Gene Therapy for WAS

Clinical Trial Description

This clinical trial is an ex vivo gene therapy trial. The investigational product corresponds to autologous CD34+ cells transduced with a lentiviral vector harboring the human WASP gene. ;

Study Design

See also
Status	Clinical Trial	Phase
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Completed	`NCT01953016` - Participation in a Research Registry for Immune Disorders
Active, not recruiting	`NCT02333760` - Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome	Phase 1/Phase 2
Terminated	`NCT01319851` - Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation	N/A
Recruiting	`NCT05687474` - Baby Detect : Genomic Newborn Screening
Recruiting	`NCT04371939` - Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome	Phase 2
Completed	`NCT00160355` - Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome	Phase 1
Recruiting	`NCT01821781` - Immune Disorder HSCT Protocol	Phase 2
Completed	`NCT01289847` - A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency	Phase 4
Completed	`NCT01347242` - Gene Therapy for Wiskott-Aldrich Syndrome (WAS)	Phase 1/Phase 2
Terminated	`NCT00006054` - Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies	N/A
Enrolling by invitation	`NCT03198195` - Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome	N/A
Recruiting	`NCT03019809` - A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients	Phase 2
Terminated	`NCT00909363` - Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients	Phase 2
Enrolling by invitation	`NCT01852370` - Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases	Phase 1/Phase 2
Completed	`NCT03513328` - Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation	Phase 1/Phase 2
Active, not recruiting	`NCT01410825` - Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome	Phase 1/Phase 2
Active, not recruiting	`NCT00004341` - Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders	N/A
Completed	`NCT03399461` - Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)
Active, not recruiting	`NCT03837483` - A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome	Phase 3

NCT number	NCT01347346
Study type	Interventional
Source	Genethon
Contact
Status	Completed
Phase	Phase 1/Phase 2
Start date	May 2011
Completion date	January 9, 2017

Gene Therapy for WAS

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms