Evaluating SMART101 for immune recovery in patients with blood cancers after stem cell transplant

A Phase I/II Study Evaluating the Safety and the Efficacy of SMART101 Injection to Accelerate Immune Reconstitution After T Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric and Adult Patients With Hematological Malignancies

This study is testing if a new treatment called SMART101 can help improve immune recovery in kids and adults who have had a stem cell transplant for blood cancers.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 36 (estimated)
• Sex: All
• Sponsor: Smart Immune SAS (Industry-sponsored)
• Locations: 1 site (New York, New York)
• Trial ID: NCT04959903 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety and efficacy of SMART101, a human T lymphoid progenitor injection, to enhance immune recovery in both pediatric and adult patients who have undergone T cell depleted allogeneic hematopoietic stem cell transplantation (HSCT) due to hematological malignancies. The trial will involve administering cultured ex-vivo T cell progenitors to participants and monitoring their immune response and overall health outcomes. It is designed to provide insights into how this innovative treatment can improve recovery times and reduce complications associated with HSCT.

Who should consider this trial

Good fit: Ideal candidates include adult and pediatric patients with high-risk acute leukemia, myelodysplastic syndrome, or other hematological malignancies who are undergoing T cell depleted HSCT.

Not a fit: Patients with low-risk hematological malignancies or those not undergoing HSCT may not benefit from this treatment.

Why it matters

Potential benefit: If successful, this treatment could significantly improve immune reconstitution and overall recovery for patients undergoing stem cell transplants for blood cancers.

How similar studies have performed: While the specific approach of using SMART101 is novel, similar studies involving T cell progenitors have shown promise in enhancing immune recovery post-transplant.

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

Where this trial is running

New York, New York

• Memorial Sloan Kettering Cancer Center (MSKCC) — New York, New York, United States (Recruiting)

Study contacts

• Principal investigator: Jaap-Jan BOELENS, MD, PhD — Memorial Sloan Kettering Cancer Center (MSKCC)
• Study coordinator: Frédéric LEHMANN, MD

Conditions Hematological Malignancies