Safety and Efficacy of SMART101 in Adult Patients With Hematological Malignancies After Haploidentical HSCT With Post-transplant Cyclophosphamide
Study Details
Study Description
Brief Summary
The purpose of this study is to evaluate the safety and the efficacy of SMART101 (Human T Lymphoid Progenitors (HTLP)) injection to accelerate immune reconstitution after haploidentical hematopoietic stem cell transplantation (HSCT) with post-transplant cyclophosphamide (PT-Cy) in adult patients with hematological malignancies.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1/Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Patients with acute leukemia or myelodysplastic syndrome and eligible for an haplo PT-Cy HSCT 2 cohorts of patients will be included in the study based on the type of conditioning regimen: The cohort A will include up to 17 patients receiving a myeloablative conditioning (MAC). The cohort B will include up to 17 patients receiving a reduced intensity conditioning (RIC). Enrollment of patients in each cohort will be done in parallel. |
Biological: Allogeneic T cell progenitors, cultured ex-vivo
Injection of T cell progenitors 6 days after haplo HSCT and 2 days after the last administration of cyclophosphamide
Other Names:
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Outcome Measures
Primary Outcome Measures
- Occurrence of Unexpected Unacceptable Toxicities (UUT) [14 days post SMART101 infusion]
to evaluate the safety profile of the study drug
- CD4+ T cell count [100 days post-HSCT]
to evaluate the efficacy of the study drug
Secondary Outcome Measures
- Occurrence of adverse events (AEs) [up to 24 months post-HSCT]
- T cell immune reconstitution [up to 12 months post-HSCT]
Time course of the T cell immune reconstitution, with a focus on naive CD4+ cells and total CD8+cells
- Cumulative incidence of infections [Day 100, and Months 6 and 12 post-HSCT]
- Non-relapse mortality (NRM) [Day 100, and Months 6, 12 and 24 post-HSCT]
Other Outcome Measures
- Overall Survival (OS) [Month 24 post-HSCT]
- Disease-free Survival [Month 24 post-HSCT]
Eligibility Criteria
Criteria
Main Inclusion Criteria:
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Patients with AML, ALL or MDS eligible for an allogeneic HSCT with a haploidentical donor with post-transplant cyclophosphamide.
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Patients must be ≥ 18 years of age at the time of signing the ICF.
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Patients must have a Karnofsky index ≥ 70%.
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Patients must have a left ventricular ejection fraction of ≥40%.
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Patients must have an intact pulmonary function or Diffusing capacity of the Lungs for Carbon Monoxide (DLCO) ≥ 45% of predicted.
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Patients must have adequate hepatic and renal functions, as assessed by standard laboratory criteria.
Main Exclusion Criteria:
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Patients who have received prior allogeneic stem cell transplantation.
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Patients who have received prior treatment with another cellular therapy within 4 weeks before the planned day of SMART101 infusion.
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Patients who plan to receive, are concurrently receiving or have received any investigational agent within 4 weeks before the planned day of SMART101 infusion.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Smart Immune SAS
Investigators
- Principal Investigator: Fabio CICERI, MD, Pr., I.R.C.C.S. Ospedale San Raffaele
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- SI101-02