A Phase 1/2 interventional study of Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene in Wiskott-Aldrich Syndrome, sponsored by Genethon. Completed at 2 sites in United Kingdom. Open to male participants. Per ClinicalTrials.gov, last updated 2021-06-03.
Sponsored by Genethon · Phase 1/2, Interventional, and Treatment
This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.
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.
37 studies on the registry are indexed under Wiskott-Aldrich Syndrome; 4 are open to participants now.
This study's enrollment of 6 is below the median of 18 across 26 interventional studies indexed under Wiskott-Aldrich Syndrome.
Browse Wiskott-Aldrich Syndrome studies →Genethon is the lead sponsor of 15 studies on the registry; 5 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
autologous CD34 positive cells transduced with a lentiviral vector containing the human WAS gene
Genetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WAS gene
Improvement in the eczema status
Improvement in the eczema status as compared with the baseline status at study entry on clinical evaluation
Time frame: 2 years
Reduction in the frequency and severity of infection episodes
Reduction in the frequency and severity of infection episodes as compared with the baseline status and the patient's historical data collected over the 2 years prior to study entry
Time frame: 2 years
Reduction in the frequency and severity of bruising and bleeding episodes
Reduction in the frequency and severity of bruising and bleeding episodes as compared with the baseline status and the patient's historical data collected over the 2 years prior to study entry
Time frame: 2 years
Reduction in the frequency and severity of autoimmune disorders
Reduction in the frequency and severity of autoimmune disorders as compared with the baseline status at study entry
Time frame: 2 years
Reduction in the number of disease related days of hospitalization
Reduction in the number of disease related days of hospitalization as compared with the patient's historical data collected over the 2 years prior to study entry
Time frame: 2 years
Occurrence and type of adverse events
Occurrence and type of adverse events reported during the course of the study
Time frame: 2 years
Change in medical conditions
Assessment of weight, vital signs, ECG and laboratory exams during the course of the study
Time frame: 2 years
Safety of lentivirus gene transfer into Hematopoietic Stem Cells
Detection of replication competent lentivirus (RCL) and lentivirus integration sites analysis
Time frame: 3, 6, 12, 24 months / 6, 12, 18, 24 months
Improvement of microthrombocytopenia
Improvement of microthrombocytopenia as compared with the baseline evaluation at study entry
Time frame: 3, 6, 12, 24 months
Decrease in the number and volume of platelets transfusions
Decrease in the number and volume of platelets transfusions as compared with patient's historical data collected over the 2 years prior to study entry
Time frame: 2 years
Evidence of sustained engrafment of WASP-expressing transduced cells
Quantification of vector copy numbers and detection of vector-derived WASP expression
Time frame: 6 weeks, 1, 3, 6, 9, 12, 18 & 24 months
Reconstitution of humoral and cell mediated immunity
Reconstitution of humoral and cell mediated immunity as compared with the baseline evaluation at study entry
Time frame: 9, 12, 18 & 24 months
This study is completed, as verified in Nov 2019. You cannot join it, but the record below documents what was studied.
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