A Phase IIa Study With Escalating Dose of MS1819-SD

NCT ID: NCT03481803

Last Updated: 2018-09-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-01-27
• Study Completion Date: 2018-06-29

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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Interventions

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.

Intervention Type: DRUG

Eligibility 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),

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

AzurRx SAS

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Mackay Institute of Research and Innovation

Mackay, Queensland, Australia

CMAX

Adelaide, South Australia, Australia

Linear Research

Perth, Western Australia, Australia

Hôpital Timone Adulte (CIC-CPCET)

Marseille, , France

Christchurch Clinical Studies Trust

Christchurch, , New Zealand

P3 Research

Wellington, , New Zealand

Countries

Australia; France; New Zealand

Other Identifiers

MS1819/16/01

Identifier Type: -

Identifier Source: org_study_id