A Study of JNJ-67571244 in Participants With Relapsed or Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

NCT03915379 | Completed Phase 1 FDA Drug

• 3 other identifiers: CR1085822018-004452-3767571244AML1001
• Study Type: interventional
• Target: 68
• Locations: 3 countries
• Sites: 17

Brief Summary

The main purpose of this study are to determine the recommended Phase 2 dose(s) (RP2D) route of administration, schedule and the maximum tolerated dose (MTD) in Part 1 and to determine the safety and tolerability of JNJ-67571244 at the RP2D regimen(s) and to evaluate the preliminary clinical activity of JNJ-67571244 in Part 2.

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (4)

• Part 1 and Part 2: Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability

  An AE is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/biological agent under study.

  Up to 100 days after the last dose of study drug or until the start of a subsequent anticancer therapy, whichever comes first (that is up to 2.3 years)

• Part 1: Number of Participants with Dose-Limiting Toxicity (DLTs)

  Number of participants with dose-limiting toxicity will be assessed. The DLTs are based on drug-related adverse events and defined as any of the following events: infusion-related reactions, non-hematologic toxicity of Grade 3 or higher, or hematologic toxicity.

  Up to 28 days

• Part 1: Severity of DLT as Assessed by National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE)

  The DLT evaluation period is defined as the first 28 days after a participant's first infusion (Day 1). Severity criteria is based on Grade 1, 2, 3, 4 and 5, will be assessed by the investigator as per below grades. Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening consequences; Grade 5: Death related to adverse event.

  Up to 28 days

• Part 2: Overall Response Rate (ORR)

  ORR is defined as the percentage of participants who have complete response (CR) and incomplete blood count recovery (CRi) (AML) or CR and partial response (PR) (MDS) as per modified International Working Group response (IWGR) criteria.

  Approximately 2.3 years

Secondary Outcomes (7)

• Part 1 and Part 2: Serum Concentrations of JNJ-67571244

  Approximately 2.3 years

• Part 1 and 2: Systemic Cytokine Concentrations

  Approximately 2.3 years

• Number of Participants with Depletion of CD33-Expressing Cells

  Approximately 2.3 years

• Part 1 and 2: Concentration of Markers of T-Cell Activation

  Up to 24 days

• Part 1 and 2: Number of Participants with JNJ-67571244 Antibodies

  Week 1 (Day 1) up to post treatment Week 8

Study Arms (2)

Part 1: Dose Escalation

EXPERIMENTAL

Participants will receive JNJ-67571244. 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.

Drug: JNJ-67571244

Part 2: Dose Expansion

EXPERIMENTAL

Participants in 2 expansion cohorts of acute myeloid leukemia (AML) or either high-risk myelodysplastic syndromes (MDS) or very high-risk MDS will receive JNJ-67571244 at the RP2D determined in Part 1.

Drug: JNJ-67571244

Interventions

JNJ-67571244 DRUG

JNJ-67571244 will be administered.

Part 1: Dose Escalation; Part 2: Dose Expansion

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• A diagnosis of:
• a) Acute Myeloid Leukemia (AML) according to the World Health Organization 2008 criteria with relapsed or refractory disease and ineligible for or have exhausted standard therapeutic options b) high-risk or very high-risk Myelodysplastic Syndrome (MDS) according to International Prognostic Scoring System (IPSS-R) and relapsed or refractory after at least 1 course of hypomethylating therapy and ineligible for or have exhausted standard therapeutic options per investigator discretion should be included
• Eastern Cooperative Oncology Group (ECOG) performance status grade of 0 or 1
• Women of childbearing potential must have a negative pregnancy test at screening and prior to the first dose of study drug using a highly sensitive pregnancy test (either serum or urine beta human chorionic gonadotropin \[beta-hCG\])
• Chemistry laboratory parameters within the following range during screening:
• a) aspartate transaminase (AST) and alanine aminotransferase (ALT) less than or equal to (\<=) 3\* upper limit of normal (ULN), b) Total bilirubin \<=1.5\*ULN; participants with congenital bilirubinemia, such as Gilbert's syndrome, may enroll if conjugated bilirubin is within normal range, c) Creatinine clearance calculated or measured creatinine clearance greater than or equal to (\>=) 30 milliliters per minute (mL/min)
• Before the first dose of study drug:
• Women of childbearing potential and fertile men who are sexually active must agree to use a highly effective method of contraception (less than \[\<\] 1 percent {%} year failure rate from the time of signing the informed consent form \[ICF\]) during the study and for 90 days after the last dose of study drug. Contraception must be consistent with local regulations regarding the use of birth control methods for participants participating in clinical trials. 1) Participant must agree to practice a highly effective method of contraception (failure rate of \<1% per year when used consistently and correctly). Examples of highly effective contraceptives include: a) user-independent methods: implantable progestogen-only hormone contraception associated with inhibition of ovulation; intrauterine device; intrauterine hormone-releasing system; vasectomized partner; b) user-dependent methods: combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation: oral, intravaginal, and transdermal; progestogen-only hormone contraception associated with inhibition of ovulation: oral and injectable, c) In addition to the highly effective method of contraception, a man: 1) Who is sexually active with a woman of childbearing potential must agree to use a barrier method of contraception (for example, condom with spermicidal foam/gel/film/cream/suppository), 2) Who is sexually active with a woman who is pregnant must use a condom, c) Women and men must agree not to donate eggs (ova, oocytes) or sperm, respectively, during the study and for 90 days after the last dose of study drug

You may not qualify if:

• Willing and able to undergo allogenic stem cell transplant \<=6 months before the first dose of study drug, has evidence of graft versus host disease, or requires immunosuppressant therapy (exception: daily doses less than 10 milligram (mg) prednisone or equivalent are allowed for adrenal replacement)
• For Part 1 only, prior treatment with CD33 targeting therapy targeting T-cell redirection (for example, CD-3 redirection technology or chimeric antigen receptor \[CAR\]-T-cell therapy)
• For Part 1 only, prior Grade 3 cytokine release syndrome (CRS) related to any T-cell redirection (for example, CD-3 redirection technology or CAR-T cell therapy)
• Prior treatment with a checkpoint inhibitor such that the first dose of JNJ-67571244 would occur within less than 5 half-lives. Prior treatment with chemotherapy, targeted therapy, immunotherapy, radiotherapy, or treatment with an investigational anticancer agent, an investigational drug (including investigational vaccines), within 2 weeks prior to the first dose or at least 4 half-lives, whichever is less, or currently receiving investigational therapy in a clinical trial. Hydroxyurea may be used
• Toxicities (except for alopecia, peripheral neuropathy, thrombocytopenia) from previous anticancer therapies that have not resolved to baseline levels or to Grade 1 or less

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (17)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

Barbara Ann Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

NYU Hematology Associates

New York, New York, 10016, United States

Location

Levine Cancer Institute, Carolinas HealthCare System

Charlotte, North Carolina, 28204, United States

Location

Sarah Cannon Research Institute

Nashville, Tennessee, 37203, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States

Location

Universitaetsklinikum Leipzig

Leipzig, 04103, Germany

Location

Klinikum der Universitaet Muenchen

München, 81377, Germany

Location

Universitätsklinikum Münster; Med. Klinik A - Germany

Münster, 48149, Germany

Location

Hosp Clinic de Barcelona

Barcelona, 08036, Spain

Location

Hosp Univ Vall D Hebron

Barcelona, 8035, Spain

Location

Hosp Univ Fund Jimenez Diaz

Madrid, 28040, Spain

Location

Clinica Univ. de Navarra

Pamplona, 31008, Spain

Location

Hosp. Virgen Del Rocio

Seville, 41013, Spain

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 29, 2019
• First Posted: April 16, 2019
• Study Start: March 28, 2019
• Primary Completion: March 26, 2022
• Study Completion: March 28, 2022
• Last Updated: May 23, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will share

Locations

US (9); DE (3); ES (5)