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Clinical trial for HLA-haploidentical transplantation using alpha-beta T cell and B cell depletio

Phase 2
Recruiting
Conditions
Fanconi anenia severe combined immunodeficiency
Registration Number
JPRN-jRCTs032230287
Lead Sponsor
Iguchi Akihiro
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Recruiting
Sex
All
Target Recruitment
3
Inclusion Criteria

1)Patients with Fanconi anemia or severe combined immunodeficiency who are difficult to receive allogeneic hematopoietic stem cell transplantation (HSCT) using PTCY.
2)At time of registration, patients who require allogeneic HSCT, but HLA7/8 antigen (A, B, C, DRB1) matched or 8/8 antigen matched donor is not available. Therefore, the intended donor is one of the following.
a. HLA4/8 antigen (A, B, C, DRB1) matched related donor.
b. HLA5/8 antigen (A, B, C, DRB1) matched related donor.
c. HLA6/8 antigen (A, B, C, DRB1) matched related donor.
3)Patients under 18 years of age at the time of HSCT.
4)Patients with an ECOG performance status (PS) score of 0-2 at enrollment.
5)Patients with adequate organ function who meet the following criteria at the time of enrollment. Laboratory findings must be within 30 days prior to enrollment.
a.Serum direct bilirubin <1.5mg/dL
b.Serum creatinine
<5 years old, <0.8mg/dL
5-9 years old, <1.2mg/dL
>10 years old, <1.5mg/dL
c.Ejection fraction>45% and QTc<0.45sec
6)Patients whose written consent from legal representative have been obtained to participate in this study.

Exclusion Criteria

1) Patients who had received prior HSCT.
2) The donor is unable to use G-CSF due to allergy or other reasons.
3) Patients who have uncontrollable infection.
4) Patients with severe mental disorder.
5) Patients with pregnant or likely to be pregnant.
6) Patients who are ineligible by physician

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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