Phase 3
Completed N=98
TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PID
Primary Immune Deficiency Disorder · Hematopoietic Stem Cell Transplantation
Source: ClinicalTrials.gov NCT02327351 ↗
Enrolled (actual)
98
Serious AEs
17.3%
Results posted
Feb 2021
Primary outcomePrimary: Overall Survival — 86; 86; 87 percentage of survival probability — p=0.95
◆ Published Evidence
Highly cited
142citations · ~13 / year
Single-Center Experience of Unrelated and Haploidentical Stem Cell Transplantation with TCRαβ and CD19 Depletion in Children with Primary Immunodeficiency Syndromes.
Summary
Treatment Study to assess of safety and efficiency of T cells receptor (TCR) alfa beta depleted graft for hematopoietic stem cell transplantation (HSCT) from haploidentical and unrelated donors in patients with primary immunodeficiency diseases
Linked Publications
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Single-Center Experience of Unrelated and Haploidentical Stem Cell Transplantation with TCRαβ and CD19 Depletion in Children with Primary Immunodeficiency Syndromes.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Survival |
86; 86; 87 | 0.95 |
| SECONDARY Transplant Related Mortality (TRM) |
11 | — |
| SECONDARY Acute Graft Versus Host Diseases (аGVHD) |
17 | — |
| SECONDARY Chronic Graft Versus Host Diseases (cGVHD) |
9 | — |
| SECONDARY Cellular Immunological Reconstitution |
67 | — |
| SECONDARY Percentage of Patients With Full Donor Chimerism |
48 | — |
| SECONDARY Viral Infections After Transplant |
50; 60 | 0.35 |
Eligibility Criteria
Inclusion Criteria
- Patients aged ≥ 1 months and 40, WHO > 4
- Signed written informed consent
Exclusion Criteria
- Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 ml / min)
- Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction <40%)
- Serious concurrent uncontrolled medical disorder
- Pregnant or breast feeding female patient
- Lack of parents' informed consent.
Data sourced from ClinicalTrials.gov (NCT02327351) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.