Table of contents

• Overview of the trial program
• Conditions studied
• Who can take part
• Trial phases and study goals
• Main endpoints measured
• Selected trials of interest
• Key terms explained

Overview of the trial program

These studies are interventional trials, which means the researchers give a treatment and then measure the results.^[1] In the source data, Idarubicin Hydrochloride appears in trials for blood cancers and one trial about cancer treatment-related heart damage.^[1][2]

The trials are in Phase 1, Phase 2, and Phase 3, so the drug is being studied both in early research and in larger comparison studies.^[1][2]

Conditions studied

The most common condition is acute myeloid leukemia (AML).^{[1][4][5][6][8][9][10][12]}

Other studies include acute lymphoblastic leukemia (ALL), including adult Philadelphia chromosome-positive ALL, Philadelphia chromosome-negative ALL, very high-risk T-lineage ALL, and relapsed childhood precursor B-cell ALL.^{[3][7][9][10][11]}

One study includes hepatocellular carcinoma in people with cirrhosis and Child A/B7 liver function status.^[2] Another study looks at anthracycline-based antineoplastic therapy and whether heart imaging can detect early myocardial damage.^[8]

Who can take part

The target populations are different across studies. Some trials are for newly diagnosed patients, while others are for relapsed or refractory disease, meaning the cancer has come back or has not responded well.^{[4][5][6][9][10][11][12]}

Some trials are for adults only, some for children and adolescents, and some for older patients with recently diagnosed non-favorable AML.^{[5][6][7][9][10][11]}

Several studies focus on special risk groups, such as KMT2A or NPM1 alterations, FLT3-ITD negative AML, MRD-negative patients, and very high-risk T-ALL.^{[1][5][6][7][9][10]}

Trial phases and study goals

In Phase 1, the main goal is usually to learn about safety, tolerability, and dose finding.^[1][5][6] One Phase 1 study in newly diagnosed non-favorable AML uses Idarubicin Hydrochloride with cytarabine and sulfasalazine to assess safety, dose-limiting toxicity, maximal tolerated dose, and recommended phase II dose.^[5]

In Phase 2, the trials look more closely at whether the treatment seems to work. Examples include disease control rate in hepatocellular carcinoma and MRD negativity after induction in very high-risk T-ALL.^[2][7][10]

In Phase 3, the studies compare treatment strategies in larger groups. These trials measure outcomes such as event-free survival, overall survival, MRD-negative remission, and relapse-free results.^{[3][4][6][9][11][12]}

Main endpoints measured

A primary endpoint is the main result a trial wants to measure.^[2][4] In these studies, the endpoints include disease control rate, overall survival, event-free survival, MRD negativity, and complete response.^{[2][3][4][5][6][7][9][10][11][12]}

Some studies also measure dose-limiting toxicity, which means side effects serious enough to affect the dose, and overall adverse event burden, which is the total burden of side effects seen by the doctor.^[5][4]

One trial uses myocardial mapping and T2-weighted relaxation time changes to look for early heart muscle damage during anthracycline-containing treatment.^[8]

Selected trials of interest

NCT05580861 studies newly diagnosed non-favorable AML in older patients and tests whether sulfasalazine can be added to cytarabine and Idarubicin Hydrochloride.^[5] The Phase 1 part focuses on safety, dose-limiting toxicity, maximal tolerated dose, and the recommended phase II dose, while the Phase II part looks at MRD-negative complete response at the end of induction.^[5]

NCT07211958 studies newly diagnosed AML with an NPM1 mutation and compares revumenib plus intensive chemotherapy with placebo plus intensive chemotherapy.^[12] It measures event-free survival and MRD-negative complete remission, and Idarubicin Hydrochloride is part of the chemotherapy backbone.^[12]

2022-500783-35-00 is a large international trial in children and adolescents with AML.^[9] It includes different groups and measures event-free survival, overall survival, and a composite endpoint that includes graft-versus-host disease, veno-occlusive disease, relapse, death, and second cancers.^[9]

2023-509392-17-00 studies childhood relapsed precursor B-cell ALL and includes Idarubicin Hydrochloride in one treatment arm.^[11] Its main focus is event-free survival and disease-free survival in different relapse groups.^[11]

Key terms explained

MRD negativity means that very small amounts of cancer cells are not found after treatment.^{[5][7][10][12]}

Complete response means the signs of cancer have gone away on the tests used by the study.^{[2][5][11][12]}

Randomization means participants are assigned by chance to one study group or another.^[3][4]

Relapsed or refractory disease means the cancer has returned or has not responded to treatment.^[4][6][11]

Disease control rate includes partial response, complete response, or stable disease, depending on the study definition.^[2]