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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04302662
Recruitment Status : Recruiting
First Posted : March 10, 2020
Last Update Posted : March 10, 2020
Sponsor:
Information provided by (Responsible Party):
AzurRx SAS

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.


Condition or disease Intervention/treatment Phase
Cystic Fibrosis Cystic Fibrosis Gastrointestinal Disease Cystic Fibrosis of Pancreas Drug: MS1819-SD Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 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 MS1819-SD on top of a stable dose of PPEs
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : June 27, 2019
Estimated Primary Completion Date : December 31, 2020
Estimated Study Completion Date : December 31, 2020

Resource links provided by the National Library of Medicine



Intervention Details:
  • 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.


Primary Outcome Measures :
  1. 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

  2. Adverse Events [ Time Frame: 81 days ]
    AE, SAE, SUSAR, immunoallergic reactions


Secondary Outcome Measures :
  1. Weight of stools [ Time Frame: 15 days ]
    evaluation of changes in weight of stools from baseline (PPEs only) to each treatment period

  2. number of daily evacuations [ Time Frame: 15 days ]
    evaluation of changes in daily evacuations from baseline (PPEs only) to each treatment period

  3. Steatorrhea, [ Time Frame: 15 days ]
    evaluation of changes in steatorrhea from baseline (PPEs only) to each treatment period

  4. Creatorrhea [ Time Frame: 15 days ]
    evaluation of changes in creatorrhea from baseline (PPEs only) to each treatment period

  5. Body weight [ Time Frame: 15 days ]
    evaluation of changes in body weight from baseline (PPEs) to each treatment period

  6. Consistency of stools [ Time Frame: 15 days ]
    evaluation of consistency of stools from baseline (PPEs) to each treatment period



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04302662


Contacts
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Contact: Katalin Birki +36 20-927-5060 kbirki@zeincro.com

Locations
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Hungary
Országos Korányi TBC és Pulmonológiai Intézet Cisztás Fibrózis Részleg Recruiting
Budapest, Hungary, 1122
Contact: Adrien Halász, MD    0036 20-579-5555    halaszadr@gmail.com   
Borsod-Abaúj-Zemplén Megyei Központi Kórház és Egyetemi Oktatókórház, Gyermekegészségügyi Központ Active, not recruiting
Miskolc, Hungary, 3526
Somogy Megyei Kaposi Mór Oktató Kórház, Mosdósi telephelye Mosdósi Gyermekrehabilitációs és Gyermekpulmonológiai Egység Recruiting
Mosdós, Hungary, 7257
Contact: Gyöngyi Székely, MD    0036 30-373-3151    sz.gyongyi58@gmail.com   
Tüdőgyógyintézet Törökbálint Gyermekpulmonológiai Osztály és Szakrendelés Active, not recruiting
Törökbálint, Hungary, 2045
Sponsors and Collaborators
AzurRx SAS
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Responsible Party: AzurRx SAS
ClinicalTrials.gov Identifier: NCT04302662    
Other Study ID Numbers: MS1819/18/02
First Posted: March 10, 2020    Key Record Dates
Last Update Posted: March 10, 2020
Last Verified: March 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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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:
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Cystic Fibrosis
Gastrointestinal Diseases
Digestive System Diseases
Exocrine Pancreatic Insufficiency
Fibrosis
Pathologic Processes
Pancreatic Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases