NCT04810611

Phase Ib Study of Select Drug Combinations in Patients With Lower Risk MDS

A Phase Ib, Multicenter, Open-label Platform Study of Select Drug Combinations in Adult Patients With Lower Risk (Very Low, Low, or Intermediate Risk) Myelodysplastic Syndrome

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 33 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study was to characterize the safety, tolerability and confirm the dose for select single agents and combinations in patients with lower risk (very low, low, and intermediate risk) MDS.

Detailed description

This was a phase Ib, multi center, open-label, platform study with multiple treatment arms. The design of this study was adaptive to allow discontinuation of poorly tolerated or ineffective treatments and to facilitate the introduction of new candidate single agents or combinations. Study design included a dose escalation/confirmation part and a dose expansion. The planned initial single agent and combination treatment arms were the following: * Arm 1: MBG453 single agent * Arm 2: NIS793 single agent * Arm 3: canakinumab single agent * Arm 4: MBG453 + NIS793 combination * Arm 5: MBG453 + canakinumab combination Patients were treated in the dose confirmation/escalation part of the study in Arms 1, 2, 3 and 5. No patients were treated in Arm 4. The study did not progress into the expansion phase.

Conditions

• Myelodysplastic Syndromes

Interventions

Type	Name	Description
DRUG	MBG453	Anti-TIM3 monoclonal antibody
DRUG	NIS793	Anti-TGF-β monoclonal antibody
DRUG	canakinumab	Anti-IL-1β monoclonal antibody

Timeline

• Start date: 2021-06-18
• Primary completion: 2024-04-19
• Completion: 2024-04-19
• First posted: 2021-03-23
• Last updated: 2025-10-10

Locations

13 sites across 7 countries: United States, Australia, Israel, Italy, Singapore, South Korea, Spain

Regulatory

• FDA-regulated drug study