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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Leukemia, Myeloid, Acute; Myelodysplastic Neoplasms
• Intervention / Treatment: Drug: JNJ-89853413

• Study Type: Interventional
• Enrollment (Actual): 64
• Phase: Phase 1

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2N 5G2
      • Arthur J E Child Comprehensive Cancer Centre
  • British Columbia
    • Vancouver, British Columbia, Canada, V5Z 1M9
      • Vancouver General Hospital
  • Ontario
    • Toronto, Ontario, Canada, M5G 2C1
      • Princess Margaret Hospital
• Spain
  • Barcelona, Spain, 08036
    • Hosp Clinic de Barcelona
  • Madrid, Spain, 28040
    • Hosp Univ Fund Jimenez Diaz
  • Pamplona, Spain, 31008
    • Clinica Univ. de Navarra
• United Kingdom
  • Cambridge, United Kingdom, Cb2 2qq
    • Addenbrookes Hospital
  • London, United Kingdom, W1T 7HA
    • University College London Hospitals
  • Manchester, United Kingdom, M20 4BX
    • The Christie NHS Foundation Trust Christie Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Drug: JNJ-89853413; JNJ-89853413 will be administered.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Janssen Research & Development, LLC
• Investigators: Study Director: Janssen Research & Development, LLC Clinical Trial, Janssen Research & Development, LLC

Study record dates

Study Major Dates

• Study Start (Actual): January 14, 2025
• Primary Completion (Estimated): November 16, 2026
• Study Completion (Estimated): November 16, 2026

Study Registration Dates

• First Submitted: September 26, 2024
• First Submitted That Met QC Criteria: September 26, 2024
• First Posted (Actual): October 1, 2024

Study Record Updates

• Last Update Posted (Actual): August 28, 2026
• Last Update Submitted That Met QC Criteria: August 27, 2026
• Last Verified: August 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia, Myeloid; Leukemia; Hemic and Lymphatic Diseases; Leukemia, Myeloid, Acute
• Other Study ID Numbers: 89853413AML1001 (Other Identifier: Janssen Research & Development, LLC); 2024-513199-16-00 (Registry Identifier: EUCT number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: The data sharing policy of Johnson & Johnson Innovative Medicine is available at innovativemedicine.jnj.com/our-innovation/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes