Hematological MalignanciesPhase 1/2NCT04959903
Safety and Efficacy of SMART101 in Pediatric and Adult Patients With Hematological Malignancies After T Cell Depleted Allo-HSCT
A Phase 1/2 study testing Allogeneic T cell progenitors, cultured ex-vivo in people with Hematological Malignancies, run by Smart Immune SAS at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hematological Malignancies
- What is being tested?
- Allogeneic T cell progenitors, cultured ex-vivo
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Mar 2022to May 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Group A (adults):
- 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+)
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- ≥ 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.
- Patient eligible for a T-depleted allogeneic HSCT
- Age ≥ 18y and clinical condition compatible with allogeneic stem cell transplantation
- Karnofsky index ≥ 70% prior to conditioning regimen
- Patients with normal organ function prior to conditioning regimen
- Group B (pediatrics):
- 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)
- Patient eligible for a T-depleted allogeneic HSCT
- Age < 18y at the time of inclusion
- Absence of a matched sibling donor (MSD)
- Lansky ≥ 70% / Karnofsky performance status ≥ 70% prior to conditioning regimen
- Patients with normal organ function prior to conditioning regimen
Who cannot join
- Groups A and B:
- Use of an HLA matched Cord Blood (8/8 allele matched) or haploidentical donor
- Prior therapy with allogeneic stem cell transplantation
- Treatment with another cellular therapy within one month before inclusion
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Laura SIMONS, MD, PhD contact
United States1 site, 1 recruiting
- Recruiting
Memorial Sloan Kettering Cancer Center (MSKCC)
New York, New York, 10065
Jaap-Jan BOELENS, MD, PhD
Miguel-Angel PERALES, MD
About this study
Official title: 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
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
- Being tested
- Allogeneic T cell progenitors, cultured ex-vivo
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic T cell progenitors, cultured ex-vivoBiological
Also called SMART101
Injection of T cell progenitors at [Day 4-Day 10] after T cell depleted allogeneic HSCT
| Study group | Role | What they receive |
|---|---|---|
| Adult patients affected by hematological malignancies | Experimental | Adult patients affected by acute leukemia (AML, ALL or acute leukemia of ambiguous lineage) or myelodysplastic syndrome eligible for a T depleted allogeneic HSCT Receives: Biological: Allogeneic T cell progenitors, cultured ex-vivo |
| Pediatric patients affected by hematological malignancies | Experimental | Pediatric patients affected by acute leukemia (AML, ALL or acute leukemia of ambiguous lineage) eligible for a T depleted allogeneic HSCT Receives: Biological: Allogeneic T cell progenitors, cultured ex-vivo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cumulative incidence of grade III-IV GvHD
to evaluate the safety profile of the study drug
Measured: 100 days post-HSCT
Occurrence of adverse events related to SMART101
Number of adverse events and serious adverse events related to SMART101 tabulated for each dose and by age group to evaluate the safety profile of the study drug
Measured: 100 days post-HSCT
CD4+ T cell count
to evaluate the efficacy of the study drug
Measured: 100 days post-HSCT
Secondary outcomes (3)
T cell immune reconstitution
Time course of the T cell immune reconstitution, with a focus on naive CD4+ cells and total CD8+ cells
Measured: up to Month 12 post-HSCT
Cumulative incidence of infections
Measured: Day 90, and Months 6, 12 and 24 post-HSCT
Non-relapse mortality (NRM)
Measured: Day 90, and Months 6, 12 and 24 post-HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jul 2021
- Study start31 Mar 2022
- Primary completionAug 2025 (estimated)
- Study completionMay 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematological MalignanciesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Smart Immune SAS
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jaap-Jan BOELENS, MD, PhD, Principal investigator, Memorial Sloan Kettering Cancer Center (MSKCC)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04959903
- Sponsor's protocol ID
- SI101-01
- Status last verified
- Mar 2023
- First submitted
- 1 Jul 2021
- Last updated
- 6 Mar 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT04959903
- Acronym / sponsor ID
- SI101-01
- Conditions
- Hematological Malignancies
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Smart Immune SASIndustry (pharma, biotech, devices)
- Study start
- 31 Mar 2022 (actual)
- Primary completion
- Aug 2025 (estimated)
- Study completion
- May 2027 (estimated)
- First posted
- 13 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Adult patients affected by hematological malignancies | Experimental | Biological: Allogeneic T cell progenitors, cultured ex-vivoAdult patients affected by acute leukemia (AML, ALL or acute leukemia of ambiguous lineage) or myelodysplastic syndrome eligible for a T depleted allogeneic HSCT |
| Pediatric patients affected by hematological malignancies | Experimental | Biological: Allogeneic T cell progenitors, cultured ex-vivoPediatric patients affected by acute leukemia (AML, ALL or acute leukemia of ambiguous lineage) eligible for a T depleted allogeneic HSCT |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (39 listed)
- Adult patients affected by:
- Patient eligible for a T-depleted allogeneic HSCT
- Age ≥ 18y and clinical condition compatible with allogeneic stem cell transplantation
- Karnofsky index ≥ 70% prior to conditioning regimen
- Patients with normal organ function prior to conditioning regimen
Key exclusion criteria (4 listed)
- Use of an HLA matched Cord Blood (8/8 allele matched) or haploidentical donor
- Prior therapy with allogeneic stem cell transplantation
- Treatment with another cellular therapy within one month before inclusion
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Allogeneic T cell progenitors, cultured ex-vivoAlso: SMART101Injection of T cell progenitors at [Day 4-Day 10] after T cell depleted allogeneic HSCT | Biological | Adult patients affected by hematological malignancies, Pediatric patients affected by hematological malignancies |
Primary outcome
Cumulative incidence of grade III-IV GvHD
Time frame: 100 days post-HSCT
to evaluate the safety profile of the study drug
Occurrence of adverse events related to SMART101
Time frame: 100 days post-HSCT
Number of adverse events and serious adverse events related to SMART101 tabulated for each dose and by age group to evaluate the safety profile of the study drug
CD4+ T cell count
Time frame: 100 days post-HSCT
to evaluate the efficacy of the study drug
3 secondary outcomes
T cell immune reconstitution
Time frame: up to Month 12 post-HSCT
Cumulative incidence of infections
Time frame: Day 90, and Months 6, 12 and 24 post-HSCT
Non-relapse mortality (NRM)
Time frame: Day 90, and Months 6, 12 and 24 post-HSCT
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Mar 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Laura SIMONS, MD, PhD contact
Jaap-Jan BOELENS, MD, PhD principal investigator
Memorial Sloan Kettering Cancer Center (MSKCC)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Allogeneic T cell progenitors, cultured ex-vivo in people with Hematological Malignancies, run by Smart Immune SAS at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 39 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Frédéric LEHMANN, MD (+32 (0) 492 46 23 55, frederic.lehmann@smart-immune.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cumulative incidence of grade III-IV GvHD (time frame: 100 days post-HSCT). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Mar 2023; AzTrials last refreshed its copy on 8 Nov 2026.