Open-Label Phase III Long-Term Safety Trial of Liprotamase

Study Overview
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Brief Summary
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This is an open-label, single-arm clinical study investigating the long-term safety of ALTU-135 treatment in patients with cystic fibrosis (CF)-related exocrine pancreatic insufficiency (PI).

Official Title
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An Open-Label Clinical Study Evaluating the Long-Term Safety of ALTU-135 for the Treatment of 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


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 A genotype with two identifiable mutations consistent with CF, or Sweat chloride > 60 milliequivalents per liter (mEq/L) by quantitative pilocarpine iontophoresis. Clinically stable with no evidence of acute upper or lower respiratory tract infection Exocrine pancreatic insufficiency (PI) determined by fecal elastase < 100 micrograms per gram (µg/g) measured at Screening Able to take pancreatic enzyme supplementation in the form of capsules Able to perform the testing and procedures required for this study, as judged by the Investigator Exclusion Criteria: Age <7 years Pregnancy, breastfeeding or of childbearing potential and not willing to use birth control Participation in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to Screening History of fibrosing colonopathy History of liver transplant, lung transplant or significant surgical resection of the bowel Any chronic diarrheal illness unrelated to PI Abnormal liver function (except for patients with Gilbert Syndrome) Signs and/or symptoms of liver cirrhosis or portal hypertension (e.g., splenomegaly, ascites, esophageal varices), or documented liver disease unrelated to CF Unable to discontinue enteral tube feedings during the study 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 Baseline coefficient of fat absorption (CFA) ≥ 93% from the Phase III efficacy study 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: Single Group Assignment
Masking: None (Open Label)


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) administered orally with each of three meals and two snacks daily for 12 months. | Drug: Liprotamase<br>* Administered orally<br>* Other names: TheraCLEC-total;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with an adverse event |  | Baseline up to 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline up to 12 months in body weight Z-scores |  | Baseline, up to 12 months | 
| Change from baseline up to 12 months in height Z-scores (up to 17 years in participants age) |  | Baseline, up to 12 months | 
| Change from baseline up to 12 months in Body Mass Index (BMI) Z-scores |  | Baseline, up to 12 months | 

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