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)

  • STATUS
    Recruiting
  • End date
    Nov 11, 2026
  • participants needed
    126
  • sponsor
    Sanofi
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Updated on 3 December 2024
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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.

Description

2.5 years.

Details
Condition Acute Lymphocytic Leukaemia, Acute Myeloid Leukaemia Refractory, Myelodysplastic Syndromes, Blastic Plasmacytoid Dendritic Cell Neoplasia
Age 1year or above
Treatment SAR443579
Clinical Study IdentifierNCT05086315
SponsorSanofi
Last Modified on3 December 2024

Eligibility

 Yes No Not Sure

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
For participants of the Escalation Part only
Adult arm: aged at least 12 years old
Pediatric arm: aged 1 to 17 years old
Primary Induction Failure (PIF) AML, defined as disease refractory to one of the following, i or ii
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
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
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
Not eligible for induction therapy and having completed ≥2 cycles of any of the
of intensive induction chemotherapy; PIF is defined as AML refractory to one of the
following: hypomethylating agent (eg, 5 azacitidine or decitabine) and/or
following less intensive regimens, 1 or 2: 1. 4 cycles of hypomethylating agents (HMA) or
venetoclax, chemotherapy, or targeted agents
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 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

Exclusion Criteria

Concurrent treatment with other investigational drugs
Radiotherapy, even if palliative in intent, may not be given during the study
Pregnant and breast-feeding women
History of solid organ transplant, including corneal transplant
The above information is not intended to contain all considerations relevant to a potential
participation in a clinical trial
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
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
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
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