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

Phase 1

Recruiting

• Conditions: Leukemia, Myeloid, Acute; Myelodysplastic Neoplasms
• Interventions: Drug: JNJ-89853413

• Registration Number: NCT06618001
• Lead Sponsor: Janssen Research & Development, LLC

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-09-26
• Study First Submitted QC: 2024-09-26
• Study First Posted: 2024-10-01
• Study Start: 2025-01-14
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-22
• Last Update Posted: 2025-05-23
• Primary Completion: 2027-07-15
• Study Completion: 2028-08-15

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

• Have a diagnosis, per World Health Organization (WHO) 2022 criteria of:

  1. relapsed/refractory acute myeloid leukemia (AML)
  2. relapsed/refractory moderate high, high, or very high risk myelodysplastic neoplasms (MDS) per Molecular International Prognostic Scoring System (IPSS-M)

• Body weight greater than or equals to (>=) 40 kilograms (kg)

• Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 - 2

• Have adequate renal function defined as Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Estimated Glomerular Filtration Rate (eGFR) >=40 milligrams per minute (mL/min)

• Participants must have laboratory parameters in the required range

Exclusion Criteria

• Has a medical history of clinically significant pulmonary compromise, particularly the need for current supplemental oxygen use to maintain adequate oxygenation
• Has evidence of an uncontrolled systemic viral, bacterial, or fungal infection
• Has known allergies, hypersensitivity, or intolerance to the excipients of JNJ-89853413
• Had major surgery or had significant traumatic injury within 14 days of planned first dose of JNJ-89853413
• Has known active central nervous system involvement

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
JNJ-89853413	JNJ-89853413	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.

Primary Outcome Measures

Name	Time	Method
Number of Participants with Adverse events (AEs) by Severity	From screening untill 30 days after last dose of study drug (that is approximately 2.5 years)	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.
Part 1: Number of Participants with Dose-Limiting Toxicity (DLTs)	14 days	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.

Secondary Outcome Measures

Name	Time	Method
Serum Concentration of JNJ- 89853413	Approximately 2.5 years	Serum samples will be analyzed to determine concentrations of JNJ-89853413 using a validated immunoassay method.
Area Under the Plasma Concentration-time (AUC[t]) Curve of JNJ-89853413	Approximately 2.5 years	AUC\[t\] is defined as the area under the plasma concentration time curve during a dosing interval at steady-state.
Maximum Serum Concentration (Cmax) of JNJ-89853413	Approximately 2.5 years	Cmax is defined as maximum serum concentration of JNJ-89853413.
Trough Observed Serum Concentration (Ctrough) of JNJ-89853413	Approximately 2.5 years	Ctrough is the trough observed serum concentration of JNJ-89853413.
Number of Participants with Presence of anti-drug Antibodies of JNJ-89853413	Approximately 2.5 years	Participants with anti JNJ-89853413 antibodies will be analyzed by a bridging electrochemiluminescence (ECL) enzyme linked immune assay.
Complete Response (CR) in Acute Myeloid Leukemia (AML)	Approximately 2.5 years	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.
Overall Response (OR) in Myelodysplastic Neoplasms (MDS)	Approximately 2.5 years	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.
Complete Response in MDS	Approximately 2.5 years	CR is achieved when a participant has a best response of CR (including CRh/CRL) according to the IWG 2023 criteria.
Duration of Response (DOR)	Approximately 2.5 years	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.
Time to response (TTR)	Approximately 2.5 years	TTR is defined for responders only, as the time from the first dose of study drug to first qualifying response.
Number of Participants Achieving Transfusion independence	Approximately 2.5 years	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.

Trial Locations

Locations (7)

The Christie NHS Foundation Trust Christie Hospital; 🇬🇧 Manchester, United Kingdom

Vancouver General Hospital; 🇨🇦 Vancouver, British Columbia, Canada

Princess Margaret Hospital; 🇨🇦 Toronto, Ontario, Canada

Hosp Clinic de Barcelona; 🇪🇸 Barcelona, Spain

Hosp Univ Fund Jimenez Diaz; 🇪🇸 Madrid, Spain

Clinica Univ. de Navarra; 🇪🇸 Pamplona, Spain

University College London Hospitals; 🇬🇧 London, United Kingdom