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First-in-human Study of SAR443579 Infusion in Male and Female Children and Adult Participants With Relapsed or Refractory Acute Myeloid Leukemia (R/R AML), B-cell Acute Lymphoblastic Leukemia (B-ALL), High Risk-myelodysplasia (HR-MDS), or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
This is an open-label, multicenter, Phase 1/Phase 2, dose escalation and dose expansion study to evaluate the safety, pharmacokinetics, pharmacodynamics and anti-leukemic activity of SAR443579 in various hematological malignancies.
Study details:
Study duration per participant is 2. 5 years.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 1 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2021-12-08
Primary completion: 2025-12-11
Study completion finish: 2030-10-15
Study type
TREATMENT
Phase
PHASE1
PHASE2
Trial ID
NCT05086315
Intervention or treatment
DRUG: SAR443579
Conditions
- • Myelodysplastic Syndromes
- • Acute Lymphocytic Leukaemia
- • Acute Myeloid Leukaemia Refractory
- • Blastic Plasmacytoid Dendritic Cell Neoplasia
Find a site
Closest Location:
Investigational Site Number :0360002
Research sites nearby
Select from list below to view details:
Investigational Site Number :0360002
Melbourne, Victoria, Australia
Investigational Site Number :0360001
Melbourne, Victoria, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Participant Group/Arm | Intervention/Treatment |
---|---|
EXPERIMENTAL: SAR443579
| DRUG: SAR443579
|
What is the study measuring?
Primary outcome
Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
---|---|---|
Escalation Part: Incidence of dose-limiting toxicity (DLT) | DLTs encompass both hematologic and nonhematologic toxicities, prespecified adverse reactions observed post-administration of SAR443579 and assessed by both the investigator and the sponsor. | Day 1 to Day 28 |
Japan Cohort C: Incidence of DLT in Japanese participants | DLTs encompass both hematologic and nonhematologic toxicities, prespecified adverse reactions observed post-administration of SAR443579 and assessed by both the investigator and the sponsor. | Day 1 to Day 28 |
Expansion/Optimization part (Cohorts A1, A2 & D), AML: Proportion of participants who have a CR + CRh + CRi according to the modified AML IWG 2003 criteria | Measure of clinical response to treatment: Proportion of participants who have a complete remission (CR) + CR with partial hematologic recovery (CRh) + CR with incomplete hematologic recovery (CRi) according to the modified acute myeloid leukemia (AML) IWG 2003 criteria. | Up to 6 months |
Expansion/Optimization part (Cohort B), MDS: Overall response rate (CR + CR equivalent + PR + CRL + CRh + HI) according to the IWG 2023 MDS response criteria | Measure of clinical response to treatment: Overall response rate (CR + CR equivalent + partial remission (PR) + CR with limited count recovery (CRL) + CRh + hematologic improvement (HI)) according to the International Working Group (IWG) 2023 myelodysplasia (MDS) response criteria. | Up to 6 months |
Secondary outcome
Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
---|---|---|
Expansion/Optimization part - Cohorts A, B and D: Recommended dose for expansion (RDE) | Recommended dose for expansion (RDE) of SAR443579. | Up to 12 months |
Escalation and Expansion/Optimization parts - Cohorts A, B, C and D: Number of participants with TEAEs | Number of participants with treatment-emergent adverse events (TEAEs). | Up to 30 months |
Escalation and Expansion/Optimization parts - Cohorts A, B, C and D: Ctrough | Concentration observed just before treatment administration during repeated dosing (Ctrough). | Day 1 to end of trial (maximum up to 30 months) |
Escalation and Expansion/Optimization parts - Cohorts A, B, C and D: Incidence of ADA | Percentage of participants with anti-drug antibody (ADA). | Up to 30 months |
Escalation and Expansion/Optimization parts - Japan Cohort C, AML: Rate of CR + CRh + CRi per AML 2003 modified IWG response criteria | Measure of clinical response to treatment. | Up to 6 months |
Escalation and expansion/Optimization parts - Japan Cohort C, MDS: CR rate and ORR rate per IWG 2023 MDS response criteria for escalation part and ORR rate per IWG 2023 | Measure of clinical response to treatment. | Up to 6 months |
Escalation and Expansion/Optimization parts - Japan Cohort C, B-ALL: Rate of CR + CRh + CRi as defined by National Comprehensive Cancer Network (NCCN) | Measure of clinical response to treatment. | Up to 6 months |
Expansion/Optimization part - Cohorts A and D: Overall response rate (ORR) | Measure of clinical response to treatment. | Up to 6 months |
Expansion/Optimization part - Cohorts A and D: Duration of CR + CRh + CRi (Duration of CRc) | Measure the length of clinical response to treatment. | Up to 30 months |
Expansion/Optimization part - Cohorts A and D: Duration of CR + CRi + CRh + PR + MLFS (Duration of overall response rate) | Measure the length of clinical response to treatment. | Up to 30 months |
Expansion/Optimization part - Cohorts A and D: Alternative CR rate | Measure of clinical response to treatment. | Up to 6 months |
Expansion/Optimization part - Cohorts A and D: Duration of CR + CRh (Duration of alternative CR) | Measure the length of clinical response to treatment. | Up to 30 months |
Expansion/Optimization part - Cohorts A and D: Event-free survival (EFS) | EFS is defined as the time interval from the first day of treatment assignment to the date of earliest evidence of relapse, treatment failure, or death. | Up to 6 months |
Expansion/Optimization part - Cohorts A and D: Overall survival (OS) | OS is defined as time interval from the first day of treatment assignment to death from any cause. | Up to 30 months |
Expansion/Optimization part - Cohorts A and D: Rate of hematopoietic stem cell transplantation (HSCT) | Rate of HSCT procedures immediately following study treatment administration but prior to subsequent therapy for treatment of AML. | Up to 30 months |
Expansion/Optimization part - Cohorts A and D: Time to treatment failure (TTF) | TTF is defined as the time from first day of treatment assignment to discontinuation for any reason excluding remission, eg, relapsed disease, refractory disease, unacceptable AE, participant preference or death. | Up to 6 months |
Expansion/Optimization part - Cohorts A and D: Rate of conversion from transfusion dependence | Rate of conversion from transfusion dependence during 56-day post-baseline period. | Day 0 to Day 56 |
Expansion/Optimization part - Cohorts A and D: Rate of participants who are transfusion independent at baseline and remain independent during 56-day postbaseline period | Rate of participants who are transfusion independent at baseline and remain independent during 56-day postbaseline period. | Day 0 to Day 56 |
Expansion/Optimization part - Cohort B: Alternative CR rate | Alternative CR rate defined as proportion of participants with CR, CR equivalent, CRuni, CRbi, and CRh. | Up to 6 months |
Expansion/Optimization part - Cohort B: Duration of ORR | The time interval from the first documented evidence of CR, CR equivalent, CRL, CRh, PR or HI to PD or relapse from CR, CR equivalent, CRL, CRh, PR or HI as per 2023 IWG recommendations or death due to any cause, whichever comes first. | Up to 30 months |
Expansion/Optimization part - Cohort B: Event-free survival (EFS) | EFS is defined as the time interval from the first day of treatment assignment to the date of protocol specified events. | Up to 30 months |
Expansion/Optimization part - Cohort B: Overall survival (OS) | OS is defined as time interval from the first day of treatment assignment to death from any cause. | Up to 30 months |
Expansion/Optimization part - Cohort B: Rate of hematopoietic stem cell transplantation (HSCT) | Rate of HSCT procedures immediately following study treatment administration but prior to subsequent therapy. | Up to 30 months |
Expansion/Optimization part - Cohort B: Time to treatment failure (TTF) | TTF is defined as the time interval from first day of treatment assignment to discontinuation for any reason excluding remission, eg, relapsed disease, disease progression, unacceptable AE, participant preference or death. | Up to 30 months |
Expansion/Optimization part - Cohort B: Duration of alternative CR (CR + CR equivalent + CRL + CRh) | Measure the length of clinical response to treatment. | Up to 30 months |
Expansion/Optimization part - Cohort B: Progression free survival (PFS) | The time interval from the first day of treatment assignment to the date of PD, relapse from CR (or CR equivalent), PR, CRL, CRh, or HI, death due to any cause, whichever comes first. | Up to 30 months |
Frequently Asked Questions
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