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Trial record 3 of 4 for:    MS1819

Phase 2 Combination Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs

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ClinicalTrials.gov Identifier: NCT04302662
Recruitment Status : Completed
First Posted : March 10, 2020
Last Update Posted : August 19, 2021
Sponsor:
Information provided by (Responsible Party):
AzurRx SAS

Tracking Information
First Submitted Date  ICMJE March 4, 2020
First Posted Date  ICMJE March 10, 2020
Last Update Posted Date August 19, 2021
Actual Study Start Date  ICMJE June 27, 2019
Actual Primary Completion Date June 3, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 9, 2020)
  • Coefficient of fat absorption [ Time Frame: 15 days ]
    determination of fat absorption based on fat intake and fat excretion over 3 days on high fat meal
  • Adverse Events [ Time Frame: 81 days ]
    AE, SAE, SUSAR, immunoallergic reactions
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 9, 2020)
  • Weight of stools [ Time Frame: 15 days ]
    evaluation of changes in weight of stools from baseline (PPEs only) to each treatment period
  • number of daily evacuations [ Time Frame: 15 days ]
    evaluation of changes in daily evacuations from baseline (PPEs only) to each treatment period
  • Steatorrhea, [ Time Frame: 15 days ]
    evaluation of changes in steatorrhea from baseline (PPEs only) to each treatment period
  • Creatorrhea [ Time Frame: 15 days ]
    evaluation of changes in creatorrhea from baseline (PPEs only) to each treatment period
  • Body weight [ Time Frame: 15 days ]
    evaluation of changes in body weight from baseline (PPEs) to each treatment period
  • Consistency of stools [ Time Frame: 15 days ]
    evaluation of consistency of stools from baseline (PPEs) to each treatment period
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 2 Combination Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs
Official Title  ICMJE A Multicenter, Open-label Phase 2 Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs, to Investigate the Efficacy and Safety of This Combination for the Compensation of Severe Exocrine Pancreatic Insufficiency in CF Patients Not Fully Compensated With Only PPEs
Brief Summary

This is a Phase 2 study sponsored by AzurRx SAS and involves testing of a new medication for the compensation of exocrine pancreatic insufficiency (EPI) caused by cystic fibrosis (CF). 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 efficacy and safety of escalating doses of study drug on top of a stable dose of PPEs in CF patients who are not fully compensated by PPEs only.

This enzyme has demonstrated an appropriate profile to compensate the pancreatic lipase (enzyme) deficiency that is common in CP (chronic pancreatitis) and CF patients.

The design of the study is open-label, meaning that all eligible patients will receive the study drug MS1819-SD. The study drug dose will increase throughout the study during dose escalation visits in each treatment period; study includes a total of three treatment periods.

The total duration of the MS1819-SD treatment phase is of 39-51 days. The total duration of patient participation in the study is of 69-81 days. Approximately 24 patients will be enrolled in this study.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE 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 MS1819-SD on top of a stable dose of PPEs
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Cystic Fibrosis
  • Cystic Fibrosis Gastrointestinal Disease
  • Cystic Fibrosis of Pancreas
Intervention  ICMJE Drug: MS1819-SD
Patients will receive increasing doses from the lowest to a maximum dose of MS1819-SD on top of a stable dose of PPEs. The total treatment phase will range from 39 to 51 days.
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 18, 2021)
20
Original Estimated Enrollment  ICMJE
 (submitted: March 9, 2020)
24
Actual Study Completion Date  ICMJE June 3, 2021
Actual Primary Completion Date June 3, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Signed and dated informed consent form.
  2. Age > 12 years at the time of screening
  3. Male or female.
  4. Under stable dose of PPE ≥ 1 month. Stable dose is defined as dose of medication not changed during this time period and the medication must be commercially available and be administered in the recommended dose range.
  5. A nutritional status as defined by:

    1. BMI ≤ 22.0 kg/m2 for female patients
    2. BMI ≤ 23.0 kg/m2 for male patients
    3. BMI ≤ 50th percentile for patients 12 to < 18 years of age.
  6. Cystic fibrosis, based on 2 clinical features consistent with CF in the opinion of the investigator AND sweat chloride concentration > 60 mmol/L by pilocarpine iontophoresis.
  7. Faecal pancreatic elastase-1 < 100 µg/g of stools at screening.
  8. Baseline CFA < 80% with a maximum daily dose of 10,000 lipase units/kg/day.
  9. Clinically stable with no documented evidence of significant respiratory symptoms that would require administration of intravenous antibiotics, oxygen supplementation, or hospitalization within the 30 days of screening.
  10. Male and female patients, if of childbearing potential, must use a reliable method of contraception during the study. A reliable method of birth control is defined as one of the following: oral or injectable contraceptives, intrauterine device, contraceptive implants, tubal ligation, hysterectomy, or a double-barrier method (diaphragm with spermicidal foam or jelly, or a condom), abstinence or vasectomy. Periodic abstinence (calendar, symptothermal, or post-ovulation methods) is not an acceptable method of contraception. The preferred and usual lifestyle of the patient must also be evaluated in determining if sexual abstinence is a reliable method of birth control.
  11. Be considered as reliable and capable of adhering to the protocol, according to the judgment of the investigator.

Exclusion Criteria:

  1. Established or suspected fibrosing colonopathy.
  2. Total or partial gastrectomy.
  3. A history of solid organ transplant or significant surgical resection of the bowel; significant resection of the bowel is defined as any resection of the terminal ileum or ileocecal valve. Patients who have had qualitative, long-term changes in nutritional status after any other bowel resection (eg, increased of new need for pancreatic enzyme supplementation compared with preoperative status to maintain the same nutritional status) should also be excluded.
  4. Any chronic diarrheal illness unrelated to pancreatic insufficiency (eg, infectious gastroenteritis, sprue, inflammatory bowel disease)
  5. Known hypersensitivity or other severe reaction to any ingredient of the investigational medicinal product (IMP).
  6. Bilirubin > 1.5 times upper limit normal (ULN).
  7. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 5 times ULN.
  8. Alkaline phosphatase (ALP) > 5 times ULN.
  9. Gamma glutamyltransferase (GGT) > 5 times ULN.
  10. Signs and/or symtoms of liver cirrhosis or portal hypertension (eg, splenomegaly, ascites, esophageal varices), or documented liver disease unrelated to CF
  11. Patients with a known allergy to the stool marker.
  12. Feeding via an enteral tube during 6 months before screening
  13. Routine use of anti-diarrheals, anti-spasmodics, or cathartic laxatives, or a change in chronic osmotic laxatives (eg, polyethylene glycol) regimen in the previous laxative therapy within the last 12 months before screening
  14. History of severe constipation with < 1 evacuation/week under appropriate laxative therapy within the last 12 months before screening.
  15. Documentation of distal intestinal pseudo-obstruction syndrome within the last 12 months before screening.
  16. Forced Expiratory Volume ≤ 30% at the screening visit.
  17. Lactation or known pregnancy or positive pregnancy test at both screening and baseline for women of childbearing potential.
  18. Participation in another clinical study involving an IMP within 30 days before inclusion or concomitantly with this study.
  19. Poorly controlled diabetes according the investigator's judgement.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Hungary,   Turkey
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04302662
Other Study ID Numbers  ICMJE MS1819/18/02
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party AzurRx SAS
Study Sponsor  ICMJE AzurRx SAS
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account AzurRx SAS
Verification Date August 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP