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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT02333760
Other study ID # GNT-WAS-03
Secondary ID
Status Active, not recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date September 2014
Est. completion date October 2032

Study information

Verified date May 2021
Source Genethon
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

An open follow up study of patients enrolled in the Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 10
Est. completion date October 2032
Est. primary completion date October 2032
Accepts healthy volunteers No
Gender Male
Age group N/A and older
Eligibility Inclusion Criteria: - Patients enrolled in the initial phase I/II WAS conducted in France and United Kingdom (GTG002.07 and GTG003.08). - Parents, guardians or patient signed informed consent, guardians or patient signed informed consent Exclusion Criteria: • Parents, guardians, patients unwilling to return for the follow up study period.

Study Design


Related Conditions & MeSH terms


Intervention

Genetic:
Autologous CD34+ cells transduced with WASP lentiviral vector
Follow up of ex vivo gene therapy transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WASP gene

Locations

Country Name City State
France Hopital Necker - Enfants Malades Paris
United Kingdom UCL Institute of Child Health London

Sponsors (1)

Lead Sponsor Collaborator
Genethon

Countries where clinical trial is conducted

France,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Incidence and type of SAEs Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality yearly from 3 years to 15 years
Primary Lentiviral integration sites Presence of lentiviral integration sites in different cells sub-populations yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Primary Vector copy numbers Quantification of vector copy numbers on sorted cells population by q-PCR yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Primary Replication competent lentivirus (RCL) Presence of RCL yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Primary Change in medical conditions Weight and complete clinical exam yearly from 3 years to 10 years
Primary Key medical events related to WAS Eczema status, infections, bleeding symptoms, autoimmune manifestation yearly from 3 years to 10 years
Primary Hematological reconstitution CBC including platelets count and size yearly from 3 years to 10 years
Primary Reconstitution of cell mediated and humoral immunity Immunophenotyping panel, whole blood lymphocytes proliferation assays, restoration of antibody production, humoral response to antigene yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida )
Secondary Need for associated treatments Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions yearly from 3 years to 15 years
Secondary Representation of TCR families Representation of TCR families by PCR TREC (TCR excision circle) and TCR V beta panel yearly from 3 years to 5 years
Secondary Bone marrow content Numbers and type of cells in bone marrow yearly from 3 years to 5 years (optional)
See also
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Completed NCT01347346 - Gene Therapy for WAS Phase 1/Phase 2
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Completed NCT01289847 - A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency Phase 4
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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