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

Cystic Fibrosis Clinical Trial

— DIGEST  

Official title:

A Phase III, Randomized, Double-Blind, Placebo-Controlled Clinical Study Evaluating the Efficacy and Safety of ALTU-135 Treatment in Patients With Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00449878
• Other study ID #: 14293
• Secondary ID: I5L-MC-TCAB726
• Status: Completed
• Phase: Phase 3
• First received: March 19, 2007
• Last updated: October 15, 2014
• Start date: May 2007
• Est. completion date: June 2008

Study information

• Verified date: October 2014
• Source: Anthera Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationArgentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia MedicaItaly: The Italian Medicines AgencyPoland: Office for Registration of Medicinal Products, Medical Devices and Biocidal ProductsSerbia and Montenegro: Agency for Drugs and Medicinal DevicesSlovakia: State Institute for Drug ControlRussia: Ministry of Health of the Russian Federation
• Study type: Interventional

Clinical Trial Summary

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

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 Inpatient, Double Blind Treatment Period during which half of patients will be withdrawn from treatment and will receive Placebo.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

• Status: Completed
• Enrollment: 163
• Est. completion date: June 2008
• Est. primary completion date: June 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 7 Years and older

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

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Exocrine Pancreatic Insufficiency
• Fibrosis

Intervention

Drug:
• Liprotamase
Administered orally
• Placebo
Administered orally

Locations

Country	Name	City	State
United States	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.	Ann Arbor	Michigan
United States	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.	Atlanta	Georgia
United States	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.	Buffalo	New York
United States	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.	Cincinnati	Ohio
United States	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.	Cleveland	Ohio
United States	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.	Dayton	Ohio
United States	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.	Denver	Colorado
United States	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.	Detroit	Michigan
United States	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.	Durham	North Carolina
United States	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.	Glenview	Illinois
United States	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.	Grand Rapids	Michigan
United States	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.	Hanover	New Hampshire
United States	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.	Indianapolis	Indiana
United States	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.	Iowa City	Iowa
United States	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.	Jackson	Mississippi
United States	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.	Jacksonville	Florida
United States	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.	Kalamazoo	Michigan
United States	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	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.	Madison	Wisconsin
United States	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.	Memphis	Tennessee
United States	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.	Miami	Florida
United States	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	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.	Minneapolis	Minnesota
United States	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	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.	Pittsburgh	Pennsylvania
United States	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.	Portland	Oregon
United States	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	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.	Sioux Falls	South Dakota
United States	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.	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
Anthera Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Secondary	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	No
Secondary	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	No
Secondary	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	No
Secondary	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	No