 2 | 2 | 2013-03-12 | Hemoglobinopathies, Sickle Cell Disease, Thalassemia | ALEMTUZUMAB, BUSULFAN, FLUDARABINE | Its record names the disease | ClinicalTrials.gov |
| NCT00993343 | Randomized Trial Comparing Sirolimus and Tacrolimus Versus Cyclosporine and Methotrexate as Graft-versus-host Disease (GVHD) Prophylaxis After Allogeneic Stem Cell Transplantation | Phase 3 | 215 | Graft-versus-host Disease, Survival | SIROLIMUS, TACROLIMUS | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT06872333 | Allo HSCT for High Risk Hemoglobinopathies | Phase 2 | 62 | Graft Failure, Sickle Cell Disease, Hemoglobinopathies | ALEMTUZUMAB, BUSULFAN, CYCLOPHOSPHAMIDE, FLUDARABINE, MYCOPHENOLATE MOFETIL, SIROLIMUS, TACROLIMUS, THIOTEPA | Its record names the disease | ClinicalTrials.gov | |
| NCT03980769 | Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Thiotepa in Treating Patients With Non-malignant Disorders | Phase 2 | 40 | Non-Neoplastic Hematopoietic and Lymphoid Cell Disorder | FLUDARABINE PHOSPHATE, THIOTEPA, TREOSULFAN | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT04776850 | Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies | Early phase 1 | 0 | Beta Thalassemia Major, Sickle Beta 0 Thalassemia, Sickle Beta Plus Thalassemia, Sickle Beta Thalassemia, Sickle Cell Disease, Sickle Cell-SS Disease | BORTEZOMIB, BUSULFAN, CYCLOPHOSPHAMIDE, DEXAMETHASONE, FLUDARABINE PHOSPHATE, MYCOPHENOLATE MOFETIL, RITUXIMAB, TACROLIMUS ANHYDROUS | Open Targets' reading of its record | ClinicalTrials.gov |
diseases-2026-09-18-0.1.0
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this list from software.
Twenty rows a page, as JSON. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0044348/trials