Open Label Safety Study of DCI-1020 in Pediatric Cystic Fibrosis (CF) Patients

An Open-Label, Multi-Center Safety and Efficacy Study of DCI-1020 in Pediatric Cystic Fibrosis Patients With Exocrine Pancreatic Insufficiency

Hypothesis:DCI 1020 capsules are safe and effective in treating exocrine pancreatic insufficiency in CF patients <= 2 years of age.

The results of this study are intended to be submitted to the FDA as part of the NDA package for marketing approval of PANCRECARB (DCI 1020).

Study Overview

• Status: Withdrawn
• Conditions: Digestive System Diseases; Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Pancreatic Disease
• Intervention / Treatment: Drug: DCI 1020

Detailed Description

A large body of data supporting the safety and efficacy of PANCRECARB® (pancrelipase) Capsules is available in patients above age 2. This study is being performed to gather the data to demonstrate the safety and efficacy of DCI1020 in pediatric CF patients (≤ 2 years) with exocrine pancreatic insufficiency. This study also takes into consideration an "age appropriate" dosing form. Specifically, the enzymes will be administered orally by opening the capsules and emptying the microspheres into a small amount of applesauce as an example of a slightly acidic soft food such as jelly, jello, etc. for feeding.

The results of this study are intended to be submitted to the FDA as part of the NDA package for marketing approval of PANCRECARB® DCI 1020(pancrelipase).

• Study Type: Interventional
• Enrollment (Anticipated): 24
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • Rainbow Babies and Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 2 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Male or female age ≤ 2 years of age
• Confirmed diagnosis of CF based on the following criteria:
• One or more clinical features consistent with the CF phenotype, AND
• Positive sweat chloride ≥ 60 mEq/liter (by pilocarpine iontophoresis), OR
• Genotype with two identifiable mutations consistent with CF
• Adequate nutritional status
• Pancreatic insufficiency documented by spot fecal elastase-1 (FE 1) greater or equal to 100 micrograms/g stool
• Clinically stable with no evidence of an acute medical condition
• Parent/Guardian able to understand and sign a written informed consent and comply with the requirements of the study

Exclusion Criteria:

• History of fibrosing colonopathy
• History of being refractory to pancreatic enzyme replacement therapy
• Solid organ transplant
• History of intra-abdominal surgery
• A current diagnosis or a history of distal intestinal obstruction syndrome (DIOS) in the past six (6) months, or 2 or more episodes of DIOS in the past twelve (12) months
• Conditions known to increase fecal fat loss including: inflammatory bowel disease , celiac disease, Crohn's disease, tropical Sprue, Whipple's disease
• A known contraindication, sensitivity or hypersensitivity to porcine pancreatic enzymes
• Active liver disease with liver enzymes (alanine aminotransferase (ALT/SGPT), aspartate aminotransferase (AST/SGOT) or bilirubin ≥ 3 times the upper limit of normal
• Acute pancreatitis or acute exacerbation of chronic pancreatitis
• Antibiotic use as follows:
• Acute treatment with any systemic (oral or IV) antibiotics two (2) weeks prior to screening
• Treatment with erythromycin and unwilling to discontinue the treatment two (2) weeks prior to the screening. (azithromycin is allowed)
• Change in chronic treatment with systemic (oral and IV) antibiotics during the trial

NOTE:

Study patient may remain on a chronic regimen of systemic (oral or IV) antibiotics (with exception of erythromycin), if he/she started the antibiotics at least 2 weeks prior to study screening, was at his/her usual bowel pattern at the time of screening, and does not stop or change these antibiotics during the study.

• Receiving enteral tube feeding during the study
• Breast feeding during the study (expressed breast milk may be used, but not feeding at the breast)
• Expected inability to cooperate with or be non-adherent to required study procedures
• Use of narcotics
• Poorly controlled diabetes
• Participation in an investigational study of a drug, biologic, or device not currently approved for marketing, within 30 days of screening visit
• A medical condition which the investigator deems significant enough to interfere with the ability of the study patient to participate in the trial or interfering with assessment of effects of enzyme therapy on fat absorption

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: 1; DCI-1020 Capsules contain an enteric-coated buffered microspheres of pancrelipase, encapsulated in clear capsules. Capsules are equivalent to 4,000 USP units of lipase	Drug: DCI 1020; capsules (4,000 units of lipase) will be administered with meals and snacks

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Quantitative fecal fat content (%of fat/g of dry stool) in the spot stool samples collected over the 3 day In-home Treatment Period	3 consecutive days

Collaborators and Investigators

• Sponsor: Digestive Care, Inc.
• Investigators: Study Director: Tibor Sipos, PhD, DCI

Study record dates

Study Major Dates

• Study Start: February 1, 2009
• Primary Completion (ANTICIPATED): August 1, 2009
• Study Completion (ANTICIPATED): November 1, 2009

Study Registration Dates

• First Submitted: April 17, 2008
• First Submitted That Met QC Criteria: April 21, 2008
• First Posted (ESTIMATE): April 22, 2008

Study Record Updates

• Last Update Posted (ESTIMATE): May 13, 2010
• Last Update Submitted That Met QC Criteria: May 12, 2010
• Last Verified: May 1, 2010

More Information

Terms related to this study

• Keywords: pediatrics; cystic fibrosis; pancreatic insufficiency; pancreatic enzymes
• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Fibrosis; Gastrointestinal Diseases; Digestive System Diseases; Cystic Fibrosis; Pancreatic Diseases; Exocrine Pancreatic Insufficiency
• Other Study ID Numbers: DCI 07-001