Wiskott-Aldrich Syndrome Clinical Trial
Official title:
Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome
Verified date | November 2019 |
Source | Genethon |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.
Status | Completed |
Enrollment | 6 |
Est. completion date | November 13, 2019 |
Est. primary completion date | November 13, 2019 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | N/A and older |
Eligibility | Inclusion Criteria: - males of all ages - severe WAS (clinical score 3-5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry - molecular confirmation by WAS gene DNA sequencing - lack of HLA-genotypically identical bone marrow or of a 10/10 antigen HLA-matched unrelated donor or cord blood after 3 month search - parental, guardian, patient signed informed consent/assent - willing to return for follow-up - only for patients who have received previous allogenic hematopoietic stem cell transplant: - failed allogenic hematopoietic stem cell transplant - contraindication to repeat transplantation Exclusion Criteria: - patient with HLA-genotypically identical bone marrow - patient with 10/10 antigen HLA-matched unrelated donor or cord blood - contraindication to leukapheresis - contraindication to bone marrow harvest - contraindication to administration of conditioning medication - HIV positive patient |
Country | Name | City | State |
---|---|---|---|
United Kingdom | Great Ormond Street Hospital | London | |
United Kingdom | Royal Free Hospital | London |
Lead Sponsor | Collaborator |
---|---|
Genethon | Great Ormond Street Hospital for Children NHS Foundation Trust, Institute of Child Health |
United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Improvement in the eczema status | Improvement in the eczema status as compared with the baseline status at study entry on clinical evaluation | 2 years | |
Primary | 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 | 2 years | |
Primary | 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 | 2 years | |
Primary | 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 | 2 years | |
Primary | 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 | 2 years | |
Secondary | Occurrence and type of adverse events | Occurrence and type of adverse events reported during the course of the study | 2 years | |
Secondary | Change in medical conditions | Assessment of weight, vital signs, ECG and laboratory exams during the course of the study | 2 years | |
Secondary | Safety of lentivirus gene transfer into Hematopoietic Stem Cells | Detection of replication competent lentivirus (RCL) and lentivirus integration sites analysis | 3, 6, 12, 24 months / 6, 12, 18, 24 months | |
Secondary | Improvement of microthrombocytopenia | Improvement of microthrombocytopenia as compared with the baseline evaluation at study entry | 3, 6, 12, 24 months | |
Secondary | 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 | 2 years | |
Secondary | Evidence of sustained engrafment of WASP-expressing transduced cells | Quantification of vector copy numbers and detection of vector-derived WASP expression | 6 weeks, 1, 3, 6, 9, 12, 18 & 24 months | |
Secondary | Reconstitution of humoral and cell mediated immunity | Reconstitution of humoral and cell mediated immunity as compared with the baseline evaluation at study entry | 9, 12, 18 & 24 months |
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