Phase 2 Combination Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04302662|
Recruitment Status : Completed
First Posted : March 10, 2020
Last Update Posted : August 19, 2021
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.
|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis Cystic Fibrosis Gastrointestinal Disease Cystic Fibrosis of Pancreas||Drug: MS1819-SD||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||20 participants|
|Intervention Model:||Sequential Assignment|
|Intervention Model Description:||Sequential assignment: eligible patients will be receiving increased doses from lower, middle to upper range of MS1819-SD on top of a stable dose of PPEs|
|Masking:||None (Open Label)|
|Official Title:||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|
|Actual Study Start Date :||June 27, 2019|
|Actual Primary Completion Date :||June 3, 2021|
|Actual Study Completion Date :||June 3, 2021|
- 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.
- Coefficient of fat absorption [ Time Frame: 15 days ]determination of fat absorption based on fat intake and fat excretion over 3 days on high fat meal
- Adverse Events [ Time Frame: 81 days ]AE, SAE, SUSAR, immunoallergic reactions
- Weight of stools [ Time Frame: 15 days ]evaluation of changes in weight of stools from baseline (PPEs only) to each treatment period
- number of daily evacuations [ Time Frame: 15 days ]evaluation of changes in daily evacuations from baseline (PPEs only) to each treatment period
- Steatorrhea, [ Time Frame: 15 days ]evaluation of changes in steatorrhea from baseline (PPEs only) to each treatment period
- Creatorrhea [ Time Frame: 15 days ]evaluation of changes in creatorrhea from baseline (PPEs only) to each treatment period
- Body weight [ Time Frame: 15 days ]evaluation of changes in body weight from baseline (PPEs) to each treatment period
- Consistency of stools [ Time Frame: 15 days ]evaluation of consistency of stools from baseline (PPEs) to each treatment period
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04302662
|Országos Korányi TBC és Pulmonológiai Intézet Cisztás Fibrózis Részleg|
|Budapest, Hungary, 1122|
|Borsod-Abaúj-Zemplén Megyei Központi Kórház és Egyetemi Oktatókórház, Gyermekegészségügyi Központ|
|Miskolc, Hungary, 3526|
|Somogy Megyei Kaposi Mór Oktató Kórház, Mosdósi telephelye Mosdósi Gyermekrehabilitációs és Gyermekpulmonológiai Egység|
|Mosdós, Hungary, 7257|
|Tüdőgyógyintézet Törökbálint Gyermekpulmonológiai Osztály és Szakrendelés|
|Törökbálint, Hungary, 2045|
|Çukurova University School of Medicine|
|Hacettepe University School of Medicine|
|Akdeniz University School of Medicine|
|Cerrahpasa University School of Medicine|
|Mamara University School of Medicine|
|Necmettin Erbakan University,Meram School of Medicine|