Wiskott-Aldrich syndrome
Conditions
Interventions
WASP cDNA-transduced autologous hematopoietic stem cells are administered to patients affected by WAS after the administration of rituximab and preconditioning chemotherapy including Fludarabine and B
Sponsors
National Center for Child Heath and Development
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Patients who meet all of the following criteria will be included. 1. Diagnosis of WAS determined by genetic mutation and at least one of the following: -Severe WASP mutation -Absent WASP expression -Severe clinical score (Zhu clinical score greater than or equal to 3) 2. Donor for HSCT -Patients with age less than 5: Negative search for related and unrelated HLA-identical donor -Patients with age greater than or equal to 5: Negative search for related HLA-identical donor
Exclusion criteria
Exclusion criteria: Patients who meet any of the following criteria will be excluded. 1. Patients positive for HIV infection 2. Patients affected by neoplasia 3. Patients with cytogenetic alterations typical of MDS/AML 4. Patients with end-organ function or any other severe disease, which, in the judgement of the investigator, would make the patients inappropriate for entry into this study 5. Patients who underwent an allogeneic hematopoietic stem cell transplantation in the previous 6 months 6. Patients who underwent an allogeneic hematopoietic stem cell transplantation with evidence of residual donor cells 7. Patients who have the possibility of severe allergic reactions, to rituximab and the products derived from cow, pig, sheep and mouse. 8. Patients who do not agree with a contraception during the trial. 9. Patients who are considered inappropriate, in the judgement of the investigator, due to any other reasons.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety of reduced conditioning regimen and LV gene transfer into HSC -Hematological reconstitution -Regimen related non-hematological toxicity -Short-term safety and tolerability of LV-transduced cell infusion -The absence of replication competent LV and abnormal clonal proliferation Efficacy of gene therapy -Overall survival -Sustained engraftment of genetically corrected hematopoietic stem cells -Improvement in immune function -Improvement in platelet count | — |
Countries
Japan
Contacts
Public ContactTORU UCHIYAMA
National Center for Child Heath and Development Division of Immunology
Outcome results
None listed