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

NCT ID: NCT00449878

Last Updated: 2014-10-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

163 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-05-31

Study Completion Date

2008-06-30

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:

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 Inpatient, Double Blind Treatment Period during which half of patients will be withdrawn from treatment and will receive Placebo.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

Conditions

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Cystic Fibrosis Exocrine Pancreatic Insufficiency

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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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.

Group Type EXPERIMENTAL

Liprotamase

Intervention Type DRUG

Administered orally

Placebo

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Administered orally

Interventions

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Liprotamase

Administered orally

Intervention Type DRUG

Placebo

Administered orally

Intervention Type DRUG

Other Intervention Names

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ALTU-135 LY3031642 TheraCLEC - Total

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
Minimum Eligible Age

7 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Anthera Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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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.

Los Angeles, California, United States

Site Status

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Stanford, California, United States

Site Status

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Denver, Colorado, United States

Site Status

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Jacksonville, Florida, United States

Site Status

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Miami, Florida, United States

Site Status

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Atlanta, Georgia, United States

Site Status

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Glenview, Illinois, United States

Site Status

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Indianapolis, Indiana, United States

Site Status

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Iowa City, Iowa, United States

Site Status

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Ann Arbor, Michigan, United States

Site Status

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Detroit, Michigan, United States

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Grand Rapids, Michigan, United States

Site Status

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Kalamazoo, Michigan, United States

Site Status

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Minneapolis, Minnesota, United States

Site Status

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Jackson, Mississippi, United States

Site Status

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Hanover, New Hampshire, United States

Site Status

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Buffalo, New York, United States

Site Status

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Durham, North Carolina, United States

Site Status

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Cincinnati, Ohio, United States

Site Status

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Cleveland, Ohio, United States

Site Status

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Dayton, Ohio, United States

Site Status

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Portland, Oregon, United States

Site Status

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Pittsburgh, Pennsylvania, United States

Site Status

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Sioux Falls, South Dakota, United States

Site Status

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Memphis, Tennessee, United States

Site Status

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.

Salt Lake City, Utah, United States

Site Status

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.

Morgantown, West Virginia, United States

Site Status

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Madison, Wisconsin, United States

Site Status

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.

Milwaukee, Wisconsin, United States

Site Status

Countries

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United States

References

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Somaraju URR, Solis-Moya A. Pancreatic enzyme replacement therapy for people with cystic fibrosis. Cochrane Database Syst Rev. 2020 Aug 5;8(8):CD008227. doi: 10.1002/14651858.CD008227.pub4.

Reference Type DERIVED
PMID: 32761612 (View on PubMed)

Other Identifiers

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I5L-MC-TCAB

Identifier Type: OTHER

Identifier Source: secondary_id

726

Identifier Type: OTHER

Identifier Source: secondary_id

14293

Identifier Type: -

Identifier Source: org_study_id

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