The purpose of Part 1 (Dose Escalation) of the study is to assess the safety and tolerability, and to identify the recommended Phase 2 dose[s] (RP2D[s]) in participants with relapsed or refractory (R/R) acute myeloid leukemia (AML) (that is a type of blood cancer that has come back after treatment/or has stopped responding to treatment) or R/R higher-risk type of myelodysplastic neoplasms (MDS, type of blood cancer). The purpose of Part 2 (Cohort Expansion) is to further assess the safety, tolerability and efficacy in participants with R/R AML or higher-risk types of MDS.

A Study of JNJ-89853413 for Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Neoplasms

Leukemia, Myeloid, Acute

Acute Myeloid Leukemia

Leukemia

Blood Cancer

Hematologic Neoplasms

Hematologic Malignancies

Hematology

Hematological Malignancies

Myelodysplastic Neoplasms

JNJ-89853413

A Phase 1, First-in-Human, Dose Escalation Study of JNJ-89853413 for Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Neoplasms

An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention. Severity will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0. Severity scale ranges from Grade 1= Mild, Grade 2= Moderate, Grade 3= Severe, Grade 4= Life-threatening and Grade 5= Death related to adverse event.

Participants with dose-limiting toxicity (DLT) will be assessed. DLT is defined as any toxicity that requires discontinuation of treatment, any Grade 5 toxicity; Non-hematologic Toxicity (Grade 3 or 4) and Hematologic Toxicity.

Serum samples will be analyzed to determine concentrations of JNJ-89853413 using a validated immunoassay method.

AUC[t] is defined as the area under the plasma concentration time curve during a dosing interval at steady-state.

Cmax is defined as maximum serum concentration of JNJ-89853413.

Ctrough is the trough observed serum concentration of JNJ-89853413.

Participants with anti JNJ-89853413 antibodies will be analyzed by a bridging electrochemiluminescence (ECL) enzyme linked immune assay.

CR is achieved when a participant has a best response of CR (complete response with partial hematologic recovery [CRh] or complete response with incomplete hematologic recovery [CRi]) according to the European Leukemia Network (ENL) 2022 criteria.

OR is achieved when a participant with MDS has a CR (any type, that is CRh or complete response with limited count recovery [CRL]), partial response (PR), or hematologic improvement (HI) according to the International Working Group (IWG) 2023 criteria.

CR is achieved when a participant has a best response of CR (including CRh/CRL) according to the IWG 2023 criteria.

DOR is defined for responsders only, as time from date of initial documentation of a response to the first documented evidence of no reponse, disease progression, relapse, initation of a new systemic anti-cancer therapy (besides hematopoietic stem cell transplant [HSCT]), or death, whichever comes first.

TTR is defined for responders only, as the time from the first dose of study drug to first qualifying response.

Transfusion independence is defined as the absence of red blood cell (RBC) and platelet transfusions for 8 weeks or longer after starting study treatment for participants with AML and 16 weeks or longer for participants with MDS.

Participants will receive JNJ-89853413 in Part 1 (Dose escalation) of the study and the dose levels will be escalated sequentially based on the decisions of the Study Evaluation Team (SET) until the recommended Phase 2 Dose (RP2D) has been identified. Participants in Part 2 (Dose expansion) will receive JNJ-89853413 at the RP2D determined in Part 1.

The purpose of Part 1 (Dose Escalation) of the study is to assess the safety and
tolerability, and to identify the recommended Phase 2 dose[s] (RP2D[s]) in participants
with relapsed or refractory (R/R) acute myeloid leukemia (AML) (that is a type of blood
cancer that has come back after treatment/or has stopped responding to treatment) or R/R
higher-risk type of myelodysplastic neoplasms (MDS, type of blood cancer). The purpose of
Part 2 (Cohort Expansion) is to further assess the safety, tolerability and efficacy in
participants with R/R AML or higher-risk types of MDS.

Vancouver General Hospital

Princess Margaret Hospital

Hosp Clinic de Barcelona

Hosp Univ Fund Jimenez Diaz

Clinica Univ. de Navarra

An AE is any untoward medical occurrence in a clinical study participant administered a
pharmaceutical (investigational or non-investigational) product. An AE does not
necessarily have a causal relationship with the intervention. Severity will be graded
according to the National Cancer Institute Common Terminology Criteria for Adverse Events
(NCI-CTCAE) version 5.0. Severity scale ranges from Grade 1= Mild, Grade 2= Moderate,
Grade 3= Severe, Grade 4= Life-threatening and Grade 5= Death related to adverse event.

Participants with dose-limiting toxicity (DLT) will be assessed. DLT is defined as any
toxicity that requires discontinuation of treatment, any Grade 5 toxicity;
Non-hematologic Toxicity (Grade 3 or 4) and Hematologic Toxicity.

Serum samples will be analyzed to determine concentrations of JNJ-89853413 using a
validated immunoassay method.

AUC[t] is defined as the area under the plasma concentration time curve during a dosing
interval at steady-state.

Participants with anti JNJ-89853413 antibodies will be analyzed by a bridging
electrochemiluminescence (ECL) enzyme linked immune assay.

CR is achieved when a participant has a best response of CR (complete response with
partial hematologic recovery [CRh] or complete response with incomplete hematologic
recovery [CRi]) according to the European Leukemia Network (ENL) 2022 criteria.

OR is achieved when a participant with MDS has a CR (any type, that is CRh or complete
response with limited count recovery [CRL]), partial response (PR), or hematologic
improvement (HI) according to the International Working Group (IWG) 2023 criteria.

CR is achieved when a participant has a best response of CR (including CRh/CRL) according
to the IWG 2023 criteria.

DOR is defined for responsders only, as time from date of initial documentation of a
response to the first documented evidence of no reponse, disease progression, relapse,
initation of a new systemic anti-cancer therapy (besides hematopoietic stem cell
transplant [HSCT]), or death, whichever comes first.

TTR is defined for responders only, as the time from the first dose of study drug to
first qualifying response.

Transfusion independence is defined as the absence of red blood cell (RBC) and platelet
transfusions for 8 weeks or longer after starting study treatment for participants with
AML and 16 weeks or longer for participants with MDS.

Participants will receive JNJ-89853413 in Part 1 (Dose escalation) of the study and the
dose levels will be escalated sequentially based on the decisions of the Study Evaluation
Team (SET) until the recommended Phase 2 Dose (RP2D) has been identified. Participants in
Part 2 (Dose expansion) will receive JNJ-89853413 at the RP2D determined in Part 1.

A Study of JNJ-89853413 for Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Neoplasms

Trials at a glance

Trials at a glance