Disease · MONDO_0044348
hemoglobinopathy
6 sourcesdiseases-2026-09-18-0.1.0
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ConductScience Library. hemoglobinopathy. Diseases, record MONDO_0044348. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0044348
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Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Name
- hemoglobinopathy
- Open Targets ID
- MONDO_0044348
- Highest stage of its drugs
- Phase 3, for 2 of its 22 drugs, from ClinicalTrials.gov
Source Open Targets
Drugs22
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| SIROLIMUS | CHEMBL413 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| TACROLIMUS | CHEMBL3989887 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| ALEMTUZUMAB | CHEMBL1201587 | Antibody | Phase 2/3 | ClinicalTrials.gov | 9 | 9 | 9 | Drugs and targets |
| BUSULFAN | CHEMBL820 | Small molecule | Phase 2/3 | ClinicalTrials.gov | 8 | 8 | 8 | Drugs and targets |
| FLUDARABINE | CHEMBL1568 | Small molecule | Phase 2/3 | ClinicalTrials.gov | 7 | 7 | 7 | Drugs and targets |
| CYCLOPHOSPHAMIDE | CHEMBL1200796 | Small molecule | Phase 2/3 | ClinicalTrials.gov | 5 | 5 | 5 | Drugs and targets |
| ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE) | CHEMBL2108981 | Phase 2/3 | ClinicalTrials.gov | 2 | 2 | 0 | Drugs and targets | |
| FILGRASTIM | CHEMBL1201567 | Protein | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| LENOGRASTIM | CHEMBL2109128 | Protein | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| PEGFILGRASTIM | CHEMBL1201568 | Protein | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
1 to 10 of 22
See all 22 drugsTargets59
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| CSF3R | Granulocyte colony-stimulating factor receptor | 3 | Granulocyte colony stimulating factor receptor agonist | Phase 2/3 | https://conductscience.org/library/genes/1441 | Drugs and targets |
| FKBP1A | Peptidyl-prolyl cis-trans isomerase FKBP1A | 3 | FK506-binding protein 1A inhibitor | Phase 3 | https://conductscience.org/library/genes/2280 | Drugs and targets |
| RRM1 | Ribonucleoside-diphosphate reductase RR1 | 2 | Ribonucleoside-diphosphate reductase RR1 inhibitor | Phase 2 | https://conductscience.org/library/genes/6240 | Drugs and targets |
| RRM2 | Ribonucleoside-diphosphate reductase RR1 | 2 | Ribonucleoside-diphosphate reductase RR1 inhibitor | Phase 2 | https://conductscience.org/library/genes/6241 | Drugs and targets |
| RRM2B | Ribonucleoside-diphosphate reductase RR1 | 2 | Ribonucleoside-diphosphate reductase RR1 inhibitor | Phase 2 | https://conductscience.org/library/genes/50484 | Drugs and targets |
| ADRM1 | 26S proteasome | 1 | 26S proteasome inhibitor | Early phase 1 | https://conductscience.org/library/genes/11047 | Drugs and targets |
| CD52 | CAMPATH-1 antigen | 1 | CAMPATH-1 antigen inhibitor | Phase 2/3 | https://conductscience.org/library/genes/1043 | Drugs and targets |
| IMPDH1 | Inosine-5'-monophosphate dehydrogenase (IMPDH) | 1 | Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitor | Phase 2 | https://conductscience.org/library/genes/3614 | Drugs and targets |
| IMPDH2 | Inosine-5'-monophosphate dehydrogenase (IMPDH) | 1 | Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitor | Phase 2 | https://conductscience.org/library/genes/3615 | Drugs and targets |
| MS4A1 | B-lymphocyte antigen CD20 | 1 | B-lymphocyte antigen CD20 binding agent | Early phase 1 | https://conductscience.org/library/genes/931 | Drugs and targets |
1 to 10 of 59
See all 59 targetsTrials15
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.
| 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 |
1 to 10 of 15
See all 15 trialsComparisons in its trials14
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Number of Participants With Infections | 57 of 84 | 9 of 14 | 98 | NCT00919503 | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): deaths from any cause | 7 of 84 | 6 of 14 | 98 | NCT00919503 | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Overall Survival | 80 of 84 | 9 of 14 | 98 | NCT00919503 | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Non-relapse Mortality | 2 of 84 | 5 of 14 | 98 | NCT00919503 | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): serious adverse events | 17 of 84 | 5 of 14 | 98 | NCT00919503 | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Number of Patients With Grade II-IV Acute Graft-versus-host Disease | 44 of 84 | 8 of 12 | 96 | NCT00919503 | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Disease Response at One Year Following Hematopoietic Cell Transplantation | 78 of 84 | 8 of 11 | 95 | NCT00919503 | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Preliminary Efficacy | 83 of 84 | 10 of 11 | 95 | NCT00919503 | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Number of Patients With of Chronic Graft-versus-host Disease | 29 of 84 | 5 of 11 | 95 | NCT00919503 | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Immune Reconstitution Following Hematopoietic Cell Transplantation | 39 of 73 | 4 of 9 | 82 | NCT00919503 | Trial comparisons |
1 to 10 of 14
See all 14 comparisons in its trialsPapers from its trials5
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
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 disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0044348
GET https://conductscience.org/api/diseases/v1/MONDO_0044348/drugs?page=2