Cystic FibrosisPhase 3NCT00981214
Study of Pancreatic Enzyme Product in Pediatric Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency
A Phase 3 study testing EUR-1008 (APT-1008) in people with Cystic Fibrosis and Exocrine Pancreatic Insufficiency, run by Forest Laboratories at 14 sites in United States.
Trial summary
This is an open-label study to evaluate the efficacy and safety of Aptalis' (formerly Eurand) pancreatic enzyme product (PEP) microtabs in pediatric participants under age 7 with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI). Read more about this study
- Who is it for?
- Ages Up to 7 years
- Conditions the study is for
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- What is being tested?
- EUR-1008 (APT-1008)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 14 sitesUnited States
- Study dates
- May 2006to Sep 2006
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 7 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participants less than 7 years of age
- Participants who have pancreatic insufficiency documented by a fecal elastase level less than 100 micrograms per gram (mcg/g), or if not documented, the fecal elastase test must be done at the screening visit
- Participants who have a need of de novo treatment with pancreatic enzymes or be able to be switched from an existing treatment
- Participants who have a body mass index greater than the twenty fifth percentile for children 2 years and older
- Participants with a weight for height index greater than the twenty fifth percentile for children less than 2 years of age
- Participants with diagnosis of CF based upon the following criteria:
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- 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 (mEq/L) by quantitative pilocarpine iontophoresis
- Participants who are clinically stable with no evidence of acute upper or lower respiratory tract infection
Who cannot join
- Participants with fibrosing colonopathy
- Participants allergic to pork or other porcine PEPs
- Participants with any respiratory condition that in the investigator's opinion would result in an intervention requiring hospitalization or intensive pulmonary treatment during the trial
- 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 (such as azithromycin 250 or 500 milligram [mg] up to 3 times per week) is allowed. Moreover, chronic treatment (that is, daily for at least 1 month) with an inhalatory antibiotic (for example, colistin, tobramycin, or ceftazidime) is allowed
- Participants who have hepatic insufficiency as defined by a history or presence of ascites, or a serum albumin level of less than 3.0 milligram per deciliter (mg/dL), or coagulopathy with an international normalized ratio that is greater than 1.7
- Participants with hyperuricemia or hyperuricosuria
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- Participants participating in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to screening visit
- Participants with history of or current screening evaluation of hyperglycemia as defined by an 8-hour fasting serum glucose equivalent to 126 mg/dL or more, or of cystic-fibrosis-related diabetes as determined according to the Cystic Fibrosis Foundation (CFF) Consensus Conference of January 1999 (Section IX Part II), that is:
- Fasting Blood Glucose (FBG) greater than126 mg/dl (7.0 milli mole [mM]) on two or more occasions
- FBG greater than 126 mg/dl (7 .0 mM) plus casual (without regard to time of day or last meal consumed) glucose level greater than200 mg/dl (11.1 mM)
- Casual (previously called random) glucose levels greater than 200 mg/dl (11.1 mM) on two or more occasions with symptoms
- Participants with any solid organ transplant or surgery affecting the bowel
- Participants using an enzyme preparation in excess of 10,000 lipase units/kg/day
- Participants with an acute dose of any steroid in the previous 2 weeks; however, low chronic doses of a steroid (less 0.5 mg/kg every other day) will be allowed
- Participants with any condition that would, in the investigator's opinion, limit the patient's ability to complete the study
- Participants with history of or current screening determination of distal ileal obstruction syndrome (DIOS), or any clinical signs and symptoms suggestive of DIOS (that is, constipation, abdominal pain, anorexia, early satiety, recurrent vomiting and palpable fecal mass) on physical examination
- Participants who are unable to discontinue excluded concomitant medications over the course of the study
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
United States14 sites
University of Michigan, Cystic Fibrosis Center
Ann Arbor, Michigan, 48109
University of Alabama
Birmingham, Alabama, 35294
Childrens Memorial Hospital
Chicago, Illinois, 60614
Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Florida College of Medicine
Gainsville, Florida, 32610-0296
University of Iowa
Iowa City, Iowa, 52242
Nemours Childrens Clinic
Jacksonville, Florida, 32250
Children's Hospital of Los Angeles
Los Angeles, California, 90027
West Virginia Health Sciences Center
Morgantown, West Virginia, 26506
Children's Hospital - Oakland
Oakland, California, 94609
Stanford University Medical Center
Palo Alto, California, 94304
University of Utah
Salt Lake City, Utah, 84108
Children's Hospital of San Diego
San Diego, California, 92123
University of Texas
Tyler, Texas, 75708
About this study
Official title: An Open-Label Study to Evaluate the Efficacy and Safety of Pancreatic Enzyme Product (PEP) Microtabs in Pediatric Patients With Cystic Fibrosis and Exocrine Pancreatic Insufficiency
This is an open-label study to evaluate the efficacy and safety of Aptalis' (formerly Eurand) pancreatic enzyme product (PEP) microtabs in pediatric participants under age 7 with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI).
Read the detailed description
The study sample will consist of evaluable participants, all of whom will be children younger than 7 years of age. Participants will receive EUR-1008 (APT-1008) Microtabs formulation. The study design involves a 4-day screening period, a 7-day dose stabilization period, and a 7-day treatment period (excluding an end-of-study evaluation).
The optimal dose of EUR-1008 (APT-1008) Microtabs, determined during the dose stabilization period, will be used during the treatment period. Participants are instructed to consume a predefined diet.
- Conditions
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- Being tested
- EUR-1008 (APT-1008)
- Keywords
- Cystic fibrosis, Exocrine Pancreatic Insufficiency, Zenpep-1009
- Medical subject headings
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EUR-1008 (APT-1008)Drug
Also called ZENPEP®, Pancrelipase
EUR-1008 (APT-1008) Microtabs contained in a capsule will be administered orally from Day 5 to Day 11 at an enzyme dose based on investigator's discretion, in dose stabilization period or the content of the capsule will be allowed to sprinkle on food, where necessary, followed by stabilized dose from Day 12 to Day 18 in treatment period, up to a maximum total dose of 10,000 lipase units per kilogram body weight per day (unit/kg/day).
| Study group | Role | What they receive |
|---|---|---|
| EUR-1008 (APT-1008) | Experimental | Not described Receives: Drug: EUR-1008 (APT-1008) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Who Were Responders After 1 Week of Treatment With Study Medication
Responders were defined as those participants without steatorrhea (defined as less than 30 percent (%) fecal fat content) and without signs and symptoms of malabsorption after 1 week of treatment with study medication.
Measured: Day 11
Percentage of Participants Who Were Responders After 2 Weeks of Treatment With Study Medication
Responders were defined as those participants without steatorrhea (defined as less than 30% fecal fat content) and without signs and symptoms of malabsorption after 2 weeks of treatment with study medication.
Measured: Day 18 (end of treatment)
Secondary outcomes (9)
Change From Baseline in Weight at Day 12, 19
Measured: Baseline, Day 12, 19
Mean Daily Number of Stools
Mean daily number of stools of each participant was calculated from frequency of stools by the participant per day. Mean daily number of stools at each period for total participants was summarized.
Measured: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)
Percentage of Stool Categorized by Consistency
Stool consistency was categorized as hard, formed/normal, soft, watery or overt diarrhea. Percentage of stools of a specific consistency of each participant was calculated as the number of stools with a specific consistency relative to the total number of stools during the collection period. Mean percentage of stool with specific consistency at each period for total participants was summarized.
Measured: Baseline, Day 5 up to Day 11 (dose stabilization period) and Day 12 up to Day 18 (treatment period)
Mean Number of Abdominal Symptoms: Bloating
Bloating is swelling of the intestinal tract caused by excessive gas formation. Symptoms of bloating were classified by severity as mild (no impairment of daily activities), moderate (slight impairment of daily activities), or severe (unable to perform daily activities). Mean number of symptoms of specific severity for each participant was calculated from frequency of symptoms by the participant per day. Mean number of symptoms at each period for total participants was summarized.
Measured: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)
Mean Number of Abdominal Symptoms: Flatulence
Flatulence is presence of excessive gas in the digestive tract. Symptoms of flatulence was classified by severity as mild (no impairment of daily activities), moderate (slight impairment of daily activities), or severe (unable to perform daily activities). Mean number of symptoms of specific severity for each participant was calculated from frequency of symptoms by the participant per day. Mean number of symptoms at each period for total participants was summarized.
Measured: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)
Mean Number of Pain Symptoms
Symptoms of pain was classified by severity as mild (no impairment of daily activities), moderate (slight impairment of daily activities), or severe (unable to perform daily activities). Mean number of symptoms of specific severity for each participant was calculated from frequency of symptoms by the participant per day. Mean number of symptoms at each period for total participants was summarized.
Measured: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)
Physician's and Parent's or Legal Guardians Assessment of Improvement in Clinical Symptoms
Clinical symptoms of exocrine pancreatic insufficiency (EPI) were assessed by the physician and parent or guardian to determine if the participant showed improvement in symptoms of EPI at end of study after the dose stabilization period. EPI is a syndrome characterized by clinical symptoms of poor absorption of fats, proteins, and to a lesser extent, carbohydrates, which manifests primarily in patients with cystic fibrosis. Number of participants with improvement in clinical symptoms was reported.
Measured: Day 19 (end of study)
Percentage of Blood in Stool
Mean percentage of stools with blood at each period for total participants was summarized.
Measured: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)
Percentage of Stool With Visible Oil or Grease
Mean percentage of oil or grease at each period for total participants was summarized.
Measured: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2009 (estimated)
- Study startMay 2006
- Primary completionSep 2006
- Study completionSep 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Forest Laboratories
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Aptalis Medical Information, Study director, Forest Laboratories
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00981214
- Sponsor's protocol ID
- EUR-1009-M
- Status last verified
- Feb 2017
- First submitted
- 21 Sep 2009
- Last updated
- 16 Mar 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study to Evaluate the Efficacy and Safety of Pancreatic Enzyme Product (PEP) Microtabs in Pediatric Patients With Cystic Fibrosis and Exocrine Pancreatic Insufficiency
- ClinicalTrials.gov ID
- NCT00981214
- Acronym / sponsor ID
- EUR-1009-M
- Conditions
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- Keywords
- Cystic fibrosis, Exocrine Pancreatic Insufficiency, Zenpep-1009
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Forest LaboratoriesIndustry (pharma, biotech, devices)
- Study start
- May 2006
- Primary completion
- Sep 2006 (actual)
- Study completion
- Sep 2006 (actual)
- First posted
- 22 Sep 2009 (estimated)
- Results
- Posted 4 Apr 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label study to evaluate the efficacy and safety of Aptalis' (formerly Eurand) pancreatic enzyme product (PEP) microtabs in pediatric participants under age 7 with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI).
Detailed description
The study sample will consist of evaluable participants, all of whom will be children younger than 7 years of age. Participants will receive EUR-1008 (APT-1008) Microtabs formulation. The study design involves a 4-day screening period, a 7-day dose stabilization period, and a 7-day treatment period (excluding an end-of-study evaluation).
The optimal dose of EUR-1008 (APT-1008) Microtabs, determined during the dose stabilization period, will be used during the treatment period. Participants are instructed to consume a predefined diet.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| EUR-1008 (APT-1008) | Experimental | Drug: EUR-1008 (APT-1008) |
Who may qualify
- Ages
- Up to 7 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Participants less than 7 years of age
- Participants who have pancreatic insufficiency documented by a fecal elastase level less than 100 micrograms per gram (mcg/g), or if not documented, the fecal elastase test must be done at the screening visit
- Participants who have a need of de novo treatment with pancreatic enzymes or be able to be switched from an existing treatment
- Participants who have a body mass index greater than the twenty fifth percentile for children 2 years and older
- Participants with a weight for height index greater than the twenty fifth percentile for children less than 2 years of age
Key exclusion criteria (17 listed)
- Participants with fibrosing colonopathy
- Participants allergic to pork or other porcine PEPs
- Participants with any respiratory condition that in the investigator's opinion would result in an intervention requiring hospitalization or intensive pulmonary treatment during the trial
- 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 (such as azithromycin 250 or 500 milligram [mg] up to 3 times per week) is allowed. Moreover, chronic treatment (that is, daily for at least 1 month) with an inhalatory antibiotic (for example, colistin, tobramycin, or ceftazidime) is allowed
- Participants who have hepatic insufficiency as defined by a history or presence of ascites, or a serum albumin level of less than 3.0 milligram per deciliter (mg/dL), or coagulopathy with an international normalized ratio that is greater than 1.7
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EUR-1008 (APT-1008)Also: ZENPEP®, PancrelipaseEUR-1008 (APT-1008) Microtabs contained in a capsule will be administered orally from Day 5 to Day 11 at an enzyme dose based on investigator's discretion, in dose stabilization period or the content of the capsule will be allowed to sprinkle on food, where necessary, followed by stabilized dose from Day 12 to Day 18 in treatment period, up to a maximum total dose of 10,000 lipase units per kilogram body weight per day (unit/kg/day). | Drug | EUR-1008 (APT-1008) |
Primary outcome
Percentage of Participants Who Were Responders After 1 Week of Treatment With Study Medication
Time frame: Day 11
Responders were defined as those participants without steatorrhea (defined as less than 30 percent (%) fecal fat content) and without signs and symptoms of malabsorption after 1 week of treatment with study medication.
Percentage of Participants Who Were Responders After 2 Weeks of Treatment With Study Medication
Time frame: Day 18 (end of treatment)
Responders were defined as those participants without steatorrhea (defined as less than 30% fecal fat content) and without signs and symptoms of malabsorption after 2 weeks of treatment with study medication.
9 secondary outcomes
Change From Baseline in Weight at Day 12, 19
Time frame: Baseline, Day 12, 19
Mean Daily Number of Stools
Time frame: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)
Percentage of Stool Categorized by Consistency
Time frame: Baseline, Day 5 up to Day 11 (dose stabilization period) and Day 12 up to Day 18 (treatment period)
Mean Number of Abdominal Symptoms: Bloating
Time frame: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)
Mean Number of Abdominal Symptoms: Flatulence
Time frame: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)
Mean Number of Pain Symptoms
Time frame: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)
Physician's and Parent's or Legal Guardians Assessment of Improvement in Clinical Symptoms
Time frame: Day 19 (end of study)
Percentage of Blood in Stool
Time frame: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)
Percentage of Stool With Visible Oil or Grease
Time frame: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Aptalis Medical Information study director
Forest Laboratories
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing EUR-1008 (APT-1008) in people with Cystic Fibrosis and Exocrine Pancreatic Insufficiency, run by Forest Laboratories at 14 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Apr 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants Who Were Responders After 1 Week of Treatment With Study Medication (time frame: Day 11). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Mar 2017.