Liprotamase Efficacy Trial in Patients With Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency

Study Overview
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Brief Summary
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This is a clinical trial which will evaluate the efficacy of Liprotamase treatment in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (PI).

Detailed Description
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This trial is divided into four distinct periods: Baseline Period during which each patient is taken off pancreatic enzyme medications. An Open-Label Treatment Period during which all patients will receive ALTU-135 (liprotamase). Inpatient, Double Blind Treatment Period during which half of patients will be withdrawn from treatment and will receive Placebo. Second Open-Label Treatment Period during which all patients will resume treatment with ALTU-135 (liprotamase).

Official Title
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A Phase III, Randomized, Double-Blind, Placebo-Controlled Clinical Study Evaluating the Efficacy and Safety of ALTU-135 Treatment in Patients With Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency

Conditions
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Cystic Fibrosis, Exocrine Pancreatic Insufficiency

Intervention / Treatment
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* Drug: Liprotamase
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Females of childbearing potential must be willing to use birth control Diagnosis of CF based upon the following criteria: two clinical features consistent with CF; and either genotype with two identifiable mutations consistent with CF, OR sweat chloride >60 mEq/L by quantitative pilocarpine iontophoresis Clinically stable with no evidence of acute upper or lower respiratory tract infection PI determined by fecal elastase <100 µg/g stool measured at screening Able to take pancreatic enzyme supplementation in the form of capsules Able to perform the testing (e.g., stool collections) and inpatient stays required for this study, as judged by the Investigator Baseline coefficient of fat absorption (CFA) less than or equal to 80% Exclusion Criteria: CFA >80% at Baseline Pregnancy, breastfeeding or of childbearing potential and not willing to use birth control during the study History of fibrosing colonopathy History of liver transplant, lung transplant or significant surgical resection of the bowel Any acute or chronic diarrheal illness unrelated to PI Unable to discontinue enteral tube feedings during the study Known hypersensitivity to food additives Inability to consume the diet required by the study, in the judgment of the Investigator Participation in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to screening Abnormal liver function (except for patients with Gilbert Syndrome) Signs and/or symptoms of liver cirrhosis, portal hypertension or documented liver disease unrelated to CF Distal intestinal obstruction syndrome (DIOS) in the last six months prior to screening Unable to discontinue the use of pancreatic enzymes Any condition that the Investigator believes would interfere with the intent of this study or would make participation not in the best interest of the patient Patient is unlikely to complete the study, as determined by the Investigator

Ages Eligible for Study
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Minimum Age: 7 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Liprotamase<br>Liprotamase is a fixed combination of lipase (32,500 units), protease (25,000 units) and amylase (3,750 units). Open Label Period: Liprotamase administered orally with each of three meals and two snacks daily for 21 days. Double Blind Treatment Period: Administered orally with each of three meals and two snacks daily for 6 days. Second Open Label Period: Administered orally with each of three meals and two snacks daily for 7 days. | Drug: Liprotamase<br>* Administered orally<br>* Other names: TheraCLEC - Total;|
| Placebo Comparator: Placebo<br>Double Blind Treatment Period: Placebo (microcrystalline cellulose) administered orally with each of three meals and two snacks daily for 6 days. | Drug: Placebo<br>* Administered orally<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from open label baseline to end of 6 day double blind treatment period in Coefficient of Fat Absorption (CFA) |  | Open label baseline, end of 6 day double blind treatment period | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from open label baseline to end of 6 day double blind treatment period in Coefficient of nitrogen absorption (CNA) |  | Open label baseline, end of 6 day double blind treatment period | 
| Change from open label baseline to end of 6 day double blind treatment period in number of stools |  | Open label baseline, end of 6 day double blind treatment period | 
| Change from open label baseline to end of 6 day double blind treatment period in stool weight |  | Open label baseline, end of 6 day double blind treatment period | 
| Change from open label baseline to end of 6 day double blind treatment period in maximum blood glucose |  | Open label baseline, end of 6 day double blind treatment period | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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cystic fibrosis-related pancreatic insufficiency