Safety and Efficacy of SMART101 in Pediatric and Adult Patients With Hematological Malignancies After T Cell Depleted Allo-HSCT

NCT ID: NCT04959903

Last Updated: 2023-03-06

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 36 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-03-31
• Study Completion Date: 2027-05-31

Brief Summary

The purpose of this study is to evaluate the safety and the efficacy of SMART101 (Human T Lymphoid Progenitor (HTLP)) injection to accelerate immune reconstitution after T cell depleted allogeneic hematopoietic stem cell transplantation (HSCT) in adult and pediatric patients with hematological malignancies.

Conditions

Hematological Malignancies

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Adult patients affected by hematological malignancies

Adult patients affected by acute leukemia (AML, ALL or acute leukemia of ambiguous lineage) or myelodysplastic syndrome eligible for a T depleted allogeneic HSCT

Group Type: EXPERIMENTAL

Allogeneic T cell progenitors, cultured ex-vivo

Intervention Type: BIOLOGICAL

Injection of T cell progenitors at \[Day 4-Day 10\] after T cell depleted allogeneic HSCT

Pediatric patients affected by hematological malignancies

Pediatric patients affected by acute leukemia (AML, ALL or acute leukemia of ambiguous lineage) eligible for a T depleted allogeneic HSCT

Group Type: EXPERIMENTAL

Allogeneic T cell progenitors, cultured ex-vivo

Intervention Type: BIOLOGICAL

Injection of T cell progenitors at \[Day 4-Day 10\] after T cell depleted allogeneic HSCT

Interventions

Allogeneic T cell progenitors, cultured ex-vivo

Injection of T cell progenitors at \[Day 4-Day 10\] after T cell depleted allogeneic HSCT

Intervention Type: BIOLOGICAL

Other Intervention Names

SMART101

Eligibility Criteria

Inclusion Criteria

Group A (adults):

1. Adult patients affected by:

• Acute leukemia (AML, ALL) defined as:

• Acute Myeloid Leukemia (AML):

• High risk AML in CR1; any adverse genetic abnormality, secondary or therapy related AML excluding good risk genetic abnormalities
• Chemo-refractory relapse (MRD+)
• ≥ CR2
• Acute Lymphoblastic Leukemia (ALL):

• Chemo-refractory relapse (MRD+)
• High risk ALL in CR1; Philadelphia (like) or any poor risk feature
• ≥ CR2
• Acute leukemia of ambiguous lineage:

• ≥ CR1 with a minimal residual disease (MRD) <5% (flow cytometry, molecular and/or cytogenetics accepted)
• Myelodysplastic Syndrome (MDS) with least one of the following:

• Revised International Prognostic Scoring System risk score of intermediate or higher at the time of transplant evaluation.
• Life-threatening cytopenia.
• Karyotype or genomic changes that indicate high risk for progression to acute myelogenous leukemia, including abnormalities of chromosome 7 or 3, mutations of TP53, or complex or monosomal karyotype.
• Therapy related disease or disease evolving from other malignant processes.

2. Patient eligible for a T-depleted allogeneic HSCT

3. Age ≥ 18y and clinical condition compatible with allogeneic stem cell transplantation

4. Karnofsky index ≥ 70% prior to conditioning regimen

5. Patients with normal organ function prior to conditioning regimen

Group B (pediatrics):

1. Pediatric patients affected by acute leukemia defined as:

• Acute Myeloid Leukemia (AML):

• High risk AML in CR1; any adverse genetic abnormality, secondary or therapy related AML excluding good risk genetic abnormalities,
• Chemo-refractory relapse (MRD+)
• ≥ CR2
• Acute Lymphoblastic Leukemia (ALL):

• Chemo-refractory relapse (MRD+)
• High risk ALL in CR1; Philadelphia (like) or any poor risk feature
• ≥ CR2
• Acute leukemia of ambiguous lineage:

• ≥ CR1 with a minimal residual disease (MRD) <5% (flow cytometry, molecular and/or cytogenetics accepted)

2. Patient eligible for a T-depleted allogeneic HSCT

3. Age < 18y at the time of inclusion

4. Absence of a matched sibling donor (MSD)

5. Lansky ≥ 70% / Karnofsky performance status ≥ 70% prior to conditioning regimen

6. Patients with normal organ function prior to conditioning regimen

Exclusion Criteria

Groups A and B:

1. Use of an HLA matched Cord Blood (8/8 allele matched) or haploidentical donor

2. Prior therapy with allogeneic stem cell transplantation

3. Treatment with another cellular therapy within one month before inclusion

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Smart Immune SAS

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Jaap-Jan BOELENS, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Memorial Sloan Kettering Cancer Center (MSKCC)

Locations

Memorial Sloan Kettering Cancer Center (MSKCC)

New York, New York, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Frédéric LEHMANN, MD

Role: CONTACT

frederic.lehmann@smart-immune.com

+32 (0) 492 46 23 55

Laura SIMONS, MD, PhD

Role: CONTACT

laura.simons@smart-immune.com

Other Identifiers

SI101-01

Identifier Type: -

Identifier Source: org_study_id