| Completed | Baby Detect : Genomic Newborn Screening NCT05687474 | Centre Hospitalier Universitaire de Liege | — |
| Unknown | Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wi NCT04371939 | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | Phase 2 |
| Active Not Recruiting | A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldric NCT03837483 | Fondazione Telethon | Phase 3 |
| Completed | Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation NCT03513328 | University of Florida | Phase 1 / Phase 2 |
| Completed | Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS) NCT03399461 | GlaxoSmithKline | — |
| Terminated | Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Pa NCT03333486 | Roswell Park Cancer Institute | Phase 2 |
| Unknown | A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS P NCT03019809 | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | Phase 2 |
| Unknown | Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome NCT03198195 | Capital Research Institute of Pediatrics | — |
| Active Not Recruiting | Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome NCT02333760 | Genethon | Phase 1 / Phase 2 |
| Completed | Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990 NCT02064933 | National Institute of Allergy and Infectious Diseases (NIAID) | — |
| Completed | Bone Marrow Transplant With Abatacept for Non-Malignant Diseases NCT01917708 | Emory University | Phase 1 |
| Completed | Participation in a Research Registry for Immune Disorders NCT01953016 | National Human Genome Research Institute (NHGRI) | — |
| Enrolling By Invitation | Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases NCT01852370 | Paul Szabolcs | Phase 1 / Phase 2 |
| Active Not Recruiting | Immune Disorder HSCT Protocol NCT01821781 | Washington University School of Medicine | Phase 2 |
| Active Not Recruiting | Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies NCT01652092 | Masonic Cancer Center, University of Minnesota | N/A |
| Completed | Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome. NCT04350164 | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | — |
| Completed | Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem NCT01529827 | Roswell Park Cancer Institute | Phase 2 |
| Completed | Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome NCT01410825 | David Williams | Phase 1 / Phase 2 |
| Completed | Gene Therapy for WAS NCT01347346 | Genethon | Phase 1 / Phase 2 |
| Completed | Gene Therapy for Wiskott-Aldrich Syndrome (WAS) NCT01347242 | Genethon | Phase 1 / Phase 2 |
| Completed | A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency NCT01289847 | Bio Products Laboratory | Phase 4 |
| Terminated | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation NCT01319851 | Emory University | N/A |
| Terminated | Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients NCT00909363 | Weill Medical College of Cornell University | Phase 2 |
| Completed | Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells NCT00730314 | Children's Hospital Los Angeles | Phase 1 / Phase 2 |
| Terminated | Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells NCT02512679 | Children's Hospital Los Angeles | Phase 2 |
| Completed | Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS) NCT00885833 | The Korean Society of Pediatric Hematology Oncology | Phase 1 / Phase 2 |
| Completed | Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases. NCT00278954 | Bio Products Laboratory | Phase 3 |
| Completed | Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome NCT00160355 | St. Jude Children's Research Hospital | Phase 1 |
| Completed | Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients NCT00220766 | Grifols Therapeutics LLC | Phase 3 |
| Unknown | Molecular and Clinical Studies of Primary Immunodeficiency Diseases NCT00006319 | National Human Genome Research Institute (NHGRI) | — |
| Terminated | Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies NCT00006054 | Fairview University Medical Center | N/A |
| Unknown | Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders NCT00004341 | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | — |