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

Anthera Pharmaceuticals214 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

214

Conditions

Cystic Fibrosis Exocrine Pancreatic Insufficiency

Interventions

LiprotamaseDRUG

Administered orally

Eligibility

Sex: ALLMin age: 7 Years

Medical Language ↔ Plain English

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

Locations (39)

Outcomes

Primary Outcomes

Number of participants with an adverse event

Time frame: Baseline up to 12 months

Secondary Outcomes

Change from baseline up to 12 months in body weight Z-scores

Time frame: Baseline, up to 12 months

Change from baseline up to 12 months in height Z-scores (up to 17 years in participants age)

Time frame: Baseline, up to 12 months

Change from baseline up to 12 months in Body Mass Index (BMI) Z-scores

Time frame: Baseline, up to 12 months

Data from ClinicalTrials.gov

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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.

Oakland, California, United States

Denver, Colorado, United States

Jacksonville, Florida, United States

Miami, Florida, United States

Atlanta, Georgia, United States

Augusta, Georgia, United States

Boise, Idaho, United States

Chicago, Illinois, United States

Glenview, Illinois, United States

Niles, Illinois, United States

...and 29 more locations