Back to resultsA Phase IIa Study With Escalating Dose of MS1819-SD
Primary Purpose
Chronic Pancreatitis, Distal Pancreatectomy
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
MS1819-SD
Sponsored by
About this trial
This is an interventional treatment trial for Chronic Pancreatitis focused on measuring Exocrine Pancreatic Insufficiency
Eligibility Criteria
Inclusion Criteria:
- Signed and dated informed consent form,
- Age >18 years,
- Male or female,
- Body weight within the range [50-100 Kg] for males or [40-90 Kg] for females,
- Distal pancreatectomy for any disorder (e.g. acute pancreatitis or its complications, CP or its complications, pancreatic endocrine or exocrine cancer, or others) and/or CP of any etiology (e.g. alcohol, genetics, hypercalcemia, or others) of grade 2 or higher (Cambridge classification),
- Faecal pancreatic elastase-1 <100 μg/g of stools at screening or within one month of the screening visit,
- CFA measurement ≤ 75% at washout
- Female patients must be post-menopausal (defined as at least 12 months post cessation of menses), surgically sterile or, if of childbearing potential, using a reliable method of contraception during the study.
- Being considered as reliable and capable of adhering to the protocol, according to the judgment of the investigator.
Exclusion Criteria:
- Cystic fibrosis,
- Total or partial gastrectomy,
- Cephalic or total duodenopancreatectomy,
- Documented fibrosing colonopathy,
- Any small bowel disease possibly responsible for malabsorption, including small intestinal bacterial overgrowth, celiac disease, small bowel resection ≥1 meter length, etc.,
- Acute pancreatitis or exacerbation of CP ≤3 months,
- Pancreatectomy for exocrine or endocrine cancer ≤1 year,
- Metastatic or locally recurrent exocrine pancreatic cancer,
- Known hypersensitivity or other severe reaction to any ingredient of the investigational medicinal product,
- Bilirubin >3 times ULN (upper limit normal),
Sites / Locations
- Mackay Institute of Research and Innovation
- CMAX
- Linear Research
- Hôpital Timone Adulte (CIC-CPCET)
- Christchurch Clinical Studies Trust
- P3 Research
Outcomes
Primary Outcome Measures
Investigate safety of escalating doses of MS1819-SD as measured by number of participants with adverse events including clinical or laboratory abnormalities
Safety variables will be monitored by physical examination with particular attention paid to immunoallergic events and digestive symptomatology. In addition, laboratory tests will include haematology and biochemistry.
Secondary Outcome Measures
Investigate the efficacy of MS1819-SD in patients by the Coefficient of Fat Absorption change from baseline
The primary efficacy endpoint is the Coefficient Fat Absorption (CFA) change from baseline.The secondary efficacy endpoints are the number of daily evacuations per day, the consistency of stools assessed by the Bristol scale (from Type 1 to Type 7) and the weight of stools during the stool collection period.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03481803
Brief Title
A Phase IIa Study With Escalating Dose of MS1819-SD
Official Title
A Multicentre Open-label Phase IIa Study With Escalating Dose of MS1819- SD, to Investigate the Efficacy and Safety of a Yarrowia Lipolytica Lipase Preparation for the Compensation of Exocrine Pancreatic Insufficiency Caused by Chronic Pancreatitis and/or Distal Pancreatectomy
Study Type
Interventional
2. Study Status
Record Verification Date
September 2018
Overall Recruitment Status
Completed
Study Start Date
January 27, 2017 (Actual)
Primary Completion Date
June 29, 2018 (Actual)
Study Completion Date
June 29, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
AzurRx SAS
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This is a Phase IIa study sponsored by AzurRx SAS and Syneos Health is a local representative sponsor and involves testing of a new medication for the compensation of exocrine pancreatic insufficiency (EPI) caused by chronic pacreatitis (CP) and/or distal pancreatectomy. 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 safety of escalating doses of study drug MS1819-SD in people with chronic pancreatitis. This enzyme has demonstrated an appropriate profile to compensate the pancreatic lipase (enzyme) deficiency that is common with CP patients. The deficiency in this enzyme can be responsible of greasy diarrhea, fecal urge and weight loss.
The design of the study is open-label, meaning that all eligible participants will receive the study drug MS1819-SD. The MS1819-SD dose will increase throughout the study during dose escalation visits in each treatment period; study includes a total of four treatment periods.
The total duration of the MS1819-SD treatment phase is of 48-60 days, The total duration of patient participation in the study is of 74-93 days.
Approximately twelve patients will be enrolled in this study.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chronic Pancreatitis, Distal Pancreatectomy
Keywords
Exocrine Pancreatic Insufficiency
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Model Description
Sequential Assignment Eligible patients will be receiving increased doses from lower, middle to upper range of study medication MS1819-SD
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
11 (Actual)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
MS1819-SD
Intervention Description
Patient will receive increasing doses from the lowest dose of MS1819-SD to a maximum dose of MS1819- SD. The total treatment phase will range from 48 days to 60 days.
Primary Outcome Measure Information:
Title
Investigate safety of escalating doses of MS1819-SD as measured by number of participants with adverse events including clinical or laboratory abnormalities
Description
Safety variables will be monitored by physical examination with particular attention paid to immunoallergic events and digestive symptomatology. In addition, laboratory tests will include haematology and biochemistry.
Time Frame
60 days
Secondary Outcome Measure Information:
Title
Investigate the efficacy of MS1819-SD in patients by the Coefficient of Fat Absorption change from baseline
Description
The primary efficacy endpoint is the Coefficient Fat Absorption (CFA) change from baseline.The secondary efficacy endpoints are the number of daily evacuations per day, the consistency of stools assessed by the Bristol scale (from Type 1 to Type 7) and the weight of stools during the stool collection period.
Time Frame
60 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Signed and dated informed consent form,
Age >18 years,
Male or female,
Body weight within the range [50-100 Kg] for males or [40-90 Kg] for females,
Distal pancreatectomy for any disorder (e.g. acute pancreatitis or its complications, CP or its complications, pancreatic endocrine or exocrine cancer, or others) and/or CP of any etiology (e.g. alcohol, genetics, hypercalcemia, or others) of grade 2 or higher (Cambridge classification),
Faecal pancreatic elastase-1 <100 μg/g of stools at screening or within one month of the screening visit,
CFA measurement ≤ 75% at washout
Female patients must be post-menopausal (defined as at least 12 months post cessation of menses), surgically sterile or, if of childbearing potential, using a reliable method of contraception during the study.
Being considered as reliable and capable of adhering to the protocol, according to the judgment of the investigator.
Exclusion Criteria:
Cystic fibrosis,
Total or partial gastrectomy,
Cephalic or total duodenopancreatectomy,
Documented fibrosing colonopathy,
Any small bowel disease possibly responsible for malabsorption, including small intestinal bacterial overgrowth, celiac disease, small bowel resection ≥1 meter length, etc.,
Acute pancreatitis or exacerbation of CP ≤3 months,
Pancreatectomy for exocrine or endocrine cancer ≤1 year,
Metastatic or locally recurrent exocrine pancreatic cancer,
Known hypersensitivity or other severe reaction to any ingredient of the investigational medicinal product,
Bilirubin >3 times ULN (upper limit normal),
Facility Information:
Facility Name
Mackay Institute of Research and Innovation
City
Mackay
State/Province
Queensland
ZIP/Postal Code
4740
Country
Australia
Facility Name
CMAX
City
Adelaide
State/Province
South Australia
ZIP/Postal Code
5000
Country
Australia
Facility Name
Linear Research
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Facility Name
Hôpital Timone Adulte (CIC-CPCET)
City
Marseille
ZIP/Postal Code
13385
Country
France
Facility Name
Christchurch Clinical Studies Trust
City
Christchurch
ZIP/Postal Code
8011
Country
New Zealand
Facility Name
P3 Research
City
Wellington
ZIP/Postal Code
6242
Country
New Zealand
12. IPD Sharing Statement
Plan to Share IPD
Undecided
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A Phase IIa Study With Escalating Dose of MS1819-SD
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