A Phase IIa Study With Escalating Dose of MS1819-SD

NCT03481803 | Completed Phase 2

• 1 other identifier: MS1819/16/01
• Study Type: interventional
• Target: 11
• Locations: 3 countries
• Sites: 6

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.

Conditions

Chronic Pancreatitis; Distal Pancreatectomy

Keywords

Exocrine Pancreatic Insufficiency

Outcome Measures

Primary Outcomes (1)

• 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.

  60 days

Secondary Outcomes (1)

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

  60 days

Interventions

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.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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),

Sponsors & Collaborators

• AzurRx SAS: lead

Study Sites (6)

Mackay Institute of Research and Innovation

Mackay, Queensland, 4740, Australia

Location

CMAX

Adelaide, South Australia, 5000, Australia

Location

Linear Research

Perth, Western Australia, 6009, Australia

Location

Hôpital Timone Adulte (CIC-CPCET)

Marseille, 13385, France

Location

Christchurch Clinical Studies Trust

Christchurch, 8011, New Zealand

Location

P3 Research

Wellington, 6242, New Zealand

Location

MeSH Terms

Conditions

Pancreatitis, Chronic; Exocrine Pancreatic Insufficiency

Condition Hierarchy (Ancestors)

Pancreatitis; Pancreatic Diseases; Digestive System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Sequential Assignment Eligible patients will be receiving increased doses from lower, middle to upper range of study medication MS1819-SD

Study Record Dates

• First Submitted: March 6, 2018
• First Posted: March 29, 2018
• Study Start: January 27, 2017
• Primary Completion: June 29, 2018
• Study Completion: June 29, 2018
• Last Updated: September 28, 2018

Record last verified: 2018-09

Locations

FR (1); NZ (2); AU (3)