Disease · MONDO_0010518
Wiskott-Aldrich syndrome
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. Wiskott-Aldrich syndrome. Diseases, record MONDO_0010518. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010518
On this page
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.
- Other names
Aldrich Syndrome, Syndrome, Aldrich, Syndrome, Wiskott-Aldrich, WAS, Wiskott-Aldrich syndrome 1, Wiskott-Aldrich syndrome, X-linked recessive, eczema-thrombocytopenia-immunodeficiency syndrome, immunodeficiency 2
- Open Targets
Drugs16
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 |
|---|---|---|---|---|---|---|---|---|
| FLUDARABINE | CHEMBL1568 | Small molecule | Phase 2 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
| CYCLOPHOSPHAMIDE | CHEMBL1200796 | Small molecule | Phase 2 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| ELTROMBOPAG | CHEMBL461101 | Small molecule | Phase 2 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| ROMIPLOSTIM | CHEMBL1201832 | Protein | Phase 2 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| ALEMTUZUMAB | CHEMBL1201587 | Antibody | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| BUSULFAN | CHEMBL820 | Small molecule | Phase 1/2 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| ALDESLEUKIN | CHEMBL1201438 | Protein | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| MELPHALAN | CHEMBL852 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| THIOTEPA | CHEMBL671 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ALEFACEPT | CHEMBL1201571 | Protein | Unknown | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
1 to 10 of 16
See all 16 drugsTargets11
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 |
|---|---|---|---|---|---|---|
| MPL | Thrombopoietin receptor | 2 | Thrombopoietin receptor agonist | Phase 2 | https://conductscience.org/library/genes/4352 | Drugs and targets |
| NR3C1 | Glucocorticoid receptor | 2 | Glucocorticoid receptor agonist | Unknown | https://conductscience.org/library/genes/2908 | Drugs and targets |
| CD2 | T-cell surface antigen CD2 | 1 | T-cell surface antigen CD2 inhibitor | Unknown | https://conductscience.org/library/genes/914 | Drugs and targets |
| CD52 | CAMPATH-1 antigen | 1 | CAMPATH-1 antigen inhibitor | Phase 2 | https://conductscience.org/library/genes/1043 | Drugs and targets |
| DHFR | Dihydrofolate reductase | 1 | Dihydrofolate reductase inhibitor | Unknown | https://conductscience.org/library/genes/1719 | Drugs and targets |
| IL2RA | Interleukin-2 receptor | 1 | Interleukin-2 receptor agonist | Phase 1 | https://conductscience.org/library/genes/3559 | Drugs and targets |
| IL2RB | Interleukin-2 receptor | 1 | Interleukin-2 receptor agonist | Phase 1 | https://conductscience.org/library/genes/3560 | Drugs and targets |
| IL2RG | Interleukin-2 receptor | 1 | Interleukin-2 receptor agonist | Phase 1 | https://conductscience.org/library/genes/3561 | Drugs and targets |
| PPIA | Peptidyl-prolyl cis-trans isomerase A | 1 | Cyclophilin A modulator | Unknown | https://conductscience.org/library/genes/5478 | Drugs and targets |
| TOP2A | DNA topoisomerase II | 1 | DNA topoisomerase II inhibitor | Unknown | https://conductscience.org/library/genes/7153 | Drugs and targets |
1 to 10 of 11
See all 11 targetsTrials9
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 |
|---|---|---|---|---|---|---|---|---|
| NCT00909363 | Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients | Phase 2 | 24 | 2019-03-18 | Wiskott-Aldrich Syndrome, Thrombocytopenia, Bleeding | ELTROMBOPAG | Its record names the disease | ClinicalTrials.gov |
| NCT02512679 | Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells | Phase 2 | 20 | 2016-06-17 | Stem Cell Transplantation, Bone Marrow Transplantation, Peripheral Blood Stem Cell Transplantation, Allogeneic Transplantation, Genetic Diseases, Thalassemia, Pediatrics, Diamond-Blackfan Anemia, Combined Immune Deficiency, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Lymphoproliferative Disease, Metabolic Diseases | ALEMTUZUMAB, CYCLOPHOSPHAMIDE, FLUDARABINE | Its record names the disease | ClinicalTrials.gov |
| NCT01319851 | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation | Not applicable | 3 | 2015-03-09 | Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic-granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Schwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Fanconi Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia | ALEFACEPT | Its record names the disease | ClinicalTrials.gov |
| NCT04350164 | Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome. | 67 | Wiskott-Aldrich Syndrome | ROMIPLOSTIM | Its record names the disease | ClinicalTrials.gov | ||
| NCT04371939 | Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome | Phase 2 | 30 | Wiskott-Aldrich Syndrome | ELTROMBOPAG, ROMIPLOSTIM | Its record names the disease | ClinicalTrials.gov | |
| NCT00774358 | Interleukin-2 Treatment for Wiskott-Aldrich Syndrome | Phase 1 | 9 | Wiskott-Aldrich Syndrome (WAS), X-linked Thrombocytopenia | ALDESLEUKIN | Its record names the disease | ClinicalTrials.gov | |
| NCT00885833 | Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS) | Phase 1/2 | 5 | Wiskott-Aldrich Syndrome | BUSULFAN, FLUDARABINE | Its record names the disease | ClinicalTrials.gov | |
| NCT00160355 | Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome | Phase 1 | 4 | Wiskott-Aldrich Syndrome | FLUDARABINE, MELPHALAN, THIOTEPA | Its record names the disease | ClinicalTrials.gov | |
| NCT00006054 | Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies | Not applicable | Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Hyper IgM Syndrome, Severe Combined Immunodeficiency, Leukocyte Adhesion Deficiency Syndrome, Virus-Associated Hemophagocytic Syndrome | BUSULFAN, CYCLOPHOSPHAMIDE, CYCLOSPORINE, ETOPOSIDE, METHOTREXATE, METHYLPREDNISOLONE, PREDNISONE | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials0
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.
None in this release.
Papers from its trials3
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_0010518
GET https://conductscience.org/api/diseases/v1/MONDO_0010518/drugs