Wiskott-Aldrich syndrome

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Information
Disease name
Wiskott-Aldrich syndrome
Disease ID
DOID:9169
Description
"A syndrome that is characterized by abnormal immune system function and a reduced ability to form blood clots resulting from a decrease in the number and size of blood cell fragments involved in clotting (microthrombocytopenia)." [url:https\://ghr.nlm.nih.gov/condition/wiskott-aldrich-syndrome]
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
Genes Mutation Description Source Links
NCT ID Status Phase Summary Start date Completion date
NCT01410825 Active, not recruiting Phase 1/Phase 2 Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome July 2011 August 2024
NCT02333760 Active, not recruiting Phase 1/Phase 2 Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome September 2014 October 2032
NCT03333486 Active, not recruiting Phase 2 Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer December 7, 2017 August 28, 2024
NCT03837483 Active, not recruiting Phase 3 A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome January 21, 2019 September 2027
NCT00730314 Completed Phase 1/Phase 2 Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells August 2008 August 2015
NCT00885833 Completed Phase 1/Phase 2 Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS) February 2007 March 2012
NCT01289847 Completed Phase 4 A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency March 2011 April 2014
NCT03513328 Completed Phase 1/Phase 2 Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation June 15, 2018 February 19, 2023
NCT01347242 Completed Phase 1/Phase 2 Gene Therapy for Wiskott-Aldrich Syndrome (WAS) March 2011 November 13, 2019
NCT01347346 Completed Phase 1/Phase 2 Gene Therapy for WAS May 2011 January 9, 2017
NCT01529827 Completed Phase 2 Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies February 28, 2012 August 29, 2019
NCT04350164 Completed Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome. April 1, 2012 June 2020
NCT00160355 Completed Phase 1 Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome May 2005 February 2009
NCT01917708 Completed Phase 1 Bone Marrow Transplant With Abatacept for Non-Malignant Diseases January 2014 September 19, 2019
NCT01953016 Completed Participation in a Research Registry for Immune Disorders September 30, 2013 July 19, 2023
NCT02064933 Completed Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990 February 2, 2014 May 1, 2019
NCT03399461 Completed Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS) January 24, 2018 September 14, 2018
NCT00220766 Completed Phase 3 Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients August 2002 March 2004
NCT00278954 Completed Phase 3 Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases. January 2006 November 2007
NCT01852370 Enrolling by invitation Phase 1/Phase 2 Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases June 20, 2013 November 2026
NCT05687474 Recruiting Baby Detect : Genomic Newborn Screening September 1, 2022 August 31, 2025
NCT01652092 Recruiting N/A Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies September 4, 2012 December 2026
NCT01821781 Recruiting Phase 2 Immune Disorder HSCT Protocol March 2013 March 2027
NCT00909363 Terminated Phase 2 Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients June 2009 June 30, 2017
NCT00006054 Terminated N/A Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies March 2000 December 2002
NCT02512679 Terminated Phase 2 Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells February 2007 February 2014
NCT01319851 Terminated N/A Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation September 2010 September 2013
NCT00004341 Unknown status Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders July 1995
NCT03019809 Unknown status Phase 2 A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients June 2016 July 2019
NCT04371939 Unknown status Phase 2 Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome November 5, 2019 July 30, 2023
NCT03198195 Unknown status Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome March 10, 2015 July 10, 2020
Disase is a (Disease Ontology)
DOID:225
Cross Reference ID (Disease Ontology)
ICD10CM:D82.0
Cross Reference ID (Disease Ontology)
ICD9CM:279.12
Cross Reference ID (Disease Ontology)
MESH:D014923
Cross Reference ID (Disease Ontology)
MIM:301000
Cross Reference ID (Disease Ontology)
NCI:C3448
Cross Reference ID (Disease Ontology)
SNOMEDCT_US_2023_03_01:36070007
Cross Reference ID (Disease Ontology)
UMLS_CUI:C0043194
Exact Synonym (Disease Ontology)
Wiskott syndrome
OrphaNumber from OrphaNet (Orphanet)
906
ICD10 preferred id (Insert disease from ICD10)
D0003320
ICD10 class code (Insert disease from ICD10)
D82.0
MeSH unique ID (MeSH (Medical Subject Headings))
D014923