Wiskott-Aldrich Syndrome clinical trials
The registry lists 41 studies for Wiskott-Aldrich Syndrome across more than 10 countries. 4 are recruiting or about to start. 21 of them are mainly about Wiskott-Aldrich Syndrome (how this is decided). Per ClinicalTrials.gov, last updated 2026-10-27.
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Status
Who runs them
National Human Genome Research Institute (NHGRI) (3) · Paul Szabolcs (3) · Federal Research Institute of Pediatric Hematology, Oncology and Immunology (3) · Genethon (3) · Roswell Park Cancer Institute (2) · Emory University (2) · Fondazione Telethon (2) · Bio Products Laboratory (2)
What is being tested here
Drugs under test in Wiskott-Aldrich Syndrome studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Fludarabine | 1 | 0 | 6 |
| Melphalan | 1 | 0 | 4 |
| Alemtuzumab | 1 | 0 | 2 |
| Thiotepa | 1 | 0 | 2 |
| Hydroxyurea | 1 | 0 | 1 |
| Cyclophosphamide | 0 | 0 | 3 |
| Fludarabine phosphate | 0 | 0 | 3 |
| Immunoglobulins, Intravenous | 0 | 2 | 3 |
| Immunoglobulin G | 0 | 2 | 3 |
| Busulfan | 0 | 0 | 2 |
Drug classes tested here Alkylating agents 1 open · Antimetabolites 1 open
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 2
- Nov 2026Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases · Paul Szabolcs
- Nov 2026Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT · Paul Szabolcs
Who these studies take
Of the 2 Wiskott-Aldrich Syndrome studies recruiting now, 0 (0%) set no upper age limit and 2 (100%) stop at 75 or younger. 2 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 2 (100%) | 2 (100%) |
| 18 | 2 (100%) | 2 (100%) |
| 30 | 2 (100%) | 2 (100%) |
| 40 | 2 (100%) | 2 (100%) |
| 50 | 2 (100%) | 2 (100%) |
| 65 | 0 (0%) | 0 (0%) |
| 75 | 0 (0%) | 0 (0%) |
| 85 | 0 (0%) | 0 (0%) |
Across all 41 studies, recruiting or not: 63% stop at 75 or younger and 56% at 65; 93% take children; 0% are for women only and 20% for men only; 7% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 15, 2026.
Results posted1
- Immune Disorder HSCT ProtocolOct 27, 2026NCT01821781
Studies
Recruiting first, then most recently updated. Showing 41 of 41. search them.
- Analysis of the Therapeutic and Clinical Profile of Cannabinoid Treatments: A Real-World StudyEnrolling by invitationNCT073624071 country
- Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICRecruitingNCT045283551 country
- Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency DiseasesEnrolling by invitationNCT01852370Phase 1/21 country
- Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTRecruitingNCT01962415Phase 21 country
- Immune Disorder HSCT ProtocolCompletedNCT01821781Phase 21 country
- Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesActive, not recruitingNCT01652092Not applicable1 country
- A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich SyndromeActive, not recruitingNCT03837483Phase 32 countries
- Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich SyndromeCompletedNCT01410825Phase 1/21 country
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood CancerTerminatedNCT03333486Phase 21 country
- Gene Therapy for Wiskott-Aldrich SyndromeCompletedNCT01515462Phase 1/21 country
- Molecular and Clinical Studies of Primary Immunodeficiency DiseasesActive, not recruitingNCT000063191 country
- THC Crossover StudyCompletedNCT04429568Not applicable1 country
- Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationCompletedNCT03513328Phase 1/21 country
- Participation in a Research Registry for Immune DisordersCompletedNCT019530161 country
- Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited DisordersCompletedNCT00919503Phase 21 country
- Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich SyndromeActive, not recruitingNCT02333760Phase 1/22 countries
- Gene Therapy for Wiskott-Aldrich Syndrome (WAS)CompletedNCT01347242Phase 1/21 country
- Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome.CompletedNCT043501641 country
- Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990CompletedNCT020649332 countries
- Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich SyndromeStatus unknownNCT04371939Phase 21 country
- Bone Marrow Transplant With Abatacept for Non-Malignant DiseasesCompletedNCT01917708Phase 11 country
- Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesCompletedNCT01529827Phase 21 country
- Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) PatientsTerminatedNCT00909363Phase 21 country
- Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)CompletedNCT033994611 country
- A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS PatientsStatus unknownNCT03019809Phase 21 country
- Gene Therapy for WASCompletedNCT01347346Phase 1/21 country
- Interleukin-2 Treatment for Wiskott-Aldrich SyndromeCompletedNCT00774358Phase 11 country
- Alefacept and Allogeneic Hematopoietic Stem Cell TransplantationTerminatedNCT01319851Not applicable1 country
- Study of Megakaryocytes From Patients With Abnormal Platelet VesiclesCompletedNCT000864761 country
- Post-transplant Cyclophosphamide in Wiskott-Aldrich SyndromeStatus unknownNCT03198195
- Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich SyndromeCompletedNCT00160355Phase 11 country
- Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood CellsTerminatedNCT02512679Phase 2
- Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric SubjectsCompletedNCT00634569Phase 41 country
- Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood CellsCompletedNCT00730314Phase 1/21 country
- A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary ImmunodeficiencyCompletedNCT01289847Phase 43 countries
- Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)CompletedNCT00885833Phase 1/21 country
- Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases.CompletedNCT00278954Phase 31 country
- Allogeneic Bone Marrow Transplantation in Patients With Primary ImmunodeficienciesTerminatedNCT00006054Not applicable1 country
- Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency PatientsCompletedNCT00220766Phase 32 countries
- Study of Genetic and Molecular Defects in Primary Immunodeficiency DisordersStatus unknownNCT000043411 country