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
Sponsor study identifier
interventional
20
2 countries
10
Study Start
June 27, 2019
Actual (reported)Primary Completion
June 3, 2021
Actual (reported)Study Completion
June 3, 2021
Actual (reported)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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
June 3, 2021
Actual (reported)Click on a node to explore related trials.
Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
June 27, 2019
Actual (reported)March 4, 2020
Actual (reported)March 10, 2020
Actual (reported)June 3, 2021
Actual (reported)June 3, 2021
Actual (reported)Coefficient of fat absorption
determination of fat absorption based on fat intake and fat excretion over 3 days on high fat meal
15 days
Adverse Events
AE, SAE, SUSAR, immunoallergic reactions
81 days
Weight of stools
15 days
number of daily evacuations
15 days
Steatorrhea,
15 days
Creatorrhea
15 days
Body weight
15 days
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.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Országos Korányi TBC és Pulmonológiai Intézet Cisztás Fibrózis Részleg
Budapest, 1122, Hungary
Borsod-Abaúj-Zemplén Megyei Központi Kórház és Egyetemi Oktatókórház, Gyermekegészségügyi Központ
Miskolc, 3526, Hungary
Somogy Megyei Kaposi Mór Oktató Kórház, Mosdósi telephelye Mosdósi Gyermekrehabilitációs és Gyermekpulmonológiai Egység
Mosdós, 7257, Hungary
Tüdőgyógyintézet Törökbálint Gyermekpulmonológiai Osztály és Szakrendelés
Törökbálint, 2045, Hungary
Çukurova University School of Medicine
Adana, Turkey (Türkiye)
Hacettepe University School of Medicine
Ankara, Turkey (Türkiye)
20 participants (actual enrollment reported by the registry).
First Submitted
March 4, 2020
First Posted
March 10, 2020
Study Start
June 27, 2019
Actual (reported)A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis
Contact the study team to confirm eligibility.
Akdeniz University School of Medicine
Antalya, Turkey (Türkiye)
Cerrahpasa University School of Medicine
Istanbul, Turkey (Türkiye)
Mamara University School of Medicine
Istanbul, Turkey (Türkiye)
Necmettin Erbakan University,Meram School of Medicine
Konya, Turkey (Türkiye)
Primary Completion
June 3, 2021
Actual (reported)Study Completion
June 3, 2021
Actual (reported)Last Updated
August 19, 2021
Record last verified: 2021-08
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Source: ClinicalTrials.gov.
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