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Allogeneic T cell progenitors, cultured ex-vivo

Phase 1

Hematological Malignancies | Monoclonal antibody | Other |MeiraGTx Holdings plc|Last Updated: Sep 25, 2023

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment76
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05768035Safety and Efficacy of SMART101 in Adult Patients With Hematological Malignancies After Haploidentical HSCT With Post-transplant CyclophosphamidePHASE1 RECRUITING 40Jun 6, 2023Jul 1, 2026Sep 25, 20234 France
NCT04959903Safety and Efficacy of SMART101 in Pediatric and Adult Patients With Hematological Malignancies After T Cell Depleted Allo-HSCTPHASE1 RECRUITING 36Mar 31, 2022May 1, 2027Mar 6, 20231 United States
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Study Endpoints
Primary Endpoints
Occurrence of Unexpected Unacceptable Toxicities (UUT) following the administration of SMART101.
14 days post SMART101 infusion

To evaluate the safety of SMART101.

CD4+ T cell count.
100 days post-HSCT

to evaluate the efficacy of the study drug

Cumulative incidence of grade III-IV GvHD
100 days post-HSCT

to evaluate the safety profile of the study drug

Occurrence of adverse events related to SMART101
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
100 days post-HSCT

to evaluate the efficacy of the study drug

Secondary Endpoints
Occurrence of adverse events (AEs)
up to 24 months post-HSCT
T cell immune reconstitution
up to 12 months post-HSCT
Cumulative incidence of infections
Day 100, and Months 6 and 12 post-HSCT
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Patients with acute leukemia or myelodysplastic syndrome and eligible for an haplo PT-Cy HSCTEXPERIMENTALSegment 1: 3 dose-level SMART101 cells/infusion 1. 1.5 x 106 CD7+ cells per kg of body weight 2. 4.5 x 106 CD7+ cells per kg of body weight 3. 9.0 x 106 CD7+ cells per kg of body weight Segment 2: 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.
Adult patients affected by hematological malignanciesEXPERIMENTALAdult 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 malignanciesEXPERIMENTALPediatric patients affected by acute leukemia (AML, ALL or acute leukemia of ambiguous lineage) eligible for a T depleted allogeneic HSCT
Interventions
NameTypeDescription
Allogeneic T cell progenitors, cultured ex-vivoBIOLOGICALInjection of T cell progenitors 6 days after haplo HSCT and 2 days after the last administration of cyclophosphamide
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites4

Main Inclusion Criteria: * Patients with AML, ALL or MDS eligible for an allogeneic HSCT with a haploidentical donor with post-transplant cyclophosphamide. * Patients must be ≥ 18 years of age at the time of signing the ICF. * Patients must have a Karnofsky index ≥ 70%. * Patients must have a left ...

Countries:FranceUnited States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04959903primaryCompletionDate: changed
LOWMay 26, 2026NCT05768035primaryCompletionDate: changed
LOWMay 24, 2026NCT04959903studyFirstPostDate: changed
LOWMay 24, 2026NCT05768035studyFirstPostDate: changed