Cystic FibrosisPhase 4NCT01100606
A Study to Evaluate the Mode of Administration and Safety of EUR-1008 (APT-1008) in Infants 1 to 12 Months of Age
A Phase 4 study testing EUR-1008 (APT-1008) in people with Cystic Fibrosis and Exocrine Pancreatic Insufficiency, run by Forest Laboratories at 6 sites in United States.
Trial summary
A study to determine the safety, effectiveness, and acceptability of 2 methods of administration of EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) 3,000 lipase units capsule, a pancreatic enzyme product (PEP), in infants with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). Read more about this study
- Who is it for?
- Ages 1 month to 12 months
- 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, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 6 sitesUnited States
- Study dates
- Jun 2010to Dec 2010
- Phase
- Phase 4
- 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 1 month to 12 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participants with diagnosis of CF based on the following criteria: one clinical feature consistent with CF, and either a genotype with 2 identifiable mutations known to cause CF or a sweat chloride concentration that is greater than 60 milliequivalent per liter by quantitative pilocarpine iontophoresis
- Participants who have the need for a PEP defined as monoclonal fecal elastase less than 200 microgram per gram (mcg/g) stool
- Caregiver must be willing to switch participant from their previous PEP (if any) to Zenpep®
- Participants who have a height to weight ratio target at greater than tenth percentile
- Participants who are clinically stable with no evidence of concomitant illness or acute upper or lower respiratory tract infection during the 7-day interval prior to screening and preceding accession into this clinical study
Who cannot join
- Participants who are less than 1 month old or are greater than 12 months old
- Participants with history of meconium ileus or small bowel atresia in the newborn period that required surgery
- Participants who are allergic to pork or other porcine PEPs
- Participants with any respiratory condition or other serious comorbidity (for example patent ductus arteriosus [PDA], or necrotizing enterocolitis [NEC]) that in the investigator's opinion would result in an intervention requiring hospitalization or intensive pulmonary or other treatment during the trial
- Participants with other comorbidities independent of CF that, in the investigator's opinion, would result in an inability to participate in the study or excess risk to the participant that is above the standard of care
- Participants with acute respiratory infection in the previous 14 days requiring antibiotics
Show all 10Show fewer
- Participants who required change in antacid dose in the 7 days before screening
- Participants with administration of oral, intramuscular (IM), intravenous (IV) glucocorticoids in the 4 weeks prior to screening
- Participants with any condition that would, in the investigator's opinion, limit the participant's ability to complete the study
- Participants currently participating in or has participated in an investigational study, with the exception of observational studies, within 30 days of the screening visit
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Akron Children's Hospital
Akron, Ohio, 44308
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
Cystic Fibrosis Care Center
Houston, Texas, 77030
Nemours Children's Clinic
Jacksonville, Florida, 32250
Children's Lung Specialists Ltd.
Las Vegas, Nevada, 89107
About this study
Official title: A Multicenter, Randomized, Open-Label, Crossover Study to Evaluate the Mode of Administration and Safety of EUR-1008 in Infants 1 to 12 Months of Age With Exocrine Pancreatic Insufficiency (EPI) Associated With Cystic Fibrosis (CF)
A study to determine the safety, effectiveness, and acceptability of 2 methods of administration of EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) 3,000 lipase units capsule, a pancreatic enzyme product (PEP), in infants with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). This study is sponsored by Aptalis Pharma (formerly Eurand).
Read the detailed description
This is a multicenter, randomized, open-label, crossover study in pediatric participants with EPI due to CF. The study will be carried out in infants between 1 and 12 months of age.
The study comprises of a screening period (up to 10 days) followed by 2 treatment periods (10 days each). During the screening period, all participants will be administered Zenpep® 5,000 (pancrelipase) mixed with a small amount of apple sauce. Once determined eligible for participation, participants will be randomized into 1 of 2 treatment sequences. Each sequence corresponds to taking one treatment in the first period and the other treatment in the second period, and were administered EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) 3,000 lipase units capsule either mixed with apple juice using a syringe nurser or apple sauce using a spoon.
- Conditions
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- Being tested
- EUR-1008 (APT-1008)
- Keywords
- Exocrine Pancreatic Insufficiency, Infants with CF
- 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) delayed release capsules
EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) from open capsule, mixed with a small amount of apple juice, will be given orally daily using a syringe nurser at dose increments of 3,000 lipase units, for 10 days in either first treatment period or second treatment period. Total dose will not exceed 10,000 lipase units per kilogram (kg) of body weight per day.
EUR-1008 (APT-1008)Drug
Also called Zenpep® (pancrelipase) delayed release capsules
EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) from open capsule, mixed with a small amount of apple sauce, will be given orally daily using a spoon at dose increments of 3,000 lipase units, for 10 days in either first treatment period or second treatment period. Total dose will not exceed 10,000 lipase units per kg of body weight per day.
| Study group | Role | What they receive |
|---|---|---|
| EUR-1008 (APT-1008) in Apple Juice | Experimental | Not described Receives: Drug: EUR-1008 (APT-1008) |
| EUR-1008 (APT-1008) in Apple Sauce | 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.
Treatment Difference for Acceptability of Treatment
Acceptability questionnaire consists of 9 question (Q) to assess ease, time, overall satisfaction of study drug. Rated on 5-point scale for Q1-Q5 and Q7-Q9; Q6 was not rated and asked for name of previous PEP administered. Q1=overall ease of administration (1=not at all easy,2=somewhat,3=easy,4=very,5=extremely); Q2=time of administration (1=very short[<2 min],2=short[2-5 min],3=moderate[5-15 min],4=long[15-25 min],5=very long[>25 min]);Q3=overall infant acceptance(1=very easily,2=easily,3=same,4=with difficulty,5=with great difficulty);Q4=clear/complete instructions(1=not clear,2=somewhat,3=clear,4=very,5=extremely);Q5=overall satisfaction with dosing method (1=not satisfied,2=somewhat,3=satisfied,4=very,5=extremely);Q7=comparative ease of administration (1=much worse,2=worse,3=same,4=better,5=much better);Q8=comparative infant acceptance (1=much more difficult,2=more,3=same,4=easier,5=much easier);Q9=comparative overall satisfaction (1=much less,2=less,3=same,4=more,5=much more).
Measured: Baseline up to end of study (Day 21)
Question 6 (Previous Pancreatic Enzyme Product [PEP])
Acceptability questionnaire consists of 9 questions (Q) to assess the ease, time, overall satisfaction of study drug. Q6 included "name of previous PEP administered". Q6 was reported as number of participants who used any PEP prior to screening.
Measured: Baseline
Secondary outcomes (8)
Daily Number of Stools
Average daily number of stools of each participant was calculated from frequency of stools by the participant per day. Average daily number of stools during the first treatment period, second treatment period and end of study for total participants was summarized.
Measured: Up to Day 10 in first and second treatment periods, end of study (Day 21)
Number of Stools Categorized as Per Consistency
Stool consistency was categorized as hard, formed/normal, soft and diarrhea. Average number of stools categorized as per consistency of each participant was calculated from number of stools of specific consistency by the participant per day. Average number of stools categorized as per consistency during the first treatment period, second treatment period and end of study for total participants was summarized.
Measured: Up to Day 10 in first and second treatment periods, end of study (Day 21)
Number of Stools With Signs of Blood and Visible Oil or Grease
Average number of stools with signs of blood and visible oil or grease of each participant was calculated from number of stools with signs of blood and visible oil or grease by the participant per day. Average number of stools with signs of blood and visible oil or grease during the first treatment period, second treatment period and end of study for total participants was summarized.
Measured: Up to Day 10 in first and second treatment periods, end of study (Day 21)
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 0=none, 1=mild (no impairment of daily activities), 2=moderate (slight impairment of daily activities), and 3=severe (unable to perform daily activities). Average number of symptoms of specific severity for each participant was calculated from frequency of symptoms by the participant per day. Average number of symptoms during the first treatment period, second treatment period and end of study for total participants was summarized.
Measured: Up to Day 10 in first and second treatment periods, end of study (Day 21)
Number of Abdominal Symptoms: Flatulence
Flatulence is presence of excessive gas in the digestive tract. Symptoms of flatulence were classified by severity as 0=none, 1=mild (no impairment of daily activities), 2=moderate (slight impairment of daily activities), and 3=severe (unable to perform daily activities). Average number of symptoms of specific severity for each participant was calculated from frequency of symptoms by the participant per day. Average number of symptoms during the first treatment period, second treatment period and end of study for total participants was summarized.
Measured: Up to Day 10 in first and second treatment periods, end of study (Day 21)
Number of Abdominal Pain Symptoms
Symptoms of pain was classified by severity as 0=none, 1=mild (no impairment of daily activities), 2=moderate (slight impairment of daily activities), and 3=severe (unable to perform daily activities). Average number of symptoms of specific severity for each participant was calculated from frequency of symptoms by the participant per day. Average number of symptoms during the first treatment period, second treatment period and end of study for total participants was summarized.
Measured: Up to Day 10 in first and second treatment periods, end of study (Day 21)
Number of Participants With Abnormal Clinical Laboratory and Vital Signs Findings
Measured: Baseline up to end of study (Day 21)
Number of Participants With Abnormal Findings With Respect to Oral Mucosa
Safety assessed by the presence of lesions observed during a physical examination at each visit. Severity of lesions measured by investigator's assessment using the following scale: mild = asymptomatic or mild symptoms and treatment not indicated; moderate = moderate pain but not interfering with oral intake, modified diet indicated; severe = severe pain, interfering with oral intake and life threatening or fatal.
Measured: Baseline up to end of study (Day 21)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2010 (estimated)
- Study startJun 2010
- Primary completionDec 2010
- Study completionDec 2010
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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 15 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
- NCT01100606
- Sponsor's protocol ID
- PR-011
- Status last verified
- Mar 2014
- First submitted
- 31 Mar 2010
- Last updated
- 10 Apr 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 10 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Open-Label, Crossover Study to Evaluate the Mode of Administration and Safety of EUR-1008 in Infants 1 to 12 Months of Age With Exocrine Pancreatic Insufficiency (EPI) Associated With Cystic Fibrosis (CF)
- ClinicalTrials.gov ID
- NCT01100606
- Acronym / sponsor ID
- PR-011
- Conditions
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- Keywords
- Exocrine Pancreatic Insufficiency, Infants with CF
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Forest LaboratoriesIndustry (pharma, biotech, devices)
- Study start
- Jun 2010
- Primary completion
- Dec 2010 (actual)
- Study completion
- Dec 2010 (actual)
- First posted
- 9 Apr 2010 (estimated)
- Results
- Posted 10 Apr 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A study to determine the safety, effectiveness, and acceptability of 2 methods of administration of EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) 3,000 lipase units capsule, a pancreatic enzyme product (PEP), in infants with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). This study is sponsored by Aptalis Pharma (formerly Eurand).
Detailed description
This is a multicenter, randomized, open-label, crossover study in pediatric participants with EPI due to CF. The study will be carried out in infants between 1 and 12 months of age.
The study comprises of a screening period (up to 10 days) followed by 2 treatment periods (10 days each). During the screening period, all participants will be administered Zenpep® 5,000 (pancrelipase) mixed with a small amount of apple sauce. Once determined eligible for participation, participants will be randomized into 1 of 2 treatment sequences. Each sequence corresponds to taking one treatment in the first period and the other treatment in the second period, and were administered EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) 3,000 lipase units capsule either mixed with apple juice using a syringe nurser or apple sauce using a spoon.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| EUR-1008 (APT-1008) in Apple Juice | Experimental | Drug: EUR-1008 (APT-1008) |
| EUR-1008 (APT-1008) in Apple Sauce | Experimental | Drug: EUR-1008 (APT-1008) |
Who may qualify
- Ages
- 1 month to 12 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Participants with diagnosis of CF based on the following criteria: one clinical feature consistent with CF, and either a genotype with 2 identifiable mutations known to cause CF or a sweat chloride concentration that is greater than 60 milliequivalent per liter by quantitative pilocarpine iontophoresis
- Participants who have the need for a PEP defined as monoclonal fecal elastase less than 200 microgram per gram (mcg/g) stool
- Caregiver must be willing to switch participant from their previous PEP (if any) to Zenpep®
- Participants who have a height to weight ratio target at greater than tenth percentile
- Participants who are clinically stable with no evidence of concomitant illness or acute upper or lower respiratory tract infection during the 7-day interval prior to screening and preceding accession into this clinical study
Key exclusion criteria (10 listed)
- Participants who are less than 1 month old or are greater than 12 months old
- Participants with history of meconium ileus or small bowel atresia in the newborn period that required surgery
- Participants who are allergic to pork or other porcine PEPs
- Participants with any respiratory condition or other serious comorbidity (for example patent ductus arteriosus [PDA], or necrotizing enterocolitis [NEC]) that in the investigator's opinion would result in an intervention requiring hospitalization or intensive pulmonary or other treatment during the trial
- Participants with other comorbidities independent of CF that, in the investigator's opinion, would result in an inability to participate in the study or excess risk to the participant that is above the standard of care
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® (pancrelipase) delayed release capsulesEUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) from open capsule, mixed with a small amount of apple juice, will be given orally daily using a syringe nurser at dose increments of 3,000 lipase units, for 10 days in either first treatment period or second treatment period. Total dose will not exceed 10,000 lipase units per kilogram (kg) of body weight per day. | Drug | EUR-1008 (APT-1008) in Apple Juice |
| EUR-1008 (APT-1008)Also: Zenpep® (pancrelipase) delayed release capsulesEUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) from open capsule, mixed with a small amount of apple sauce, will be given orally daily using a spoon at dose increments of 3,000 lipase units, for 10 days in either first treatment period or second treatment period. Total dose will not exceed 10,000 lipase units per kg of body weight per day. | Drug | EUR-1008 (APT-1008) in Apple Sauce |
Primary outcome
Treatment Difference for Acceptability of Treatment
Time frame: Baseline up to end of study (Day 21)
Acceptability questionnaire consists of 9 question (Q) to assess ease, time, overall satisfaction of study drug. Rated on 5-point scale for Q1-Q5 and Q7-Q9; Q6 was not rated and asked for name of previous PEP administered. Q1=overall ease of administration (1=not at all easy,2=somewhat,3=easy,4=very,5=extremely); Q2=time of administration (1=very short[<2 min],2=short[2-5 min],3=moderate[5-15 min],4=long[15-25 min],5=very long[>25 min]);Q3=overall infant acceptance(1=very easily,2=easily,3=same,4=with difficulty,5=with great difficulty);Q4=clear/complete instructions(1=not clear,2=somewhat,3=clear,4=very,5=extremely);Q5=overall satisfaction with dosing method (1=not satisfied,2=somewhat,3=satisfied,4=very,5=extremely);Q7=comparative ease of administration (1=much worse,2=worse,3=same,4=better,5=much better);Q8=comparative infant acceptance (1=much more difficult,2=more,3=same,4=easier,5=much easier);Q9=comparative overall satisfaction (1=much less,2=less,3=same,4=more,5=much more).
Question 6 (Previous Pancreatic Enzyme Product [PEP])
Time frame: Baseline
Acceptability questionnaire consists of 9 questions (Q) to assess the ease, time, overall satisfaction of study drug. Q6 included "name of previous PEP administered". Q6 was reported as number of participants who used any PEP prior to screening.
8 secondary outcomes
Daily Number of Stools
Time frame: Up to Day 10 in first and second treatment periods, end of study (Day 21)
Number of Stools Categorized as Per Consistency
Time frame: Up to Day 10 in first and second treatment periods, end of study (Day 21)
Number of Stools With Signs of Blood and Visible Oil or Grease
Time frame: Up to Day 10 in first and second treatment periods, end of study (Day 21)
Number of Abdominal Symptoms: Bloating
Time frame: Up to Day 10 in first and second treatment periods, end of study (Day 21)
Number of Abdominal Symptoms: Flatulence
Time frame: Up to Day 10 in first and second treatment periods, end of study (Day 21)
Number of Abdominal Pain Symptoms
Time frame: Up to Day 10 in first and second treatment periods, end of study (Day 21)
Number of Participants With Abnormal Clinical Laboratory and Vital Signs Findings
Time frame: Baseline up to end of study (Day 21)
Number of Participants With Abnormal Findings With Respect to Oral Mucosa
Time frame: Baseline up to end of study (Day 21)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Apr 2014.
- 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
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing EUR-1008 (APT-1008) in people with Cystic Fibrosis and Exocrine Pancreatic Insufficiency, run by Forest Laboratories at 6 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 5 inclusion and 10 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 10 Apr 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 10 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?
Treatment Difference for Acceptability of Treatment (time frame: Baseline up to end of study (Day 21)). 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 10 Apr 2014.