Unknown Cancer NIHR-supported project Digestion, Kidneys & Other Organs

DSP-5336-101 - A Phase 1/2, Open-Label, Dose-Escalation, Dose-Expansion Study of Enzomenib (DSP-5336) in Adult Patients with Acute Leukemia and Other Selected Hematologic Malignancies, with and without Mixed Lineage Leukemia (MLL) rearrangement or Nucleophosmin 1 (NPM1) Mutation

Funder NIHR (part of DHSC)
Recipient organisation NIHR Wellcome Trust UCL Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Mar 2025 — Ongoing

In plain English

AI plain-English summary

A new drug called DSP-5336 is being tested in a global clinical trial for people with acute leukaemias that carry specific genetic faults—alterations in the MLL gene or mutations in the NPM1 gene. These blood cancers are notoriously difficult to treat, especially in older adults, and current options are limited for patients whose disease has returned or stopped responding to therapy. The trial is designed to find the safest and most effective dose of DSP-5336, both alone and in combination with existing AML drugs, and to understand how the body processes it. If the drug proves safe and effective, it could offer a new treatment pathway for patients who currently have few alternatives. The study targets leukaemias defined by specific genetic changes—MLL rearrangements and NPM1 mutations—meaning that success could lead to more personalised, genetically targeted therapies for these hard-to-treat blood cancers. The UK is participating in all three parts of the study, including the early dose-finding phase and later expansion in patients with the relevant genetic profiles.

View original technical description
Sumitomo Pharma America, Inc. is conducting a study in the globe including US, EU and APAC to test a new drug called DSP-5336 for treating certain types of difficult-to-treat blood cancers. The study focuses on acute leukemia, a severe blood cancer that's particularly challenging to treat, especially in older adults. There are two main types: acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL), both of which can be life-threatening.The study targets leukemias with specific genetic changes, including alterations in the MLL gene (found in 5-10% of adult cases and more frequently in children) and mutations in the NPM1 gene (present in about 30% of adult AML cases).About 130 patients will be included in Phase 1. Around 1 to 6 patients will be enrolled at each dose level. For Phase 2 about 20 to 40 patients will be included in each of the three arms.The UK is participating in all three parts of the study:1. Phase 1 Testing DSP-5336 alone in patients whose leukemia has returned or not responded to treatment arm A and B.2. Phase 1 Evaluating DSP-5336 in combination with other drugs used for AML (venetoclax/azacitidine or gilteritinib) in adults with difficult-to-treat AML (Arm E and Arm F)3. Phase 2 Assessing DSP-5336 by itself in patients with specific genetic changes who haven't been treated with similar drugs before. (Arm G) relapsed/refractory AML with MLL rearrangement. (Arm H) relapsed/refractory AML with NPM1 mutation. (Arm I) relapsed/refractory ALL with MLL rearrangement.The researchers aim to determine:• The most effective dose of the drug• Its safety profile• How the body processes the drug• Whether it helps control the cancerThis study is important because it targets types of leukemia that are currently hard to treat effectively, potentially offering new hope for patients with limited treatment options.

Researchers

Jenny O'Nions (Principal Investigator)

Related Research

Grants with similar aims, by meaning.

A Phase 1/2, Open-Label, Dose-Escalation, Dose-Expansion Study of DSP 5336 in Adult Patients with Acute Leukemia and Other Selected Hematologic Malignancies, with and without Mixed Lineage Leukemia (MLL) rearrangement or Nucleophosmin 1 (NPM1) Mutation
Sumitomo - A Phase 1/2, Open-Label, Dose-Escalation, Dose-Expansion Study of DSP-5336 in Adult Patients with Acute Leukaemia and Other Selected Hematologic Malignancies, with and without Mixed Lineage Leukaemia (MLL) rearrangement or Nucleophosmin 1 (NPM1) Mutation
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CL1-65487-003 - Phase I / II, open label, dose escalation part (phase I) followed by non-comparative expansion part (phase II), multi-centre study, evaluating safety, pharmacokinetics and efficacy of S65487, a Bcl2 inhibitor combined with azacitidine in adult patients with previously untreated acute myeloid leukemia not eligible for intensive treatment
A Phase 1, First-in-Human, Dose Escalation Study of JNJ-89853413 for Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Neoplasms

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