A Phase I/II Study of Enzomenib (DSP-5336) in Adult Patients with Acute Leukemia

Zusammenfassung der Studie

This clinical trial aims to evaluate different doses of DSP-5336 to determine the dose that is most likely to show an effect and is safe for patients with acute leukemia. Another goal of the study is to gather information about the potential clinical effects of DSP-5336 that could benefit patients with acute leukemias. DSP-5336 is a new drug and is not approved. Laboratory studies have shown that DSP-5336 affects leukemia cells, particularly those with certain genetic abnormalities, such as those referred to as 'MLL rearrangement' and 'NPM1 mutation.' The results of this study will provide information on how DSP-5336 is absorbed, distributed, and metabolized by the body, as well as any effects the investigational drug may have on the body and/or in the treatment of acute leukemias. This clinical trial is intended for patients with acute leukemia who have already received standard therapy but whose leukemia has returned. Patients may also be included if the standard leukemia treatment did not work, if the patient had to discontinue standard therapy due to side effects, or if the patient is otherwise medically unable to receive standard therapy. The study has 2 parts - a Phase I part and a Phase II part. The goal of the Phase I part of the study is to find the dose(s) of DSP-5336 that can be safely administered without causing too many side effects. Once the best dose(s) are selected in Phase 1, additional patients will participate in Phase 2. The aim of Phase 2 is to test the best dose of DSP-5336 in more patients to see if it works and to better understand the side effects.

(BASEC)

Untersuchte Intervention

This clinical trial aims to assess the safety of a new drug called DSP-5336 at various doses to determine what effects it may have on humans and to evaluate the clinical effect of DSP-5336 at a recommended Phase 2 dose. The following will be measured and assessed:

- Type, frequency, and severity of side effects during the study.

- Improvement of symptoms and medical assessment of how a patient's ability to carry out daily activities changes.

- Proportion of patients whose disease slows down, stabilizes, worsens, or improves, as well as the extent of improvement after starting DSP-5336 treatment.

- Average survival time of patients after starting DSP-5336 treatment.

- Possible effects of DSP-5336 on patients' heart health.

- Absorption, distribution, or metabolism of DSP-5336 by the body.

DSP-5336 is a tablet taken twice daily.

DSP-5336 is administered according to a schedule referred to as a 'cycle.' In each cycle, patients take DSP-5336 daily for 28 days. During the 21 days before the first treatment, several examinations are conducted to ensure that the liver, lungs, heart, and kidneys are functioning adequately and that vital signs and physical condition are normal. On the first day of treatment, after taking the tablet, multiple blood samples will be taken over a period of 8 hours in Phase 1 and 2 hours in Phase 2 to determine the level of DSP-5336 in the blood. Blood samples will also be taken for special tests to determine how DSP-5336 affects leukemia.

In the first cycle, the patient will visit the clinic for 5 days for a physical examination, vital sign assessment, and blood draws. In the second cycle, the patient will visit the clinic for a physical examination, vital parameters, and blood draws for 4 days in the Phase 1 part or for 2 days in the Phase 2 part. During subsequent cycles, the patient will come to the clinic on Day 1 to undergo the same examinations.

A bone marrow sample will be taken at the beginning of the study, at the start of Cycle 2, at the start of Cycle 3, and then at the beginning of each odd-numbered study cycle (in some cases, however, patients may need a bone marrow biopsy earlier than normally scheduled). This is an important examination to learn more about how DSP-5336 affects leukemia.

The study will also involve monitoring the heart using electrocardiograms (also known as ECGs) and regular echocardiograms (ultrasound examinations of the heart).

During this study, participants will be asked questions in addition to taking DSP-5336 to determine if they tolerate the drug and if their disease symptoms improve.

Study participants will receive their study treatment as long as their disease does not worsen, they do not tolerate the investigational drugs, or the participant no longer wishes to participate in the study, whichever occurs first.

(BASEC)

Untersuchte Krankheit(en)

Acute Myeloid Leukemia

(BASEC)

Sponsor

Sumitomo Pharma America, Inc. USA PAREXEL International (CH) AG

(BASEC)

Name der bewilligenden Ethikkommission (bei multizentrischen Studien nur die Leitkommission)

Ethikkommission Zürich

(BASEC)

Datum der Bewilligung durch die Ethikkommission

27.02.2026

(BASEC)

Ergebnisse der Studie