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

Anthera Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Cystic Fibrosis; Exocrine Pancreatic Insufficiency

Treatments: Drug: Placebo; Drug: Liprotamase

Study type: Interventional

Funder types: Industry

Identifiers

NCT00449878

14293

726 (Other Identifier)

I5L-MC-TCAB (Other Identifier)

Details and patient eligibility

About

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

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 Inpatient, Double Blind Treatment Period during which half of patients will be withdrawn from treatment and will receive Placebo.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

Enrollment: 163 patients

Sex: All

Ages: 7+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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163 participants in 2 patient groups, including a placebo group

Liprotamase

Experimental group

Description:

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.

Treatment:

Drug: Liprotamase

Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo