A Phase 4 interventional study of EUR-1008 (APT-1008) and EUR-1008 (APT-1008) in Cystic Fibrosis and Exocrine Pancreatic Insufficiency, sponsored by Forest Laboratories. Completed at 6 sites in United States. Open to participants aged 1 Month to 12 Months. Per ClinicalTrials.gov, last updated 2014-04-10.
Sponsored by Forest Laboratories · Phase 4, Interventional, and Treatment
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).
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.
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's enrollment of 15 is below the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Forest Laboratories is the lead sponsor of 165 studies on the registry; none are open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 6 (100%) have results posted.
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Exclusion Criteria:
Drug: EUR-1008 (APT-1008)
Drug: EUR-1008 (APT-1008)
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.
Also known as: 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.
Also known as: Zenpep® (pancrelipase) delayed release capsules
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).
Time frame: 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.
Time frame: Baseline
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.
Time frame: 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.
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
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
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
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.
Time frame: Baseline up to end of study (Day 21)
| Milestone | EUR-1008 (APT-1008) in Apple Juice First, Then in Apple Sauce | EUR-1008 (APT-1008) in Apple Sauce First, Then in Apple Juice |
|---|---|---|
| Started | 8 | 7 |
| Completed | 8 | 7 |
| Not completed | 0 | 0 |
| Milestone | EUR-1008 (APT-1008) in Apple Juice First, Then in Apple Sauce | EUR-1008 (APT-1008) in Apple Sauce First, Then in Apple Juice |
|---|---|---|
| Started | 8 | 7 |
| Completed | 8 | 7 |
| Not completed | 0 | 0 |
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.
| average number of stools per day | EUR-1008 (APT-1008) in Apple Juice | EUR-1008 (APT-1008) in Apple Sauce |
|---|---|---|
| Daily Number of Stools | 2.76 ± 0.771 | 2.44 ± 0.766 |
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.
| average number of stools per day | EUR-1008 (APT-1008) in Apple Juice | EUR-1008 (APT-1008) in Apple Sauce |
|---|---|---|
| Hard | 0.04 ± 0.089 | 0.14 ± 0.184 |
| Normal | 1.77 ± 0.864 | 1.37 ± 0.770 |
| Soft | 0.62 ± 0.710 | 0.83 ± 0.813 |
| Diarrhea | 0.24 ± 0.527 | 0.07 ± 0.135 |
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.
| average number of stools per day | EUR-1008 (APT-1008) in Apple Juice | EUR-1008 (APT-1008) in Apple Sauce |
|---|---|---|
| With blood | 0 ± 0 | 0.01 ± 0.047 |
| With visible oil or grease | 0.09 ± 0.191 | 0.17 ± 0.368 |
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.
| average number of bloating per day | EUR-1008 (APT-1008) in Apple Juice | EUR-1008 (APT-1008) in Apple Sauce |
|---|---|---|
| Mild Bloating | 0.22 ± 0.632 | 0.26 ± 0.857 |
| Moderate Bloating | 0 ± 0 | 0 ± 0 |
| Severe Bloating | 0 ± 0 | 0 ± 0 |
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.
| average number of flatulence per day | EUR-1008 (APT-1008) in Apple Juice | EUR-1008 (APT-1008) in Apple Sauce |
|---|---|---|
| Mild Flatulence | 1.0 ± 1.583 | 0.62 ± 0.869 |
| Moderate Flatulence | 0.31 ± 0.559 | 0.32 ± 0.670 |
| Severe Flatulence | 0.04 ± 0.144 | 0 ± 0 |
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.
| average number of pain symptoms per day | EUR-1008 (APT-1008) in Apple Juice | EUR-1008 (APT-1008) in Apple Sauce |
|---|---|---|
| Mild Abdominal Pain | 0.10 ± 0.259 | 0.13 ± 0.343 |
| Moderate Abdominal Pain | 0.09 ± 0.238 | 0.07 ± 0.231 |
| Severe Abdominal Pain | 0 ± 0 | 0 ± 0 |
No measurements were reported for this outcome.
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.
| participants | EUR-1008 (APT-1008) in Apple Juice | EUR-1008 (APT-1008) in Apple Sauce |
|---|---|---|
| Number of Participants With Abnormal Findings With Respect to Oral Mucosa | 0 | 0 |
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).
| units on a scale | EUR-1008 (APT-1008) in Apple Juice | EUR-1008 (APT-1008) in Apple Sauce |
|---|---|---|
| Ease of Administration | 1.5 (1 to 3) | 3.5 (2 to 5) |
| Time of Administration | 2.5 (1 to 4) | 1.0 (1 to 3) |
| Overall Infant Acceptance | 3.5 (2 to 5) | 1.5 (1 to 4) |
| Dispensing Instructions | 3.0 (1 to 5) | 4.5 (3 to 5) |
| Overall Satisfaction | 1.0 (1 to 3) | 3.0 (2 to 4) |
| Comparative Ease of Administration | 2.0 (1 to 5) | 3.0 (2 to 5) |
| Comparative Infant Acceptance | 2.0 (1 to 5) | 3.0 (3 to 5) |
| Comparative Overall Satisfaction | 1.0 (1 to 4) | 3.0 (3 to 5) |
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.
| participants | Entire Study Population |
|---|---|
| Zenpep® | 4 |
| Creon® | 9 |
| Ultrase® | 1 |
| Pancrecarb® | 1 |
Collected over Baseline up to end of study (Day 21). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Zenpep® | — | 0/15 (0%) | 11/15 (73.3%) |
| EUR-1008 (APT-1008) in Apple Juice | — | 0/12 (0%) | 10/12 (83.3%) |
| EUR-1008 (APT-1008) in Apple Sauce | — | 0/15 (0%) | 14/15 (93.3%) |
| Event | Zenpep® | EUR-1008 (APT-1008) in Apple Juice | EUR-1008 (APT-1008) in Apple Sauce |
|---|---|---|---|
| FlatulenceGastrointestinal disorders | 6/15 | 1/12 | 5/15 |
| CoughRespiratory, thoracic and mediastinal disorders | 1/15 | 2/12 | 6/15 |
| Abdominal painGastrointestinal disorders | 4/15 | 3/12 | 4/15 |
| SteatorrhoeaGastrointestinal disorders | 3/15 | 2/12 | 4/15 |
| Abdominal distensionGastrointestinal disorders | 2/15 | 3/12 | 2/15 |
| DiarrhoeaGastrointestinal disorders | 3/15 | 2/12 | 1/15 |
| PyrexiaGeneral disorders | 1/15 | 2/12 | 3/15 |
| TeethingGastrointestinal disorders | 2/15 | 1/12 | 1/15 |
| ConstipationGastrointestinal disorders | 0/15 | 0/12 | 2/15 |
| Faeces hardGastrointestinal disorders | 0/15 | 0/12 | 2/15 |
Safety analysis population included all participants who received at least 1 dose of study medication.
| Age, Continuous(months) | Entire Study Population |
|---|---|
| Mean | 5.7 ± 3.15 |
| Sex: Female, Male(Participants) | Entire Study Population |
|---|---|
| Female | 6 |
| Male | 9 |
| Average Daily Number of Stools(average number of stools per day) | Entire Study Population |
|---|---|
| Mean | 2.73 ± 1.393 |
| Number of Stools Categorized as Per Consistency(average number of stools per day) | Entire Study Population |
|---|---|
| Hard | 0.11 ± 0.255 |
| Normal | 1.18 ± 1.129 |
| Soft | 0.91 ± 0.777 |
| Diarrhea | 0.48 ± 0.801 |
| Number of Stools With Signs of Blood and Visible Oil or Grease(average number of stools per day) | Entire Study Population |
|---|---|
| With blood | 0 ± 0 |
| With visible oil or grease | 0.11 ± 0.147 |
| Number of Abdominal Symptoms: Bloating(average number of bloating per day) | Entire Study Population |
|---|---|
| Mild Bloating | 0.12 ± 0.281 |
| Moderate Bloating | 0.04 ± 0.101 |
| Severe Bloating | 0 ± 0 |
| Number of Abdominal Symptoms: Flatulence(average number of flatulence per day) | Entire Study Population |
|---|---|
| Mild Flatulence | 1.00 ± 1.208 |
| Moderate Flatulence | 0.51 ± 0.728 |
| Severe Flatulence | 0.03 ± 0.099 |
| Number of Abdominal Pain Symptoms(average number of pain symptoms per day) | Entire Study Population |
|---|---|
| Mild Abdominal Pain | 0.23 ± 0.409 |
| Moderate Abdominal Pain | 0.09 ± 0.185 |
| Severe Abdominal Pain | 0.02 ± 0.066 |
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