NCT06052813

A Study of BN104 in the Treatment of Acute Leukemia

A PhaseI/II, Multicenter, Open-label Clinical Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of the Menin Inhibitor BN104 in the Treatment of Patients With Relapsed/Refractory Acute Leukemia

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 66 (actual)
• Sponsor: Institut de Recherches Internationales Servier (I.R.I.S.) · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The Phase I/II trial is to learn the safety, pharmacokinetics, and preliminary efficacy of BN104 taken once daily or twice daily in patients with acute lymphocytic leukemia or acute myeloblastic leukemia.

Detailed description

The study is divided into 2 phases. Phase1 part will enroll 98 patients to evaluate safety and tolerance of BN104 in patients with relapsed/refractory (R/R) Acute Leukemia to determine maximum tolerated dose and recommended phase2 dose (RP2D), including approximately 66 adult patients and 32 adolescent patients with specific mutations (KMT2A gene rearrangement, NPM1 gene mutation, NUP98 mutation) enrolled at phaseI. Phase II expansion part will enroll 168 patients and be conducted at the selected dose level to further evaluate the safety and tolerability of BN104, as well as preliminary efficacy in Acute leukemia subjects with specific mutations (KMT2A gene rearrangement or NPM1 gene mutation). Patients will be allocated into 2 Acute Leukemia subgroup cohorts depends on their genotype. * Cohort A: Patients with Relapsed/refractory AML subjects with NPM1 mutations * Cohort B: Patients with relapsed/refractory acute leukaemia with KMT2A rearrangement (including AML, ALL, or MPL) Patients will receive orally administrated BN104 once daily or twice daily. Study drug will be administered in 28-day cycles until disease progression or unacceptable toxicity, death, Informed consent withdraw ect. Laboratory tests will be performed weekly in Cycles 1-2, bi-weekly in Cycle3 and every 4weeks from Cycle 4 onwards. Efficacy assessment will be performed on baseline, C2D1, C3D1 and every 2 cycles from Cycle3 onwards. Additional clinical assessments and laboratory tests may be performed at discretion of the investigator as clinically indicated.

Conditions

• ALL, Adult
• AML, Adult
• Acute Leukaemia
• KMT2A Rearrangement
• NPM1 Mutation
• NUP98 Gene Rearrangement
• Menin Inhibitors

Interventions

Type	Name	Description
DRUG	BN104 monotherapy	Phase I(adults): Will be administered orally once daily (approximately every 24 hours) for the first cohort or twice daily (approximately every 12 hours) for the subsequent cohorts.
DRUG	BN104 monotherapy	Phase I(adolescent): Will be administered orally twice daily (approximately every 12 hours)
DRUG	BN104 monotherapy - rp2d	receiving oral BN104 treatment at a dose of 600 mg BID (300 mg BID when co-administered with strong CYP3A4 inhibitors)

Timeline

• Start date: 2023-10-19
• Primary completion: 2026-12-01
• Completion: 2027-12-01
• First posted: 2023-09-25
• Last updated: 2026-02-25

Locations

1 site across 1 country: China

Regulatory

• FDA-regulated drug study