Suspended

Phase I Study of TK216 in Patients With Relapsed and Refractory Leukemias

What is being tested

Group 1: TK216

+ Group 2: TK216

+ Part 2: Decitabine 10mg/m2

Drug

Who is being recruted

Hematologic Diseases+4

+ Hemic and Lymphatic Diseases

+ Leukemia

Over 18 Years

+16 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 1

Interventional

Study Start: March 2019

See protocol details

Summary

Principal SponsorM.D. Anderson Cancer Center

Last updated: January 27, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: March 31, 2019

Actual date on which the first participant was enrolled.

PRIMARY OBJECTIVES: I. To determine the maximum tolerated dose (MTD) and dose limiting toxicities (DLT) of Ets-family transcription factor inhibitor TK216 (TK216) in patients with relapsed and refractory (R/R) acute myeloid leukemia (AML). (Phase I Dose Escalation) II. To determine the safety and tolerability of TK216 combined with decitabine in patients with relapsed and refractory AML. (Combination Cohort) SECONDARY OBJECTIVES: I. Safety profile of TK216 as characterized by adverse event (AE) type, severity, timing and relationship to study drug, as well as laboratory abnormalities in the first and subsequent treatment cycles. (Phase I Dose Escalation) II. To explore the efficacy (complete remission \[CR\], complete remission without platelet recovery \[CRp\], complete remission without blood count recovery \[CRi\], or partial remission \[PR\]), of TK216 as a single-agent in patients with R/R AML. (Phase I Dose Escalation) III. To assess overall survival (OS), and disease free survival (DFS) in patients with R/R AML treated with TK216. (Phase I Dose Escalation) IV. Duration of disease control defined as first date of disease control identified (either CR/CRp/CRi, PR or SD) until the date of progression. (Phase I Dose Escalation) V. To explore biomarkers of response and resistance in patients with R/R AML treated with TK216. (Phase I Dose Escalation) VI. Safety profile of TK216 in combination with decitabine as characterized by adverse event (AE) type, severity, timing and relationship to study drug, as well as laboratory abnormalities in the first and subsequent treatment cycles. (Combination Cohort) VII. To explore the efficacy (complete remission \[CR\], complete remission without platelet recovery \[CRp\], complete remission without blood count recovery \[CRi\], or partial remission \[PR\], of TK216 in combination with decitabine in patients with R/R AML. (Combination Cohort) VIII. To assess overall survival (OS), and progression free survival (PFS) in patients with R/R AML treated with TK216 + decitabine. (Combination Cohort) IX. Duration of disease control defined as first date of disease control identified (either CR/CRp/CRi, PR or SD) until the date of progression. (Combination Cohort) X. To explore biomarkers of response and resistance in patients with R/R AML treated with TK216 + decitabine. (Combination Cohort) OUTLINE: This is a dose-escalation study. Patients receive TK216 intravenously (IV) continuously on days 1-7 every 21 days, or continuously on days 1-7 and 15-21 every 28 days. Patients also receive decitabine IV over 60 minutes on days 1-10 every 28 days. Treatment continues in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up for 30 days.

Official TitlePhase I Study of TK216 in Patients With Relapsed and Refractory Leukemias

NCT03752138

Principal SponsorM.D. Anderson Cancer Center

Last updated: January 27, 2026

Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Over 18 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Hematologic DiseasesHemic and Lymphatic DiseasesLeukemiaLeukemia, MyeloidNeoplasmsNeoplasms by Histologic TypeLeukemia, Myeloid, Acute

Criteria

9 inclusion criteria required to participate

Patients with a diagnosis of histologically confirmed relapsed or refractory (R/R) acute myeloid leukemia for which no available standard therapies are indicated or anticipated to result in a durable response

Patients must not have had leukemia therapy for 14 days prior to starting TK216. However, patients with rapidly proliferative disease may receive hydroxyurea as needed until 24 hours prior to starting therapy on this protocol and during the first cycle of study

Bilirubin =< 2 mg/dL

Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) =< 3 x upper limit of normal (ULN) -- or =< 5 x ULN if related to leukemic involvement

Show More Criteria

7 exclusion criteria prevent from participating

Pregnant women are excluded from this study because the agent used in this study has the potential for teratogenic or abortifacient effects. Because there is a potential risk for adverse events in nursing infants secondary to treatment of the mother with the chemotherapy agents, breastfeeding should also be avoided

Uncontrolled intercurrent illness including, but not limited to active uncontrolled infection, symptomatic congestive heart failure (New York Heart Association [NYHA] class III or IV), unstable angina pectoris, clinically significant cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements

Patient with documented hypersensitivity to any of the components of the therapy program

Patients with active, uncontrolled central nervous system (CNS) leukemia will not be eligible

Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

5 intervention groups are designated in this study

This study does not include a placebo group

Treatment Groups

Group I

Experimental

Patients receive TK216 IV continuously on days 1-7 every 21 days.

Group II

Experimental

Patients receive TK216 IV on days 1-7 and 15-21 every 28 days.

Group III

Experimental

Patients receive recommended dose of TK216 from Part 1 plus Decitabine.

Group IV

Experimental

Patients receive recommended dose of TK216 from Part 1 plus Decitabine.

Group 5

Experimental

All patients in the expansion cohort will receive the RP2D of TK216 and Decitabine.

Study Objectives

Primary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has no location dataSave this study to your profile to know when the location data is available.

SuspendedNo study centers