Clinical Trials Logo

Clinical Trial Summary

This is an open-label, single arm study to evaluate the cryopreserved formulation of OTL-103 Gene Therapy. OTL-103 consists of autologous CD34+ hematopoietic stem cells in which the gene encoding for the Wiskott-Aldrich Syndrome is introduced by means of a third generation lentiviral vector.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT03837483
Study type Interventional
Source Fondazione Telethon
Contact
Status Active, not recruiting
Phase Phase 3
Start date January 21, 2019
Completion date September 2027

See also
  Status Clinical Trial Phase
Recruiting NCT01652092 - Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies N/A
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 NCT01347346 - Gene Therapy for WAS Phase 1/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 NCT01347242 - Gene Therapy for Wiskott-Aldrich Syndrome (WAS) Phase 1/Phase 2
Completed NCT01289847 - A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency Phase 4
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)