A Phase IIa Study With Escalating Dose of MS1819-SD

• ClinicalTrials.gov Identifier: NCT03481803
• Recruitment Status: Completed
• First Posted: March 29, 2018
• Last Update Posted: September 28, 2018
• Sponsor: AzurRx SAS
• Information provided by (Responsible Party): AzurRx SAS

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.

Condition or disease	Intervention/treatment	Phase
Chronic Pancreatitis; Distal Pancreatectomy	Drug: MS1819-SD	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 11 participants
• Allocation: Non-Randomized
• Intervention Model: Sequential Assignment
• Intervention 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)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: January 27, 2017
• Actual Primary Completion Date: June 29, 2018
• Actual Study Completion Date: June 29, 2018

Arms and Interventions

Intervention Details:

• Drug: MS1819-SD
  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.

Outcome Measures

Primary Outcome Measures :

1. 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 Outcome Measures :

1. 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.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

1. Signed and dated informed consent form,
2. Age >18 years,
3. Male or female,
4. Body weight within the range [50-100 Kg] for males or [40-90 Kg] for females,
5. 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),
6. Faecal pancreatic elastase-1 <100 μg/g of stools at screening or within one month of the screening visit,
7. CFA measurement ≤ 75% at washout
8. 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.
9. Being considered as reliable and capable of adhering to the protocol, according to the judgment of the investigator.

Exclusion Criteria:

1. Cystic fibrosis,
2. Total or partial gastrectomy,
3. Cephalic or total duodenopancreatectomy,
4. Documented fibrosing colonopathy,
5. Any small bowel disease possibly responsible for malabsorption, including small intestinal bacterial overgrowth, celiac disease, small bowel resection ≥1 meter length, etc.,
6. Acute pancreatitis or exacerbation of CP ≤3 months,
7. Pancreatectomy for exocrine or endocrine cancer ≤1 year,
8. Metastatic or locally recurrent exocrine pancreatic cancer,
9. Known hypersensitivity or other severe reaction to any ingredient of the investigational medicinal product,
10. Bilirubin >3 times ULN (upper limit normal),

Contacts and Locations

Locations

Australia, Queensland

Mackay Institute of Research and Innovation

Mackay, Queensland, Australia, 4740

Australia, South Australia

CMAX

Adelaide, South Australia, Australia, 5000

Australia, Western Australia

Linear Research

Perth, Western Australia, Australia, 6009

France

Hôpital Timone Adulte (CIC-CPCET)

Marseille, France, 13385

New Zealand

Christchurch Clinical Studies Trust

Christchurch, New Zealand, 8011

P3 Research

Wellington, New Zealand, 6242

Sponsors and Collaborators

AzurRx SAS

More Information

• Responsible Party: AzurRx SAS
• ClinicalTrials.gov Identifier: NCT03481803
• Other Study ID Numbers: MS1819/16/01
• First Posted: March 29, 2018
• Last Update Posted: September 28, 2018
• Last Verified: September 2018

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Undecided

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by AzurRx SAS:

Exocrine Pancreatic Insufficiency

Additional relevant MeSH terms:

Pancreatitis; Pancreatitis, Chronic; Exocrine Pancreatic Insufficiency; Pancreatic Diseases; Digestive System Diseases