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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05086315
Registration number
NCT05086315
Ethics application status
Date submitted
19/10/2021
Date registered
20/10/2021
Date last updated
29/05/2024
Titles & IDs
Public 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)
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Scientific 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)
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Secondary ID [1]
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U1111-1266-7399
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Secondary ID [2]
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TCD17197
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Acute Lymphocytic Leukaemia
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Acute Myeloid Leukaemia Refractory
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Myelodysplastic Syndromes
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Blastic Plasmacytoid Dendritic Cell Neoplasia
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Condition category
Condition code
Cancer
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Leukaemia - Acute leukaemia
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Cancer
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Leukaemia - Chronic leukaemia
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Cancer
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Children's - Leukaemia & Lymphoma
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Cancer
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Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma
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Cancer
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Lymphoma (non Hodgkin's lymphoma) - Low grade lymphoma
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Blood
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Haematological diseases
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Blood
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Other blood disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - SAR443579
Experimental: SAR443579 - Dose Escalation: SAR443579 administered intravenously at escalating dose levels.
Dose Expansion: SAR443579 administered intravenously at the recommended dose and schedule determined from the dose escalation.
Treatment: Drugs: SAR443579
Powder for solution for infusion; by IV infusion
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence of dose-limiting toxicity (DLT) (Escalation Part)
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Assessment method [1]
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Timepoint [1]
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Day 1 to Day 28
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Primary outcome [2]
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Proportion of participants who have a CR (Complete Remission) + CRi (Complete Remission with Incomplete Hematological Recovery) (Expansion Part)
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Assessment method [2]
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Timepoint [2]
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Up to 6 months
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Secondary outcome [1]
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Recommended Dose for Expansion (RDE)
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Assessment method [1]
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Timepoint [1]
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Up to 12 months
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Secondary outcome [2]
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Number of participants with treatment-emergent adverse events (TEAEs) (Escalation and Expansion Parts)
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Assessment method [2]
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Timepoint [2]
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Up to 30 months
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Secondary outcome [3]
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Cmax: Maximum observed concentration
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Assessment method [3]
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Timepoint [3]
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Day 1 to end of trial (maximum up to 30 months)
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Secondary outcome [4]
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Incidence of anti-drug antibody (ADA) (Escalation and Expansion Parts)
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Assessment method [4]
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Timepoint [4]
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Up to 30 months
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Secondary outcome [5]
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Anti-leukemic activity as define by International Working Group (IWG) for AML (modified) and MDS, or National Comprehensive Cancer Network (NCCN) for B-ALL (Escalation Part)
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Assessment method [5]
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Timepoint [5]
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Up to 6 months
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Secondary outcome [6]
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Proportion of participants with CR + complete remission with partial hematological recovery (CRh) (Expansion Part)
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Assessment method [6]
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Timepoint [6]
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Up to 6 months
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Secondary outcome [7]
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Rate of CR + CRh + CRi + morphological leukemia-free state (MLFS) (Expansion Part)
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Assessment method [7]
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Timepoint [7]
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Up to 6 months
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Secondary outcome [8]
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Time interval from first documented evidence of Composite Complete Remission (CRc: (CR or CRi)) until disease relapse as per modified IWG or death due to any cause, whichever comes first (Expansion Part)
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Assessment method [8]
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Timepoint [8]
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Up to 30 months
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Secondary outcome [9]
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Time from the first documented evidence of CR or CRh until disease relapse or death due to any cause, whichever comes first (Expansion Part)
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Assessment method [9]
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Timepoint [9]
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Up to 30 months
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Secondary outcome [10]
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Time from the first documented evidence of CR or CRi or CRh or MLFS until disease relapse or death due to any cause, whichever comes first (Expansion Part)
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Assessment method [10]
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Timepoint [10]
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Up to 30 months
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Secondary outcome [11]
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Time interval from the first SAR443579 administration to the date of earliest evidence of relapse, treatment failure, or death (Expansion Part)
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Assessment method [11]
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Timepoint [11]
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Up to 30 months
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Secondary outcome [12]
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Proportion of survivors from the first SAR443579 administration to death from any cause (Expansion Part)
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Assessment method [12]
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Timepoint [12]
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Up to 12 months
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Secondary outcome [13]
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Rate of hematopoietic stem cell transplantation (HSCT) through SAR443579 treatment but before subsequent therapy (Expansion Part)
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Assessment method [13]
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Timepoint [13]
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Up to 30 months
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Secondary outcome [14]
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Time from first SAR443579 administration to discontinuation for any reason excluding remission, ie, disease progression/disease relapse, treatment toxicity, participant preference or death (Expansion Part)
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Assessment method [14]
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Timepoint [14]
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Up to 30 months
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Secondary outcome [15]
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Time from first SAR443579 administration to death due to any cause
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Assessment method [15]
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Timepoint [15]
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Up to 30 months
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Eligibility
Key inclusion criteria
- Participant must be at least 1 year 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 12 years old.
- Pediatric arm: aged 1 to 17 years old.
For participants of the Escalation Part only:
- Adult and Pediatric Arms: Confirmed diagnosis of primary or secondary AML [any subtype
except acute promyelocytic leukemia (APL) and juvenile myelomonocytic leukemia (JMML)]
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.
Examples include but are not limited to:
- One cycle of high dose cytarabine (HiDAC) containing regimen
- One cycle of liposomal cytarabine and daunorubicin
- Two cycles of standard dose cytarabine containing regimen
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 duration < 6 months on prior
induction treatment
c) Leukemia in first or higher relapse
d) For participants aged 1 to 17 years old, primary induction failure is defined as disease
refractory after two cycles of induction therapy.
- Adult arm only: Confirmed diagnosis of cluster of differentiation 123 (CD123) +
HR-MDS, with a Revised International Prognostic Scoring System (IPSS-R) risk category
of intermediate or higher and are limited to those with no available (or are
ineligible) therapy with known clinical benefit.
- Not eligible for induction therapy and having completed =2 cycles of any of the
following: hypomethylating agent (eg, 5 azacitidine or decitabine) and/or
venetoclax, chemotherapy, or targeted agents.
- Not eligible for autologous stem cell transplant (ASCT) and having completed =1
course of induction therapy.
- Adult and Pediatric arms and escalation part only: Confirmed diagnosis of CD123+ B-ALL
without extramedullary lesions that have no available (or are ineligible) therapy with
known clinical benefit.
For Participants in the Expansion Part Only (Adults only):
- For participants in Cohort A: Participants meeting inclusion criteria for AML
participants that have been primary refractory (PIF) to prior induction treatment or
who have had ER occurring 6 months or less after an initial remission on prior
induction treatment.
- For participants in Cohort B: Participants meeting inclusion criteria for AML
participants that have had late relapse (LR), occurring more than 6 months after an
initial remission on prior induction treatment.
- 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.
- Pediatric arm and expansion part only: For participants in Cohort C: Participants with
AML who have relapsed according to inclusion criteria for AML or have recurrent
disease resistant or intolerant to available therapies.
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Minimum age
1
Year
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Eastern Cooperative Oncology Group (ECOG) performance status >2 (=18 years-old).
Karnovsky Scale (16-17 years-old) <50% or Lansky Scale (<16 years-old) <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 that requires active therapy (adjuvant hormonal
therapy is allowed) other than the one treated in this study, with the exception of
resected/ablated basal or squamous-cell carcinoma of the skin or carcinoma in situ of
the cervix, or other local tumors considered cured by local treatment.
- Evidence of active central nervous system leukemia at the time of enrollment as
evidenced by cytology or pathology. Except for participants aged 1 to 17 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 graft-versus-host disease (GVHD).
- Receiving at the time of first investigational medicinal product (IMP) administration
corticosteroid as a concomitant medication with corticosteroid dose >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.
- Radiotherapy, even if palliative in intent, may not be given during the study.
- Prophylactic use of hematopoietic growth factors (eg, granulocyte-colony stimulating
factor (G-CSF), granulocyte-macrophage colony-stimulating factor (GM-CSF),
erythropoietin) during the DLT observation period in the Dose Escalation Part only. -
Individuals accommodated in an institution because of regulatory or legal order;
prisoners or participants who are legally institutionalized.
- Pregnant and breast-feeding women.
- History of solid organ transplant, including corneal transplant.
- Average QTc (using the Fridericia correction calculation) greater than 470
milliseconds (msec) at screening. For pediatric arm participants only, inadequate
ejection fraction as per institutional standards at screening or any clinically
significant cardiac conditions (including but not limited to congestive heart failure,
myocarditis, pericarditis, arrythmias).
- 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.
The above information is not intended to contain all considerations relevant to a potential
participation in a clinical trial.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 1/Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
8/12/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
11/11/2026
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Actual
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Sample size
Target
126
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Investigational Site Number :0360002 - Melbourne
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Recruitment hospital [2]
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Investigational Site Number :0360001 - Melbourne
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Recruitment postcode(s) [1]
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3000 - Melbourne
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Recruitment postcode(s) [2]
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3004 - Melbourne
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Georgia
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Country [3]
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United States of America
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State/province [3]
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Massachusetts
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Country [4]
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United States of America
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State/province [4]
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New York
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Country [5]
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United States of America
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State/province [5]
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Ohio
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Country [6]
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United States of America
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State/province [6]
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Oregon
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Country [7]
0
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United States of America
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State/province [7]
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Pennsylvania
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Country [8]
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United States of America
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State/province [8]
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Texas
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Country [9]
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United States of America
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State/province [9]
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Washington
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Country [10]
0
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China
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State/province [10]
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Tianjin
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Country [11]
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China
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State/province [11]
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Zhengzhou
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Country [12]
0
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France
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State/province [12]
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Marseille
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Country [13]
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France
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State/province [13]
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Paris
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Country [14]
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France
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State/province [14]
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Villejuif
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Country [15]
0
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Netherlands
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State/province [15]
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Amsterdam
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Country [16]
0
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Netherlands
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State/province [16]
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Groningen
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Country [17]
0
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Netherlands
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State/province [17]
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Nijmegen
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Country [18]
0
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Netherlands
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State/province [18]
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Rotterdam
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Country [19]
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Netherlands
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State/province [19]
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Utrecht
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Sanofi
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05086315
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Clinical Sciences & Operations
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Address
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Sanofi
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Trial Transparency email recommended (Toll free for US & Canada)
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Address
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Country
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Phone
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800-633-1610
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05086315
Download to PDF