-
Immune mediated adverse events in the randomized phase 3 TOPAZ 1 study of durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer1 Topic1 Quiz
-
Belantamab mafodotin plus bortezomib and dexamethasone in patients with relapsed or refractory multiple myeloma DREAMM7 updated overall survival analysis from a global randomised open label phase 3 trial1 Topic1 Quiz
-
PACULit Daily Literature Update: Real-world patient profile and step-up dosing process of early initiators of teclistamab for multiple myeloma in US hospitals An analysis using the Premier Healthcare Database1 Topic1 Quiz
-
PACULit Daily Literature Update: Effects of BojungikkiTang on immune response and clinical outcomes in NSCLC patients receiving immune checkpoint inhibitors a randomized pilot study1 Topic1 Quiz
-
PACULit Daily Literature Update: Long acting lipegfilgrastim and antimicrobials as vigorous primary prophylaxis in bendamustine treated patients with indolent B cell non Hodgkin lymphoma a multicentric real life experience1 Topic1 Quiz
-
First-line treatment with HDACis plus tislelizumab combined with chemotherapy in advanced NSCLC a single-arm phase II study1 Topic1 Quiz
-
Comparison of outcomes with elranatamab and real world treatments in the UK for triple class exposed relapsed and refractory multiple myeloma1 Topic1 Quiz
-
Overall Survival with Inavolisib in PIK3CA-Mutated Advanced Breast Cancer1 Topic1 Quiz
-
Enhanced CAR T-Cell Therapy for Lymphoma after Previous Failure1 Topic1 Quiz
-
Phase I II clinical trial on the safety and preliminary efficacy of donor derived anti leukemia cytotoxic T lymphocytes for the prevention of leukemia relapse in children given haploidentical hematopoietic stem cell transplantation study rational and design1 Topic1 Quiz
-
Brentuximab vedotin plus chemotherapy for the treatment of frontline systemic anaplastic large cell lymphoma subgroup analysis of the ECHELON2 study at 5 years followup1 Topic1 Quiz
-
Effectiveness and Safety of Immunotherapy for Hepatocellular Carcinoma in Clinical Practice A Brazilian Multicenter Study1 Topic1 Quiz
-
Talquetamab improves patient reported symptoms and health related quality of life in relapsed or refractory multiple myeloma Results from the phase 12 MonumenTAL1 study1 Topic1 Quiz
-
Encorafenib, Cetuximab, and mFOLFOX6 in BRAF-Mutated Colorectal Cancer1 Topic1 Quiz
-
Durvalumab Alone or Combined With Novel Agents for Unresectable Stage III Non Small Cell Lung Cancer Update From the COAST Randomized Clinical Trial1 Topic1 Quiz
-
Real world patient profile and step up dosing process of early initiators of teclistamab for multiple myeloma in US hospitals An analysis using the Premier Healthcare Database1 Topic1 Quiz
-
Virtual reality for outpatient management of cancer pain a pilot dosing study1 Topic1 Quiz
-
Brentuximab vedotin plus chemotherapy for the treatment of frontline systemic anaplastic large cell lymphoma1 Topic1 Quiz
-
Optimal treatment duration in metastatic renal cell carcinoma patients responding to immune checkpoint inhibitors should we treat beyond two years1 Topic1 Quiz
-
Effects of Metformin on Survival and Toxicity in Patients with Metastatic Non Small Cell Lung Cancer Treated with Nivolumab1 Topic1 Quiz
-
ACPE Required Forms: PACULit Literature Updates September 2025: Oncology3 Topics
Participants 440
Lesson 10,
Topic 1
In Progress
Phase I II clinical trial on the safety and preliminary efficacy of donor derived anti leukemia cytotoxic T lymphocytes for the prevention of leukemia relapse in children given haploidentical hematopoietic stem cell transplantation study rational and design
Lesson Progress
0% Complete
Daily Literature Update
Phase I II clinical trial on the safety and preliminary efficacy of donor derived anti leukemia cytotoxic T lymphocytes for the prevention of leukemia relapse in children given haploidentical hematopoietic stem cell transplantation study rational and design
Montagna D, Comoli P, Tanzi M, et al. Phase I/II clinical trial on the safety and preliminary efficacy of donor-derived anti-leukemia cytotoxic T lymphocytes for the prevention of leukemia relapse in children given haploidentical hematopoietic stem cell transplantation: study rational and design. Front Immunol. 2025;16:1601961-. PMID: 40547030.
Introduction
Relapse prevention after haploidentical hematopoietic stem cell transplantation remains a major challenge in pediatric acute leukemia. Donor-derived anti-leukemia cytotoxic T lymphocytes (CTLs) may offer a targeted immunotherapeutic approach to improve outcomes.
Study Overview
Study Type: Phase I/II clinical trial
Population: Pediatric ALL/AML patients with high relapse risk undergoing haplo-HCT
Intervention: Infusion of donor-derived anti-leukemia CTLs expanded under GMP conditions
Outcomes: Safety (GVHD incidence) and preliminary efficacy (leukemia relapse rates)
- Designed to evaluate safety of donor-derived anti-leukemia CTLs in pediatric haplo-HCT recipients
- CTL product predominantly CD3+/CD8+ memory/effector T cells with strong anti-leukemic function
- Infusions planned within 60 days post-transplant with dose escalation
- Primary safety endpoint: incidence of acute and chronic GVHD
Context & Related Research
- Montagna D et al., 2025: Phase I/II pediatric trial rationale and design assessing donor anti-leukemia CTLs for relapse prevention (PMID:40547030), addressing critical pediatric gaps.
- Ferulli F et al., 2019: Demonstrated feasibility of generating potent WT1-specific donor CTLs with cytotoxicity against leukemia blasts in pediatric haplo-HCT setting (PMID:31279696).
- Chapuis AG et al., 2013: Adult HCT recipients showed anti-leukemic activity and T-cell persistence with low GVHD rates after WT1-reactive T cell therapy (PMID:23447018).
Clinical Implications
- Donor-derived anti-leukemia CTL therapy is a promising relapse prevention strategy in high-risk pediatric HCT.
- Careful GVHD monitoring essential due to immunomodulatory therapy.
- GMP-compliant production protocols facilitate clinical translation.
Strengths & Limitations
| Strengths | Limitations |
|---|---|
| First pediatric clinical trial targeting relapse prevention with donor-derived CTLs | No clinical outcome data available yet |
| GMP-compliant manufacturing process with functional CTL characterization | Restricted to patients with available leukemia blast samples for CTL production |
| Precision patient selection based on MRD and cytogenetics | Unknown long-term CTL persistence and GVHD impact |
Future Directions
Ongoing trial results will clarify safety and preliminary efficacy; optimization of CTL expansion and combination therapies remain areas for research.
Conclusion
The Leuk-CTL-001 trial seeks to establish the safety and potential efficacy of donor-derived anti-leukemia CTLs as a relapse-preventing strategy in high-risk pediatric haploidentical HCT recipients, representing a significant advancement in targeted cellular therapy for pediatric leukemia.
Listen to the Podcast
A short discussion of today’s highlight.
Citations
- Montagna D, Comoli P, Tanzi M, et al. Phase I/II clinical trial on the safety and preliminary efficacy of donor-derived anti-leukemia cytotoxic T lymphocytes for the prevention of leukemia relapse in children given haploidentical hematopoietic stem cell transplantation: study rational and design. Front Immunol. 2025;16:1601961-. PMID: 40547030.
- Ferulli F et al., 2019. WT1-specific donor cytotoxic T lymphocytes and pediatric haploidentical HCT. PMID: 31279696.
- Chapuis AG et al., 2013. Anti-leukemic activity and T-cell persistence post WT1-reactive T cell therapy in adults. PMID: 23447018.