Study to Evaluate the Safety and Efficacy of EUR-1008 (APT-1008) Pancreatic Enzyme Product in Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT00297167
• Date of registration: 27/02/2006
• Prospective Registration: Yes
• Primary sponsor: Forest Laboratories
• Public title: Study to Evaluate the Safety and Efficacy of EUR-1008 (APT-1008) Pancreatic Enzyme Product in Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency
• Scientific title: A Randomized, Double-Blind, Placebo-Controlled, Two-Treatment, Crossover Study to Evaluate the Safety and Efficacy of Eurand Pancreatic Enzyme Product (PEP) in Patients With Cystic Fibrosis and Exocrine Pancreatic Insufficiency
• Date of first enrolment: May 2006
• Target sample size: 34
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00297167
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Double.
• Phase: Phase 3

Countries of recruitment

United States

Contacts

• Name: Aptalis Medical Information
• Affiliation: Forest Laboratories

Key inclusion & exclusion criteria

Inclusion Criteria:

- Participants with age greater than or equal to (>=) 7 years at the time of enrollment

- Participants with weight 70 kg or less and be in an adequate nutritional status as
indicated by a body mass index (BMI) >=20 kg/m^2 for ages 18 and above, or a BMI above
the twenty fifth percentile for participants aged 7 to 17 years

- Participants with confirmed diagnosis of CF who have 2 clinical features consistent
with CF, and have either a genotype with 2 identifiable mutations consistent with CF
or a sweat chloride concentration that is more than 60 milliequivalent per liter by
quantitative pilocarpine iontophoresis

- Participants with confirmed diagnosis of EPI who are currently receiving treatment
with a commercially available PEP and have documented with a fecal elastase of <100
microgram per gram stool (if no documentation was available, a stool sample was taken
at Screening for determination of fecal elastase).

- Clinically stable participants with no evidence of acute respiratory disease or any
other acute condition

- Participants who are willing and able to interrupt current CF treatment for CF-related
malabsorption along with any medications that may affect gastric motility or stomach
power of hydrogen (pH)

- Participants 18 years of age and older had to a) understand the requirements of the
study, b) provide written informed consent, c) agree to abide by the study
restrictions, and d) return for the required assessments

- Participants 7 to 17 years of age must have a parent(s) or legal guardian who provides
written informed consent, agree to abide by the study restrictions

- Females participants of childbearing potential must have a negative serum pregnancy
test at screening and must agree to use adequate birth control during the study

Exclusion Criteria:

- Participants with fibrosing colonopathy, hyperuricemia or hyperuricosuria

- Participants who are allergic to pork or other porcine PEPs

- Participants with forced expiratory volume (FEV1) <30 percent of predicted FEV1 at
screening

- Participants with any acute systemic administration of an antibiotic for any reason in
the previous 4 weeks; however, a low stable dose of an antibiotic or chronic treatment
with an inhalatory antibiotic is allowed

- Participants with hepatic insufficiency as defined by history or presence of ascites
or serum albumin level of < 3.0 milligram/deciliter, or a coagulopathy with an
international normalized ratio that is greater than 1.7

- Participants who have used an acute dose of any steroid in the previous 2 weeks;
however, low chronic doses of a steroid is allowed

- Participants with history of or current diagnosis of distal ileal obstruction syndrome
(DIOS) as evidenced or suggested by constipation, abdominal pain, anorexia, early
satiety, recurrent vomiting, and palpable fecal mass on physical examination (the
absence of DIOS will be confirmed by an X-ray of the abdomen taken at screening)

- Participants with any solid organ transplant or surgery affecting the bowel.
Participants with a history of appendectomy and inguinal (non-incarcerated)
hernioplasty or meconium ileus without the need for bowel resection could be enrolled.
Gastrointestinal-tube-fed patients, in absence of dumping syndrome, were also eligible

- Participants with history of or current screening evaluation of hyperglycemia as
defined by an 8-hour fasting blood glucose (FBG) of >126 mg/dL, or of CF-related
diabetes as determined according to the Cystic Fibrosis Foundation (CFF) Consensus
Conference of January 1999 (Section IX Part II), that is: a) FBG >126 mg/dL (7.0
millimoles per liter [mmol/L]) on two or more occasions b)FBG >126 mg/dL (7.0 mmol/L)
plus casual (without regard to time of day or last meal consumed) glucose level >200
mg/dL (11.1 mmol/L) c)Casual (previously called random) glucose levels >200 mg/dL
(11.1 mmol/L) on two or more occasions with symptoms

- Participants using an enzyme preparation in excess of 10,000-lipase units/kg/day

- Participants using an immunosuppressive drug

- Participants who are expecting an inability to tolerate the washout period and/or the
placebo treatment

- Participants participating in an investigational study of a drug, biologic, or device
not currently approved for marketing, within 30 days of screening visit

- Female participants who are pregnant or breastfeeding, or unwilling to use effective
birth control during study

- Participants with any condition that would, in the investigator's opinion, limit the
participant's ability to complete the study



Age minimum: 7 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Exocrine Pancreatic Insufficiency

Cystic Fibrosis

Intervention(s)

Drug: Placebo

Drug: EUR-1008 (APT-1008)

Primary Outcome(s)

Percent Coefficient of Fat Absorption (CFA%) [Time Frame: Day 3 up to Day 6 of hospital treatment in first and second double-blind intervention periods]

Secondary Outcome(s)

Mean Daily Number of Stools [Time Frame: Day 3 up to Day 6 during first and second double-blind intervention periods]

Vitamin E Levels [Time Frame: End of treatment (Day 6 during first and second double-blind intervention periods)]

Mean Number of Abdominal Symptoms [Time Frame: Day 3 up to Day 6 during first and second double-blind intervention periods]

Percent Coefficient of Nitrogen Absorption (CNA%) [Time Frame: Day 3 up to Day 6 of hospital treatment in first and second double-blind intervention periods]

Vitamin A Levels [Time Frame: End of treatment (Day 6 during first and second double-blind intervention periods)]

Lipid Levels [Time Frame: End of treatment (Day 6 during first and second double-blind intervention periods)]

Percentage of Stool Categorized as Per Consistency [Time Frame: Day 3 up to Day 6 during first and second double-blind intervention periods]

Secondary ID(s)

EUR-1008-M

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 04/04/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00297167