Brief Summary
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.
Brief Title
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)
Detailed Description
Study duration per participant is 2.5 years.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Acute Lymphocytic Leukaemia
Acute Myeloid Leukaemia Refractory
Myelodysplastic Syndromes
Blastic Plasmacytoid Dendritic Cell Neoplasia
Eligibility Criteria
Inclusion Criteria:
* Participant must be at least 1 year (for France: 2 years) old at the time the trial participant or legal guardian signs the informed consent form and will be assigned as follows:
* Adult arm: aged at least 18 years old.
* Pediatric arm: aged 1 (for France: 2 years) to less than 18 years old.
* Adult and Pediatric Arms: Escalation and Expansion/Optimization Cohorts A1, A2, C, D: Confirmed diagnosis of primary or secondary AML (any subtype) according to World Health Organization (WHO) 2022 classification. Participants with AML must meet one of the following criteria, a), b), c) or d) and are limited to those with no available (or are ineligible) therapy with known clinical benefit.
a) Primary Induction Failure (PIF) AML, defined as disease refractory to one of the following, i or ii.
i) An intensive induction attempt, per institution. Induction attempts include high-dose and/or standard-dose cytarabine ± an anthracyclines/anthracenedione ± an anti-metabolite, with or without growth factor or targeted therapy containing regimens.
ii) For adults who are age 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy; PIF is defined as AML refractory to one of the following less intensive regimens, 1 or 2:
1. 4 cycles of hypomethylating agents (HMA) or
2. 2 cycles HMA + venetoclax b) Early relapse (ER) AML, defined as AML in relapse with CR, CRh or CRi duration less than 6 months on prior induction treatment c) Leukemia in first or higher relapse d) For participants aged 1 (for France: 2 years) to less than 18 years old, primary induction failure is defined as disease refractory after two cycles of induction therapy.
* Adult Arm (Escalation and Expansion/Optimization Cohorts B and Japan Cohort C only): Confirmed diagnosis of MDS, meeting the following criteria:
1. intermediate or high-risk category as per a Revised International Prognostic Scoring System (IPSS-R) AND
2. confirmed CD123 + expression status determined by local institutional standards AND
3. limited to those with no available (or are ineligible) therapy with known clinical benefit.
* Pediatric arms escalation part and Japan Cohort C only: Confirmed diagnosis of CD123+ BALL without extramedullary lesions that have no available (or are ineligible) therapy with known clinical benefit. Participants with non-CNS chloromatous disease are not allowed in the study.
* Body weight at least 10 kg.
* Pediatric arm and escalation part only: Confirmed diagnosis of BPDCN according to World Health Organization (WHO) 2022 classification, who have relapsed or refractory disease with no available (or are ineligible) therapy with known clinical benefit.
* Japan participants (Cohort C): Participant must be at least 18 years old at the time the trial participant signs the informed consent form
Exclusion Criteria:
* Eastern Cooperative Oncology Group (ECOG) performance status greater than 2 (at least 18 years-old). Karnofsky Scale (16 to 17 years-old) less than 50% or Lansky Scale (less than 16 years-old) less than 50%.
* Ongoing or recent (within 5 years) evidence of significant autoimmune disease that requires or required treatment with systemic immunosuppressive treatments, which may suggest a risk for immune-related adverse events. The following are not exclusionary: vitiligo, childhood asthma that has resolved, residual hypothyroidism that required only hormone replacement or psoriasis that does not require systemic treatment.
* History of an invasive malignancy within the last 3 years prior to first IMP administration that requires active therapy (adjuvant hormonal therapy is allowed) other than the one treated in this study.
* Evidence of active central nervous system leukemia at the time of enrollment as evidenced by cytology or pathology. Except for participants aged 1 (for France: 2 years) to less than 18 years, central nervous system 1 disease (CNS1) and CNS2 disease are allowed.
* Known acquired immunodeficiency syndrome (AIDS-related illnesses) or human immunodeficiency virus (HIV) disease requiring antiretroviral treatment, or having active hepatitis B or C infection, or severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection.
* Prior treatment with an anti-CD123-directed agent (except for participants with BPDCN in the pediatric arm).
* Prior HSCT with relapse beyond 3 months or prior CAR-T therapy in B-ALL with relapse beyond 2 months may be included only if off immunosuppression for a minimum of 4 weeks and no evidence of GVHD.
* Receiving at the time of first investigational medicinal product (IMP) administration corticosteroid as a concomitant medication with corticosteroid dose more than 10 mg/day of oral prednisone or the equivalent.
* AML, BPDCN, or HR-MDS participants with prior treatment with cellular therapy, eg, chimeric antigen receptor T cell (CAR-T) or chimeric antigen receptor NK cell (CAR-NK). Prior CAR-T therapy is allowed for participants with B-ALL.
* Concurrent treatment with other investigational drugs.
* Pregnant and breast-feeding women.
* History of solid organ transplant, including corneal transplant.
* Average QTc (using the Fridericia correction calculation) greater than 470 millisecond (msec) at screening.
* Pediatric arm only: Participants with known inherited bone marrow failure syndromes (e.g., bloom syndrome, ataxia-telangiectasia, Fanconi anemia, Kostmann syndrome, Shwachman syndrome). Participants with Down syndrome with adequate organ function as per Investigator discretion are allowed to participate in the study.
* Adult arm Expansion/Optimization- Participants with MDS evolving from a pre-existing myeloproliferative neoplasm (MPN), MDS/MPN including chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (aCML), unclassifiable MDS/MPN and therapy-related MDS (t-MDS).
* Confirmed diagnosis of acute promyelocytic leukemia (APL) or juvenile myelomonocytic leukemia (JMML) according to WHO 2022 classification.
The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
* Participant must be at least 1 year (for France: 2 years) old at the time the trial participant or legal guardian signs the informed consent form and will be assigned as follows:
* Adult arm: aged at least 18 years old.
* Pediatric arm: aged 1 (for France: 2 years) to less than 18 years old.
* Adult and Pediatric Arms: Escalation and Expansion/Optimization Cohorts A1, A2, C, D: Confirmed diagnosis of primary or secondary AML (any subtype) according to World Health Organization (WHO) 2022 classification. Participants with AML must meet one of the following criteria, a), b), c) or d) and are limited to those with no available (or are ineligible) therapy with known clinical benefit.
a) Primary Induction Failure (PIF) AML, defined as disease refractory to one of the following, i or ii.
i) An intensive induction attempt, per institution. Induction attempts include high-dose and/or standard-dose cytarabine ± an anthracyclines/anthracenedione ± an anti-metabolite, with or without growth factor or targeted therapy containing regimens.
ii) For adults who are age 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy; PIF is defined as AML refractory to one of the following less intensive regimens, 1 or 2:
1. 4 cycles of hypomethylating agents (HMA) or
2. 2 cycles HMA + venetoclax b) Early relapse (ER) AML, defined as AML in relapse with CR, CRh or CRi duration less than 6 months on prior induction treatment c) Leukemia in first or higher relapse d) For participants aged 1 (for France: 2 years) to less than 18 years old, primary induction failure is defined as disease refractory after two cycles of induction therapy.
* Adult Arm (Escalation and Expansion/Optimization Cohorts B and Japan Cohort C only): Confirmed diagnosis of MDS, meeting the following criteria:
1. intermediate or high-risk category as per a Revised International Prognostic Scoring System (IPSS-R) AND
2. confirmed CD123 + expression status determined by local institutional standards AND
3. limited to those with no available (or are ineligible) therapy with known clinical benefit.
* Pediatric arms escalation part and Japan Cohort C only: Confirmed diagnosis of CD123+ BALL without extramedullary lesions that have no available (or are ineligible) therapy with known clinical benefit. Participants with non-CNS chloromatous disease are not allowed in the study.
* Body weight at least 10 kg.
* Pediatric arm and escalation part only: Confirmed diagnosis of BPDCN according to World Health Organization (WHO) 2022 classification, who have relapsed or refractory disease with no available (or are ineligible) therapy with known clinical benefit.
* Japan participants (Cohort C): Participant must be at least 18 years old at the time the trial participant signs the informed consent form
Exclusion Criteria:
* Eastern Cooperative Oncology Group (ECOG) performance status greater than 2 (at least 18 years-old). Karnofsky Scale (16 to 17 years-old) less than 50% or Lansky Scale (less than 16 years-old) less than 50%.
* Ongoing or recent (within 5 years) evidence of significant autoimmune disease that requires or required treatment with systemic immunosuppressive treatments, which may suggest a risk for immune-related adverse events. The following are not exclusionary: vitiligo, childhood asthma that has resolved, residual hypothyroidism that required only hormone replacement or psoriasis that does not require systemic treatment.
* History of an invasive malignancy within the last 3 years prior to first IMP administration that requires active therapy (adjuvant hormonal therapy is allowed) other than the one treated in this study.
* Evidence of active central nervous system leukemia at the time of enrollment as evidenced by cytology or pathology. Except for participants aged 1 (for France: 2 years) to less than 18 years, central nervous system 1 disease (CNS1) and CNS2 disease are allowed.
* Known acquired immunodeficiency syndrome (AIDS-related illnesses) or human immunodeficiency virus (HIV) disease requiring antiretroviral treatment, or having active hepatitis B or C infection, or severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection.
* Prior treatment with an anti-CD123-directed agent (except for participants with BPDCN in the pediatric arm).
* Prior HSCT with relapse beyond 3 months or prior CAR-T therapy in B-ALL with relapse beyond 2 months may be included only if off immunosuppression for a minimum of 4 weeks and no evidence of GVHD.
* Receiving at the time of first investigational medicinal product (IMP) administration corticosteroid as a concomitant medication with corticosteroid dose more than 10 mg/day of oral prednisone or the equivalent.
* AML, BPDCN, or HR-MDS participants with prior treatment with cellular therapy, eg, chimeric antigen receptor T cell (CAR-T) or chimeric antigen receptor NK cell (CAR-NK). Prior CAR-T therapy is allowed for participants with B-ALL.
* Concurrent treatment with other investigational drugs.
* Pregnant and breast-feeding women.
* History of solid organ transplant, including corneal transplant.
* Average QTc (using the Fridericia correction calculation) greater than 470 millisecond (msec) at screening.
* Pediatric arm only: Participants with known inherited bone marrow failure syndromes (e.g., bloom syndrome, ataxia-telangiectasia, Fanconi anemia, Kostmann syndrome, Shwachman syndrome). Participants with Down syndrome with adequate organ function as per Investigator discretion are allowed to participate in the study.
* Adult arm Expansion/Optimization- Participants with MDS evolving from a pre-existing myeloproliferative neoplasm (MPN), MDS/MPN including chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (aCML), unclassifiable MDS/MPN and therapy-related MDS (t-MDS).
* Confirmed diagnosis of acute promyelocytic leukemia (APL) or juvenile myelomonocytic leukemia (JMML) according to WHO 2022 classification.
The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Inclusion Criteria
Inclusion Criteria:
* Participant must be at least 1 year (for France: 2 years) old at the time the trial participant or legal guardian signs the informed consent form and will be assigned as follows:
* Adult arm: aged at least 18 years old.
* Pediatric arm: aged 1 (for France: 2 years) to less than 18 years old.
* Adult and Pediatric Arms: Escalation and Expansion/Optimization Cohorts A1, A2, C, D: Confirmed diagnosis of primary or secondary AML (any subtype) according to World Health Organization (WHO) 2022 classification. Participants with AML must meet one of the following criteria, a), b), c) or d) and are limited to those with no available (or are ineligible) therapy with known clinical benefit.
a) Primary Induction Failure (PIF) AML, defined as disease refractory to one of the following, i or ii.
i) An intensive induction attempt, per institution. Induction attempts include high-dose and/or standard-dose cytarabine ± an anthracyclines/anthracenedione ± an anti-metabolite, with or without growth factor or targeted therapy containing regimens.
ii) For adults who are age 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy; PIF is defined as AML refractory to one of the following less intensive regimens, 1 or 2:
1. 4 cycles of hypomethylating agents (HMA) or
2. 2 cycles HMA + venetoclax b) Early relapse (ER) AML, defined as AML in relapse with CR, CRh or CRi duration less than 6 months on prior induction treatment c) Leukemia in first or higher relapse d) For participants aged 1 (for France: 2 years) to less than 18 years old, primary induction failure is defined as disease refractory after two cycles of induction therapy.
* Adult Arm (Escalation and Expansion/Optimization Cohorts B and Japan Cohort C only): Confirmed diagnosis of MDS, meeting the following criteria:
1. intermediate or high-risk category as per a Revised International Prognostic Scoring System (IPSS-R) AND
2. confirmed CD123 + expression status determined by local institutional standards AND
3. limited to those with no available (or are ineligible) therapy with known clinical benefit.
* Pediatric arms escalation part and Japan Cohort C only: Confirmed diagnosis of CD123+ BALL without extramedullary lesions that have no available (or are ineligible) therapy with known clinical benefit. Participants with non-CNS chloromatous disease are not allowed in the study.
* Body weight at least 10 kg.
* Pediatric arm and escalation part only: Confirmed diagnosis of BPDCN according to World Health Organization (WHO) 2022 classification, who have relapsed or refractory disease with no available (or are ineligible) therapy with known clinical benefit.
* Japan participants (Cohort C): Participant must be at least 18 years old at the time the trial participant signs the informed consent form
* Participant must be at least 1 year (for France: 2 years) old at the time the trial participant or legal guardian signs the informed consent form and will be assigned as follows:
* Adult arm: aged at least 18 years old.
* Pediatric arm: aged 1 (for France: 2 years) to less than 18 years old.
* Adult and Pediatric Arms: Escalation and Expansion/Optimization Cohorts A1, A2, C, D: Confirmed diagnosis of primary or secondary AML (any subtype) according to World Health Organization (WHO) 2022 classification. Participants with AML must meet one of the following criteria, a), b), c) or d) and are limited to those with no available (or are ineligible) therapy with known clinical benefit.
a) Primary Induction Failure (PIF) AML, defined as disease refractory to one of the following, i or ii.
i) An intensive induction attempt, per institution. Induction attempts include high-dose and/or standard-dose cytarabine ± an anthracyclines/anthracenedione ± an anti-metabolite, with or without growth factor or targeted therapy containing regimens.
ii) For adults who are age 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy; PIF is defined as AML refractory to one of the following less intensive regimens, 1 or 2:
1. 4 cycles of hypomethylating agents (HMA) or
2. 2 cycles HMA + venetoclax b) Early relapse (ER) AML, defined as AML in relapse with CR, CRh or CRi duration less than 6 months on prior induction treatment c) Leukemia in first or higher relapse d) For participants aged 1 (for France: 2 years) to less than 18 years old, primary induction failure is defined as disease refractory after two cycles of induction therapy.
* Adult Arm (Escalation and Expansion/Optimization Cohorts B and Japan Cohort C only): Confirmed diagnosis of MDS, meeting the following criteria:
1. intermediate or high-risk category as per a Revised International Prognostic Scoring System (IPSS-R) AND
2. confirmed CD123 + expression status determined by local institutional standards AND
3. limited to those with no available (or are ineligible) therapy with known clinical benefit.
* Pediatric arms escalation part and Japan Cohort C only: Confirmed diagnosis of CD123+ BALL without extramedullary lesions that have no available (or are ineligible) therapy with known clinical benefit. Participants with non-CNS chloromatous disease are not allowed in the study.
* Body weight at least 10 kg.
* Pediatric arm and escalation part only: Confirmed diagnosis of BPDCN according to World Health Organization (WHO) 2022 classification, who have relapsed or refractory disease with no available (or are ineligible) therapy with known clinical benefit.
* Japan participants (Cohort C): Participant must be at least 18 years old at the time the trial participant signs the informed consent form
Gender
All
Gender Based
false
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Minimum Age
1 Year
NCT Id
NCT05086315
Org Class
Industry
Org Full Name
Sanofi
Org Study Id
TCD17197
Overall Status
Terminated
Phases
Phase 1
Phase 2
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
An Open-label, First-in-human, Dose-escalation/Expansion Study of SAR443579 Administered as Single Agent by Intravenous Infusion in Adult and Pediatric 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)
Primary Outcomes
Outcome Description
DLTs encompass both hematologic and nonhematologic toxicities, prespecified adverse reactions observed post-administration of SAR443579 and assessed by both the investigator and the sponsor.
Outcome Measure
Escalation Part: Incidence of dose-limiting toxicity (DLT)
Outcome Time Frame
Day 1 to Day 28
Outcome Description
DLTs encompass both hematologic and nonhematologic toxicities, prespecified adverse reactions observed post-administration of SAR443579 and assessed by both the investigator and the sponsor.
Outcome Measure
Japan Cohort C: Incidence of DLT in Japanese participants
Outcome Time Frame
Day 1 to Day 28
Outcome Description
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.
Outcome Measure
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
Outcome Time Frame
Up to 6 months
Outcome Description
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.
Outcome Measure
Expansion/Optimization part (Cohort B), MDS: Overall response rate (CR + CR equivalent + PR + CRL + CRh + HI) according to the IWG 2023 MDS response criteria
Outcome Time Frame
Up to 6 months
Secondary Ids
Secondary Id
U1111-1266-7399
Secondary Id
2023-508357-58
Secondary Id
2021-004287-98
Secondary Outcomes
Outcome Description
Recommended dose for expansion (RDE) of SAR443579.
Outcome Time Frame
Up to 12 months
Outcome Measure
Expansion/Optimization part - Cohorts A, B and D: Recommended dose for expansion (RDE)
Outcome Description
Number of participants with treatment-emergent adverse events (TEAEs).
Outcome Time Frame
Up to 30 months
Outcome Measure
Escalation and Expansion/Optimization parts - Cohorts A, B, C and D: Number of participants with TEAEs
Outcome Description
Concentration observed just before treatment administration during repeated dosing (Ctrough).
Outcome Time Frame
Cycle 1 from Day 1 to Day 28
Outcome Measure
Cohorts A, B, C and D: Ctrough at Cycle 1
Outcome Description
Percentage of participants with anti-drug antibody (ADA) against SAR443579.
Outcome Time Frame
Up to 30 months
Outcome Measure
Cohorts A, B, C and D: Incidence of ADA
Outcome Description
Measure of clinical response to treatment.
Outcome Time Frame
Up to 6 months
Outcome Measure
Escalation and Expansion/Optimization parts - Japan Cohort C, AML: Rate of CR + CRh + CRi per AML 2003 modified IWG response criteria
Outcome Description
Measure of clinical response to treatment.
Outcome Time Frame
Up to 6 months
Outcome Measure
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
Outcome Description
Measure of clinical response to treatment.
Outcome Time Frame
Up to 6 months
Outcome Measure
Escalation and Expansion/Optimization parts - Japan Cohort C, B-ALL: Rate of CR + CRh + CRi as defined by National Comprehensive Cancer Network (NCCN)
Outcome Description
Measure of clinical response to treatment.
Outcome Time Frame
Up to 6 months
Outcome Measure
Expansion/Optimization part - Cohorts A and D: Overall response rate (ORR)
Outcome Description
Measure the length of clinical response to treatment.
Outcome Time Frame
Up to 30 months
Outcome Measure
Expansion/Optimization part - Cohorts A and D: Duration of CR + CRh + CRi (Duration of CRc)
Outcome Description
Measure the length of clinical response to treatment.
Outcome Time Frame
Up to 30 months
Outcome Measure
Expansion/Optimization part - Cohorts A and D: Duration of CR + CRi + CRh + PR + MLFS (Duration of overall response rate)
Outcome Description
Measure of clinical response to treatment.
Outcome Time Frame
Up to 6 months
Outcome Measure
Expansion/Optimization part - Cohorts A and D: Alternative CR rate
Outcome Description
Measure the length of clinical response to treatment.
Outcome Time Frame
Up to 30 months
Outcome Measure
Expansion/Optimization part - Cohorts A and D: Duration of CR + CRh (Duration of alternative CR)
Outcome Description
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.
Outcome Time Frame
Up to 6 months
Outcome Measure
Expansion/Optimization part - Cohorts A and D: Event-free survival (EFS)
Outcome Description
OS is defined as time interval from the first day of treatment assignment to death from any cause.
Outcome Time Frame
Up to 30 months
Outcome Measure
Expansion/Optimization part - Cohorts A and D: Overall survival (OS)
Outcome Description
Rate of HSCT procedures immediately following study treatment administration but prior to subsequent therapy for treatment of AML.
Outcome Time Frame
Up to 30 months
Outcome Measure
Expansion/Optimization part - Cohorts A and D: Rate of hematopoietic stem cell transplantation (HSCT)
Outcome Description
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.
Outcome Time Frame
Up to 6 months
Outcome Measure
Expansion/Optimization part - Cohorts A and D: Time to treatment failure (TTF)
Outcome Description
Rate of conversion from transfusion dependence during 56-day post-baseline period.
Outcome Time Frame
Day 0 to Day 56
Outcome Measure
Expansion/Optimization part - Cohorts A and D: Rate of conversion from transfusion dependence
Outcome Description
Rate of participants who are transfusion independent at baseline and remain independent during 56-day postbaseline period.
Outcome Time Frame
Day 0 to Day 56
Outcome Measure
Expansion/Optimization part - Cohorts A and D: Rate of participants who are transfusion independent at baseline and remain independent during 56-day postbaseline period
Outcome Description
Alternative CR rate defined as proportion of participants with CR, CR equivalent, CRuni, CRbi, and CRh.
Outcome Time Frame
Up to 6 months
Outcome Measure
Expansion/Optimization part - Cohort B: Alternative CR rate
Outcome Description
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.
Outcome Time Frame
Up to 30 months
Outcome Measure
Expansion/Optimization part - Cohort B: Duration of ORR
Outcome Description
EFS is defined as the time interval from the first day of treatment assignment to the date of protocol specified events.
Outcome Time Frame
Up to 30 months
Outcome Measure
Expansion/Optimization part - Cohort B: Event-free survival (EFS)
Outcome Description
OS is defined as time interval from the first day of treatment assignment to death from any cause.
Outcome Time Frame
Up to 30 months
Outcome Measure
Expansion/Optimization part - Cohort B: Overall survival (OS)
Outcome Description
Rate of HSCT procedures immediately following study treatment administration but prior to subsequent therapy.
Outcome Time Frame
Up to 30 months
Outcome Measure
Expansion/Optimization part - Cohort B: Rate of hematopoietic stem cell transplantation (HSCT)
Outcome Description
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.
Outcome Time Frame
Up to 30 months
Outcome Measure
Expansion/Optimization part - Cohort B: Time to treatment failure (TTF)
Outcome Description
Measure the length of clinical response to treatment.
Outcome Time Frame
Up to 30 months
Outcome Measure
Expansion/Optimization part - Cohort B: Duration of alternative CR (CR + CR equivalent + CRL + CRh)
Outcome Description
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.
Outcome Time Frame
Up to 30 months
Outcome Measure
Expansion/Optimization part - Cohort B: Progression free survival (PFS)
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
1
Investigators
Investigator Type
Principal Investigator
Investigator Name
Ioannis Mantzaris
Investigator Email
IMANTZAR@montefiore.org