A Study of JNJ-95804306 for Relapsed or Refractory Hematological Malignancies

A Phase 1, First-in-human, Dose Escalation Study of JNJ-95804306 for Relapsed or Refractory Hematological Malignancies

The purpose of Part 1 (Dose Escalation) of the study is to assess how safe and tolerable JNJ-95804306 is and to find out the most suitable dose (recommended phase 2 dose [RP2D]) of JNJ-95804306. The purpose of Part 2 (Dose Expansion) is to further assess the safety of JNJ-95804306 and determine the anti-tumor activity alone and/or when administered in addition to standard of care (SoC) therapy at the putative RP2D(s) regimens in participants with hematological malignancies (cancer that begins in blood-forming tissue, such as the bone marrow, or in the cells of the immune system). For US sites: The purpose of Part 1 (Dose Escalation) of the study is to assess how safe and tolerable JNJ-95804306 is and to find out the most suitable dose (recommended phase 2 dose [RP2D]) of JNJ-95804306. The purpose of Part 2 (Dose Expansion) is to further assess the safety of JNJ-95804306 and determine the anti-tumor activity alone at the putative RP2D(s) regimens in participants with hematological malignancies (cancer that begins in blood-forming tissue, such as the bone marrow, or in the cells of the immune system).

Study Overview

• Status: Recruiting
• Conditions: Hematologic Neoplasms
• Intervention / Treatment: Drug: JNJ-95804306; Drug: AML SoC; Drug: CLL/SLL SoC

• Study Type: Interventional
• Enrollment (Estimated): 360
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Study Contact; Phone Number: 844-434-4210; Email: Participate-In-This-Study1@its.jnj.com

Study Locations

• Australia
  • Melbourne, Australia, 3000
    • Recruiting
    • Peter MacCallum Cancer Centre
• Belgium
  • Edegem, Belgium, 2650
    • Recruiting
    • UZ Antwerpen
• Denmark
  • Aarhus N, Denmark, DK-8200
    • Recruiting
    • Aarhus University Hospital
• France
  • Marseille, France, 13009
    • Recruiting
    • Institut Paoli Calmettes
  • Pierre-Bénite, France, 69495
    • Recruiting
    • CHU Lyon Sud
  • Toulouse, France, 31059
    • Recruiting
    • Institut Claudius Regaud
• Spain
  • Madrid, Spain, 28040
    • Recruiting
    • Hosp Univ Fund Jimenez Diaz
  • Pamplona, Spain, 31008
    • Recruiting
    • Clinica Univ. de Navarra
• United Kingdom
  • Manchester, United Kingdom, M20 4BX
    • Recruiting
    • The Christie NHS Foundation Trust
• United States
  • Indiana
    • Indianapolis, Indiana, United States, 46237
      • Recruiting
      • Indiana Blood & Marrow Transplantation
  • Michigan
    • Grand Rapids, Michigan, United States, 49546
      • Recruiting
      • START MidWest
  • New Jersey
    • Piscataway, New Jersey, United States, 08854
      • Recruiting
      • Rutgers Cancer Institute of New Jersey
  • New York
    • New York, New York, United States, 10016
      • Recruiting
      • NYU Langone Health
  • Tennessee
    • Nashville, Tennessee, United States, 37203
      • Recruiting
      • Sarah Cannon Cancer Institute
  • Texas
    • Houston, Texas, United States, 77030
      • Recruiting
      • MD Anderson Cancer Center - University of Texas

Participation Criteria

Eligibility Criteria

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

Description

Inclusion criteria:

For Arm A:

• Have a diagnosis of: Acute myeloid leukemia (AML) per International Consensus Classification (ICC) 2022 or myelodysplastic syndromes (MDS) per world health organization (WHO) 2022 classified as moderate high, high, or very high-risk per the molecular international prognostic scoring system (IPSSM). All participants must have relapsed or refractory disease and have exhausted or are ineligible for standard therapeutic options
• Body weight greater than or equal to (>=) 40 kilograms (kg)
• Eastern cooperative oncology group (ECOG) performance status of 0 to 2

For Arm B:

• All participants must have relapsed or refractory disease with no other approved therapies available that would be more appropriate in the investigator's judgement. Have a diagnosis of either: Chronic lymphocytic leukemia (CLL)/Small lymphocytic lymphoma (SLL) meeting 2018 International workshop on chronic lymphocytic leukemia (iwCLL) National cancer institute (NCI) working group guidelines (Hallek 2018) that meet the following criteria:

  a. Participants must have received at least 2 prior lines of therapy; b. Have clinically measurable disease

• Body weight >= 40 kg
• ECOG performance status of 0 to 2
• Must sign an Informed consent form (ICF)
• For US sites: Have a diagnosis of CLL/SLL that meets iwCLL, NCI Working Group Guidelines which is relapsed or refractory and requires treatment with no other approved therapies available that would be more appropriate in the investigator's judgement. a. Participants must have received at least 2 prior lines of therapy

Exclusion criteria:

For Arm A:

• Has acute promyelocytic leukemia according to world health organization (WHO) 2022 criteria or known active central nervous system (CNS) involvement of AML/MDS, unless in specific cohort (s) per study evaluation team (SET) decision
• Need for supplemental oxygen use to maintain adequate oxygenation
• Have evidence of uncontrolled systemic viral, bacterial, or fungal infection. Antimicrobial prophylaxis is permitted
• For US sites: Has acute promyelocytic leukemia according to WHO 2022 criteria or known active CNS involvement of AML/MDS

For Arm B:

• Need for supplemental oxygen use to maintain adequate oxygenation
• Have evidence of uncontrolled systemic viral, bacterial, or fungal infection requiring initiation of parenteral treatment as medical intervention
• Developed Richter's transformation or prolymphocytic leukemia
• Known active CNS or leptomeningeal involvement of CLL/SLL/Non-Hodgkin lymphoma (NHL)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Arm A: R/R Acute Myeloid Leukemia (AML)/ High-Risk Myelodysplastic Syndrome (HR MDS); Participants with relapsed/refractory (R/R) AML/HR-MDS will receive JNJ-95804306 monotherapy (Arm A1) or as an addition to standard of care (SoC) therapy in AML (Arm A2) to determine the putative recommended phase 2 dose (RP2D) in Part 1 (Dose escalation) of the study. In Part 2 (Dose expansion) participants will receive JNJ-95804306 monotherapy (Arm A1) or as an addition to SoC therapy in AML (Arm A2) at the determined RP2D regimen(s). For US sites: Participants with R/R AML/HR-MDS will receive JNJ-95804306 monotherapy to determine the putative RP2D in Part 1 (Dose escalation) of the study. In Part 2 (Dose expansion) participants will receive JNJ-95804306 monotherapy at the determined RP2D regimen(s). AML SoC will not be administered for US sites.	Drug: JNJ-95804306; JNJ-95804306 will be administered orally.; Drug: AML SoC; AML SoC will be administered subcutaneously/intravenously.
Experimental: Arm B: R/R Chronic Lymphocytic Leukemia (CLL)/ Small Lymphocytic Lymphoma (SLL) or NHL monotherapy; Participants with R/R CLL/SLL/NHL will receive JNJ-95804306 monotherapy (Arm B1) or as an addition to SoC therapy in R/R CLL/SLL (Arm B2 or B3) to determine the putative RP2D in Part 1 (Dose escalation) of the study. In Part 2 (Dose expansion) participants will receive JNJ-95804306 monotherapy (Arm B1) or as an addition to SoC therapy in R/R CLL/SLL (Arm B2 or B3) at the determined RP2D regimen(s). For US sites: Participants with R/R CLL/SLL/NHL will receive JNJ-95804306 monotherapy to determine the putative RP2D in Part 1 (Dose escalation) of the study. In Part 2 (Dose expansion) participants will receive JNJ-95804306 monotherapy at the determined RP2D regimen(s). CLL/SLL/NHL SoC will not be administered for US sites.	Drug: JNJ-95804306; JNJ-95804306 will be administered orally.; Drug: CLL/SLL SoC; CLL/SLL SoC will be administered orally/ intravenously.

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 participant administered a pharmaceutical (investigational or non investigational) product. An AE does not necessarily have a causal relationship with the treatment. Severity of AEs will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version (v) 6.0. by using standard grades as follows: Grade 1: Mild; asymptomatic or mild symptoms; Grade 2: Moderate; minimal, local or noninvasive intervention indicated; Grade 3: Severe but not immediately life threatening; hospitalization or prolongation of hospitalization indicated; Grade 4: Life-threatening consequences; and Grade 5: Death related to AE.	Up to 6 years 5 months
Part 1: Number of Participants with Dose Limiting Toxicities (DLTs)	DLT is defined as any toxicity that requires discontinuation of treatment; any toxicity resulting in dose reduction of study treatment, any toxicity resulting in a participant receiving less than (<) 2/3 of their intended dose; any grade 5 toxicity; non-hematologic toxicity (grade 3 or 4); and unacceptable hematologic toxicity.	Up to 28 days after first full dose of study drug

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
For US sites: Part 1: Number of Participants with Dose Limiting Toxicities (DLTs)	DLT is defined as any toxicity that requires discontinuation of treatment; any toxicity resulting in dose reduction of study treatment, any toxicity resulting in a participant receiving less than (<) 2/3 of their intended dose; any grade 5 toxicity; non-hematologic toxicity (grade 3 or 4); and unacceptable hematologic toxicity.	Up to first 28 days after first dose of study drug
Serum Concentrations of JNJ-95804306	Serum samples will be analyzed to determine concentrations of JNJ-95804306.	Up to approximately 6 years 5 months
Area Under the Curve From Time of Administration until End of Dosing Interval (AUC[t]) of JNJ-95804306	AUC[t] is defined as the area under the plasma concentration time curve during a dosing interval at steady-state.	Up to approximately 6 years 5 months
Maximum Plasma Concentration (Cmax) of JNJ-95804306	Cmax is defined as the maximum serum concentration of JNJ-95804306.	Up to approximately 6 years 5 months
Minimum Plasma Concentration (Cmin) of JNJ-95804306	Cmin is defined as the minimum plasma concentration of JNJ-95804306.	Up to approximately 6 years 5 months
Complete Response (CR) in Participants with Acute Myeloid Leukemia (AML)	Complete response (CR) is achieved when a participant with AML 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 (ELN) 2022 criteria.	Up to 6 years 5 months
Overall Response (OR) in Participants with Myelodysplastic Syndrome (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.	Up to 6 years 5 months
Complete Response (CR) in Participants with MDS	CR is achieved when a participant with MDS has a best response of CR (including CRh/CRL) according to the IWG 2023 criteria.	Up to 6 years 5 months
Overall Response (OR) in Participants with Chronic Lymphocytic Leukemia (CLL)	OR is achieved when a participant with CLL has a CR or PR according to the International Workshop on Chronic Lymphocytic Leukemia (iwCLL) criteria.	Up to 6 years 5 months
Complete Response in Participants with CLL	Complete response as per iwCLL response criteria will be reported.	Up to 6 years 5 months
Duration of Response (DoR)	DOR is defined for responders only, as time from date of initial documentation of a response to the first documented evidence of no response, disease progression, relapse, initiation of a new systemic anti-cancer therapy (besides hematopoietic stem cell transplant [HSCT]), or death, whichever comes first.	Up to 6 years 5 months
Time to Response (TTR)	TTR is defined for responders only, as the time from the first dose of any study treatment to first qualifying response.	Up to 6 years 5 months
Overall Response in Participants with Non-Hodgkin Lymphoma (NHL) Subtypes Including Small Lymphocytic Lymphoma (SLL)	Overall response is achieved when a participant with NHL subtypes including SLL has a CR or PR according to the revised response criteria for malignant lymphoma.	Up to 6 years 5 months
Complete Response in Participants With NHL Subtypes Including SLL	Complete response is achieved when a participant with NHL subtypes including SLL has a best response of CR according to the revised response criteria for malignant lymphoma.	Up to 6 years 5 months
Overall Response in WM (Waldenstrom's Macroglobulinemia)	Overall response is achieved when a participant with WM has a CR or PR according to international workshop on waldenstrom's macroglobulinemia 2013	Up to 6 years 5 months
Complete Response in WM	Complete response is achieved when a participant with WM has a best response of CR according to international workshop on waldenstrom's macroglobulinemia 2013.	Up to 6 years 5 months
Best Overall Response (BOR) Based on Indication-Specific Criteria	Participants with BOR based on indication-specific criteria will be reported.	Up to 6 years 5 months

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): May 13, 2026
• Primary Completion (Estimated): May 18, 2029
• Study Completion (Estimated): September 24, 2032

Study Registration Dates

• First Submitted: April 30, 2026
• First Submitted That Met QC Criteria: April 30, 2026
• First Posted (Actual): May 7, 2026

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 by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Hematologic Neoplasms
• Other Study ID Numbers: 95804306EDI1001 (Other Identifier: Janssen Research & Development, LLC); 2026-525234-39 (EudraCT Number); 2026-525234-39-00 (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 www.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