NCT04988555

A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1)

A Phase 1/2, Open-Label, Dose-Escalation, Dose-Expansion Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia and Other Selected Hematologic Malignancies, With and Without Mixed Lineage Leukemia (MLL) Rearrangement or Nucleophosmin 1 (NPM1) Mutation (Horizen-1)

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 606 (estimated)
• Sponsor: Sumitomo Pharma America, Inc. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

A phase 1/2 dose escalation / dose expansion study of Enzomenib (DSP-5336) in patients with acute leukemia.

Detailed description

DSP-5336-101 is a phase 1/2 open-label, dose escalation, dose expansion study in which the safety, PK, pharmacodynamics, and clinical activity of orally administered DSP-5336 will be evaluated in patients with relapsed or refractory AML, ALL, or acute leukemia of ambiguous lineage, and in selected sites and regions, in adult patients with high-risk relapsed or refractory MDS or relapsed MM. Additionally, the safety and clinical activity of orally administered DSP-5336 will be evaluated in combination with Standard-of-Care (SOC) AML treatments including: (a) the SOC nonintensive regimen (venetoclax + azacitidine) or (b) the SOC intensive regimen (cytarabine + daunorubicin induction, 7+3) in patients with newly diagnosed AML who have MLLr or NPM1m.

Conditions

• Leukemia, Myeloid, Acute
• Leukemia, Lymphocytic, Acute
• Multiple Myeloma
• Myelodysplastic Syndromes

Interventions

Type	Name	Description
DRUG	Enzomenib	DSP-5336 orally
DRUG	azoles	Posaconazole, Voriconazole, or Fluconazole
DRUG	Venetoclax	Venetoclax orally
DRUG	Gilteritinib	Gilteritinib orally
DRUG	Azacitidine (AZA)	Azacitidine orally
DRUG	Intensive chemotherapy with 7 + 3	chemotherapy

Timeline

• Start date: 2022-02-28
• Primary completion: 2027-06-30
• Completion: 2027-12-31
• First posted: 2021-08-03
• Last updated: 2026-03-24

Locations

104 sites across 12 countries: United States, Belgium, Canada, France, Italy, Japan, Singapore, South Korea, Spain, Switzerland, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study