Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome - Gene therapy for WAS

GENETHO0 sites10 target enrollmentDecember 9, 2010

Overview

No summary available.

Registry

who.int

Start Date

December 9, 2010

End Date

TBD

Last Updated

8 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

Male

Sponsor

GENETHO

•1\. a. Males of all ages
•b. Severe WAS (clinical score 3 – 5\) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry
•c. Molecular confirmation by WAS gene DNA sequencing
•2\. Unless desease severity indicates that one cannot wait for 3 months (score 5; refractory thrombocytopenia with platelets \<5000 with bleeding or severe autoimmunity),
•a. Lack of HLA\-genotypically identical bone marrow after 3 month search
•b. Lack of a 10/10 or 9/10 antigen HLA\-matched unrelated donor after 3 month search
•c. Lack of a HLA\-matched cord blood after 3 month search
•3\. Parental, guardian, patient signed informed consent/assessment
•4\. Willing to return for follow\-up during the 2 year study and lifelong for off study review
•5\. Only for patients who have received previous allogenic haematopoietic stem cell transplant:

•1\. a. Patient with HLA\-genotypically identical bone marrow
•b. Patient with 10/10 or 9/10 antigen HLA\-matched unrelated donor or with HLA\-matched cord blood
•2\. a. Contraindication to leukapheresis
•i. Anaemia (Hb \< 8g/dl)
•ii.Severe vascularitis
•iii.Refractory thrompopenia
•b. Contraindication to bone marrow harvest
•c. Contraindication to administration of conditioning medication
•3\. HIV seropositive patient