Status:
NOT_YET_RECRUITING
Total Lymphoid Irradiation Pre-HSCT in Severe Congenital Neutropenia
Lead Sponsor:
Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Conditions:
Severe Congenital Neutropenia
GATA2 Deficiency
Eligibility:
All Genders
18-21 years
Phase:
PHASE2
Brief Summary
Severe congenital neutropenia (SCN) is a group of primary immunodeficiencies caused by distinct gene mutations and characterized by neutrophil maturation impairment, which leads to neutropenia, predis...
Eligibility Criteria
Inclusion
- Clinical indications for HSCT in SCN: clinical diagnosis of SCN with (1) no adequate response to G-CST therapy or (2) with malignant transformation or (3) unfavorable mutations of known SCN genes
- GATA2 deficiency
- SCN patients age at HSCT 18 months - 21 years
- GATA2 deficiency patients age at HSCT more than 10 years
- Signed informed consent to participate in the study
- Presence of HLA-matched unrelated or HLA-mismatched related donor
Exclusion
- Presence of HLA matched related donor in absence of pathologic SCN gene mutation
- Inability to perform TCRab/CD19 graft depletion
- Contraindications for HSCT due to patients somatic condition
Key Trial Info
Start Date :
April 14 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 1 2026
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT04844177
Start Date
April 14 2021
End Date
April 1 2026
Last Update
April 14 2021
Active Locations (1)
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1
HSCT department
Moscow, Russia, 117198