NCT03946670

A Study of MBG453 in Combination With Hypomethylating Agents in Subjects With IPSS-R Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS).

A Randomized, Double-blind, Placebo-controlled Phase II Multi-center Study of Intravenous MBG453 Added to Hypomethylating Agents in Adult Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R Criteria

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 127 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

This Phase II was a multicenter, randomized, two-arm parallel-group, double-blind, placebo-controlled study of MBG453 or placebo added to hypomethylating agents (azacitidine or decitabine) in adult subjects with IPSS-R intermediate, high or very high risk myelodysplastic syndrome (MDS) not eligible for Hematopoietic Stem Cell Transplant (HSCT) or intensive chemotherapy.

Detailed description

The 2 primary objectives were as follows: To determine if MBG453 combined with standard HMA therapy improved complete remission in subjects with intermediate, high, or very high risk MDS. To determine if MBG453 combined with standard HMA therapy improved progression free survival (PFS) in subjects with intermediate, high or very high risk MDS. This Phase II study was a multicenter, randomized, two-arm parallel-group, double-blind, placebo-controlled study of MBG453 or placebo added to hypomethylating agents (azacitidine or decitabine, as per investigators' choice based on local standard of care (SOC)) in adult subjects with IPSS-R intermediate, high or very high risk MDS not eligible for HSCT or intensive chemotherapy. A total of 127 subjects were randomized in a 1:1 ratio to treatment arms as follows: * MBG453 400 mg IV Q2W and decitabine or azacitidine * Placebo IV Q2W and decitabine or azacitidine The randomization was stratified by 2 stratification factors: a) HMA (decitabine or azacitidine) selected by the investigator as per the local SOC and b) IPSS-R prognostic risk categories (intermediate, high or very high) at randomization. Crossover between treatment arms was not permitted at any time during the study.

Conditions

• Myelodysplastic Syndromes

Interventions

Type	Name	Description
DRUG	MBG453	MBG453 is being administered i.v.
DRUG	Placebo	Placebo is being administered i.v.
DRUG	Hypomethylating agents	Decitabine is being administered i.v. Azacitidine is being administered i.v or s.c.

Timeline

• Start date: 2019-06-04
• Primary completion: 2022-04-26
• Completion: 2024-07-15
• First posted: 2019-05-13
• Last updated: 2026-01-13
• Results posted: 2023-07-07

Locations

48 sites across 17 countries: United States, Austria, Belgium, Canada, Czechia, France, Germany, Greece, Hong Kong, Hungary, Italy, Japan, South Korea, Spain, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study