NCT04302662

Phase 2 Combination Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs

A Multicenter, Open-label Phase 2 Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs, to Investigate the Efficacy and Safety of This Combination for the Compensation of Severe Exocrine Pancreatic Insufficiency in CF Patients Not Fully Compensated With Only PPEs

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: AzurRx SAS · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2 study sponsored by AzurRx SAS and involves testing of a new medication for the compensation of exocrine pancreatic insufficiency (EPI) caused by cystic fibrosis (CF). The new medication is called MS1819 spray dried (MS1819-SD) which is a lipase produced by the Lip2 gene of Yarrowia lipolytica using recombinant DNA technology. The primary purpose of this study is to investigate the efficacy and safety of escalating doses of study drug on top of a stable dose of PPEs in CF patients who are not fully compensated by PPEs only. This enzyme has demonstrated an appropriate profile to compensate the pancreatic lipase (enzyme) deficiency that is common in CP (chronic pancreatitis) and CF patients. The design of the study is open-label, meaning that all eligible patients will receive the study drug MS1819-SD. The study drug dose will increase throughout the study during dose escalation visits in each treatment period; study includes a total of three treatment periods. The total duration of the MS1819-SD treatment phase is of 39-51 days. The total duration of patient participation in the study is of 69-81 days. Approximately 24 patients will be enrolled in this study.

Conditions

• Cystic Fibrosis
• Cystic Fibrosis Gastrointestinal Disease
• Cystic Fibrosis of Pancreas

Interventions

Type	Name	Description
DRUG	MS1819-SD	Patients will receive increasing doses from the lowest to a maximum dose of MS1819-SD on top of a stable dose of PPEs. The total treatment phase will range from 39 to 51 days.

Timeline

• Start date: 2019-06-27
• Primary completion: 2021-06-03
• Completion: 2021-06-03
• First posted: 2020-03-10
• Last updated: 2021-08-19

Locations

10 sites across 2 countries: Hungary, Turkey (Türkiye)