Unique ID issued by UMIN | UMIN000030806 |
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Receipt number | R000035089 |
Scientific Title | A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome |
Date of disclosure of the study information | 2018/01/15 |
Last modified on | 2021/03/15 10:52:12 |
A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome
Hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome
A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome
Hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome
Japan |
Wiskott-Aldrich syndrome
Medicine in general | Hematology and clinical oncology | Pediatrics |
Others
YES
The objective of the present gene therapy clinical trial is to evaluate the safety of the transduced stem cells infusion in patients affected by WAS after a reduced-intensity conditioning regimen and its efficacy in improving the patients' immune-function and thrombocytopenia.
Safety,Efficacy
Exploratory
Explanatory
Phase I,II
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
Interventional
Single arm
Non-randomized
Open -no one is blinded
Uncontrolled
1
Treatment
Medicine | Gene |
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 Busulfan.
1. Rituximab (day-22)
375 mg/m2
2. Preconditioning chemotherapy
Fludarabine 30mg/m2 x 2 (day-3, day-2)
Busulfan cumulative target AUC 48000 ng/mL*h (day-3 to -1, every 6 hours)
3. Infusion of WASP cDNA-transduced CD34 positive HSC
5 x 10^6/kg (at least 3 x 10^6/kg)
Not applicable |
Not applicable |
Male
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
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.
3
1st name | |
Middle name | |
Last name | MASAFUMI ONODERA |
National Center for Child Heath and Development
Division of Immunology
2-10-1 Okura, Setagaya-ku, Tokyo, Japan
03-5494-7295
onodera-m@ncchd.go.jp
1st name | |
Middle name | |
Last name | TORU UCHIYAMA |
National Center for Child Heath and Development
Division of Immunology
2-10-1 Okura, Setagaya-ku, Tokyo, Japan
03-5494-7035
uchiyama-t@ncchd.go.jp
National Center for Child Heath and Development
Japan Agency for Medical Research and Development
Japanese Governmental office
NO
2018 | Year | 01 | Month | 15 | Day |
Unpublished
No longer recruiting
2017 | Year | 10 | Month | 06 | Day |
2017 | Year | 10 | Month | 19 | Day |
2018 | Year | 01 | Month | 17 | Day |
2025 | Year | 03 | Month | 31 | Day |
2018 | Year | 01 | Month | 14 | Day |
2021 | Year | 03 | Month | 15 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000035089
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