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)
- Conditions
- Acute leukemiabloodcancer10024324
- Registration Number
- NL-OMON55908
- Lead Sponsor
- Sanofi BV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 18
- Participant must be >=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 >=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)
classification. Patients 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
b) Early relapse (ER) AML
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 according
to Children*s Oncology Group (COG) protocol guidelines (40).
- Adult arm only: Confirmed diagnosis of cluster of differentiation 123 (CD123)
+ HR-MDS
-- 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. Participants with non-CNS
chloromatous disease are not allowed in the study.
For Participants in the Expansion Part Only (adults only):
- For participants in Cohort A: Participants meeting inclusion criteria for AML
patients 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
patients that have had late relapse (LR), occurring more than 6 months after an
initial remission on prior induction treatment.
- Pediatric arm and escalation part only: Confirmed diagnosis of BPDCN
according to World Health Organization (WHO) 2022 classification (39), 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 I 02 or have recurrent
disease resistant or intolerant to available therapies.
-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 squamouscell 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 according to
COG classification (41) are
allowed
-Known acquired immunodeficiency syndrome (AIDS-related illnesses) or HIV
disease requiring antiretroviral treatment, or having active hepatitis B or C
infection, or SARS-CoV-2 infection.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method