ActiveCancerNIHR-supported projectDigestion, Kidneys & Other Organs
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
Recipient organisationNIHR University College London Hospitals Biomedical Research Centre
NIHR supportRecorded as supported by this research centre
PeriodMar 2025 — Sept 2031
In plain English
AI plain-English summary
A new drug called DSP-5336 is being tested in around 130 patients across the UK, US, EU, and Asia to see if it can treat acute leukemias that have stopped responding to existing therapies. These blood cancers are particularly deadly in older adults, and about 30% of adult acute myeloid leukemia (AML) cases carry a mutation in the NPM1 gene, while 5–10% have a rearrangement of the MLL gene—both genetic changes that make the disease harder to treat. Current options for patients whose leukemia returns or resists treatment are limited, and no drug specifically targets these mutations. If DSP-5336 proves safe and effective, it could offer a new treatment pathway for patients who currently have few alternatives. The study is structured in two phases: Phase 1 tests the drug alone and in combination with standard AML drugs (venetoclax/azacitidine or gilteritinib) to find the right dose and check safety; Phase 2 then assesses the drug’s ability to control the cancer in three groups of patients with relapsed or refractory disease, each defined by a specific genetic marker. Success would mean a targeted therapy for a set of leukemias that today carry a poor prognosis.
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.
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