Clinical trial · Interventional
Trial to Assess the Efficacy of a TCR Alfa Beta Depleted Graft in Pediatric Affected by ALL or AML and Receiving an HSCT
Phase I/II Study of Allogeneic Hematopoietic Stem Cell Transplantation From an HLA-partially Matched Family Donor After TCR Alfa Beta Negative Selection in Pediatric Patients Affected by Hematological Disorders
NCT01810120CI-TRIAL-00025582completedPhase 1 / Phase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Allocation: Non-Randomized Endpoint Classification: Safety/Feasibility Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment Study to assess the feasibility and safety of the infusion of a T cells receptor (TCR) alfa beta depleted graft in pediatric patients affected by malignant and non-malignant hematological disorders and receiving an Hematopoietic stem cell transplantation (HSCT) from a Human leukocyte antigen (HLA) partially matched family donor.
Conditions
Conditions (4)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Acute Lymphoblastic Leukemia | Acute Lymphoblastic Leukemia | ONTOLOGY_EXACT | 0.98 |
| Leukemia Acute Myeloid - AML | — | UNRESOLVED | — |
| Myelodysplastic Syndromes | Myelodysplastic Syndrome | ALIAS | 0.90 |
| Non-Hodgkin Lymphoma | Non-Hodgkin Lymphoma | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| TCR alfa beta T cell depletion | Biological | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- TCR alfa beta depleted graft, infusion
- description
- The leukapheresis product will undergo TCR alfa beta negative selection following the standardized protocol.
- interventionNames
- Biological: TCR alfa beta T cell depletion
Primary outcomes (1)
- measure
- CD34+ cells
- timeFrame
- up to 3 month
- description
- Target number of CD34+ cells in at least 80% of the patients
Secondary outcomes (4)
- measure
- Primary and secondary graft failure
- timeFrame
- up to 24 months after transplantation
- description
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 3 Months
- Maximum age
- 20 Years
Show eligibility criteria text
Inclusion Criteria: * Patients aged ≥ 3 months and \< 21 years * Patients diagnosed with malignant hemopathies (Acute Lymphoblastic leukemia (ALL), Acute Myeloid Leukemia (AML), Non-Hodgkin Lymphoma (NHL)) in complete morphological remission or Myelodysplastic Syndromes (MDS), Solid Tumors or non malignant hematological disorders (SCID, Acquired and Congenital Aplastic Anemia, other Primary Immunodeficiencies, Life-threatening Cytopenia) eligible for an allogeneic transplantation and lacking a related or unrelated HLA-matched donor * Patients displaying an HLA-partially matched family donor * Lansky/Karnofsky score \> 40, WHO \> 4 * Signed written informed consent Exclusion Criteria: * Grade \>II acute GvHD or chronic extensive GvHD at the time of inclusion * Patient receiving an immunosuppressive treatment for GvHD treatment at the time of inclusion * 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%) * Current active infectious disease (including positive HIV serology or viral RNA) * Serious concurrent uncontrolled medical disorder * Pregnant or breast feeding female patient * Lack of parents' informed consent.
References
Publications (29)
- BACKGROUNDCopelan EA. Hematopoietic stem-cell transplantation. N Engl J Med. 2006 Apr 27;354(17):1813-26. doi: 10.1056/NEJMra052638. No abstract available. PMID 16641398
- BACKGROUNDRocha V, Locatelli F. Searching for alternative hematopoietic stem cell donors for pediatric patients. Bone Marrow Transplant. 2008 Jan;41(2):207-14. doi: 10.1038/sj.bmt.1705963. Epub 2007 Dec 17. PMID 18084331
- BACKGROUNDGluckman E. Cord blood transplantation. Biol Blood Marrow Transplant. 2006 Aug;12(8):808-12. doi: 10.1016/j.bbmt.2006.05.011. No abstract available. PMID 16864050
- BACKGROUNDLocatelli F, Pende D, Maccario R, Mingari MC, Moretta A, Moretta L. Haploidentical hemopoietic stem cell transplantation for the treatment of high-risk leukemias: how NK cells make the difference. Clin Immunol. 2009 Nov;133(2):171-8. doi: 10.1016/j.clim.2009.04.009. Epub 2009 May 29. PMID 19481979
- BACKGROUNDMartelli MF, Aversa F, Bachar-Lustig E, Velardi A, Reich-Zelicher S, Tabilio A, Gur H, Reisner Y. Transplants across human leukocyte antigen barriers. Semin Hematol. 2002 Jan;39(1):48-56. doi: 10.1053/shem.2002.29255. PMID 11799529
- BACKGROUNDReisner Y, Kapoor N, Kirkpatrick D, Pollack MS, Cunningham-Rundles S, Dupont B, Hodes MZ, Good RA, O'Reilly RJ. Transplantation for severe combined immunodeficiency with HLA-A,B,D,DR incompatible parental marrow cells fractionated by soybean agglutinin and sheep red blood cells. Blood. 1983 Feb;61(2):341-8. PMID 6217853
- BACKGROUNDAntoine C, Muller S, Cant A, Cavazzana-Calvo M, Veys P, Vossen J, Fasth A, Heilmann C, Wulffraat N, Seger R, Blanche S, Friedrich W, Abinun M, Davies G, Bredius R, Schulz A, Landais P, Fischer A; European Group for Blood and Marrow Transplantation; European Society for Immunodeficiency. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. Lancet. 2003 Feb 15;361(9357):553-60. doi: 10.1016/s0140-6736(03)12513-5.