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

Phase 3

Completed

Brief Summary: 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: This trial is divided into four distinct periods:

1. Baseline Period during which each patient is taken off pancreatic enzyme medications.

2. An Open-Label Treatment Period during which all patients will receive ALTU-135 (liprotamase).

3. Inpatient, Double Blind Treatment Period during which half of patients will be withdrawn from treatment and will receive Placebo.

4. Second Open-Label Treatment Period during which all patients will resume treatment with ALTU-135 (liprotamase).

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
Liprotamase	Liprotamase	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.
Placebo	Placebo	Double Blind Treatment Period: Placebo (microcrystalline cellulose) administered orally with each of three meals and two snacks daily for 6 days.

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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 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 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

Locations (1): For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.
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Milwaukee, Wisconsin, United States