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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03481803




Registration number
NCT03481803
Ethics application status
Date submitted
6/03/2018
Date registered
29/03/2018
Date last updated
28/09/2018

Titles & IDs
Public title
A Phase IIa Study With Escalating Dose of MS1819-SD
Scientific 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
Secondary ID [1] 0 0
MS1819/16/01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Chronic Pancreatitis 0 0
Distal Pancreatectomy 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - MS1819-SD

Treatment: Drugs: 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 code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Investigate safety of escalating doses of MS1819-SD as measured by number of participants with adverse events including clinical or laboratory abnormalities
Timepoint [1] 0 0
60 days
Secondary outcome [1] 0 0
Investigate the efficacy of MS1819-SD in patients by the Coefficient of Fat Absorption change from baseline
Timepoint [1] 0 0
60 days

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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),

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
27/01/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
29/06/2018
Sample size
Target
Accrual to date
Final
11
Recruitment in Australia
Recruitment state(s)
QLD,SA,WA
Recruitment hospital [1] 0 0
Mackay Institute of Research and Innovation - Mackay
Recruitment hospital [2] 0 0
CMAX - Adelaide
Recruitment hospital [3] 0 0
Linear Research - Perth
Recruitment postcode(s) [1] 0 0
4740 - Mackay
Recruitment postcode(s) [2] 0 0
5000 - Adelaide
Recruitment postcode(s) [3] 0 0
6009 - Perth
Recruitment outside Australia
Country [1] 0 0
France
State/province [1] 0 0
Marseille
Country [2] 0 0
New Zealand
State/province [2] 0 0
Christchurch
Country [3] 0 0
New Zealand
State/province [3] 0 0
Wellington

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
AzurRx SAS
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03481803
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03481803