A Phase IIa Study With Escalating Dose of MS1819-SD

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.

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Key information

Conditions

Chronic Pancreatitis Chronic Disease Digestive System Diseases Disease Attributes Distal Pancreatectomy Exocrine Pancreatic Insufficiency Pancreatic Diseases Pancreatitis Pancreatitis, Chronic Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Mackay Institute of Research and Innovation, Mackay, Queensland, Australia

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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),

Treatment and study plan

MS1819-SD

Drug

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 outcomes

Investigate safety of escalating doses of MS1819-SD as measured by number of participants with adverse events including clinical or laboratory abnormalities

Time frame: 60 days

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 outcomes

Investigate the efficacy of MS1819-SD in patients by the Coefficient of Fat Absorption change from baseline

Time frame: 60 days

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.

Sponsors and collaborators

Lead sponsor

AzurRx SAS

Industry

Registry information

Official study 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

Important dates

Study start: 2017
Primary completion: 2018
Study completion: 2018
First posted: Mar 29, 2018
Registry last updated: Sep 28, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed