Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Conditions
Keywords
Exocrine Pancreatic Insufficiency, Infants with CF
Brief 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). 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.
Interventions
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.
Sponsors
Forest Laboratories
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Months to 12 Months
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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 * 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment Difference for Acceptability of Treatment | 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]) | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Stools With Signs of Blood and Visible Oil or Grease | Up to Day 10 in first and second treatment periods, end of study (Day 21) | 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. |
| Number of Abdominal Symptoms: Bloating | Up to Day 10 in first and second treatment periods, end of study (Day 21) | 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. |
| Number of Abdominal Symptoms: Flatulence | Up to Day 10 in first and second treatment periods, end of study (Day 21) | 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. |
| Daily Number of Stools | Up to Day 10 in first and second treatment periods, end of study (Day 21) | 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. |
| Number of Participants With Abnormal Clinical Laboratory and Vital Signs Findings | Baseline up to end of study (Day 21) | — |
| Number of Participants With Abnormal Findings With Respect to Oral Mucosa | Baseline up to end of study (Day 21) | 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. |
| Number of Abdominal Pain Symptoms | Up to Day 10 in first and second treatment periods, end of study (Day 21) | 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. |
| Number of Stools Categorized as Per Consistency | Up to Day 10 in first and second treatment periods, end of study (Day 21) | 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. |
Countries
United States
Participant flow
Pre-assignment details
All enrolled participants were administered Zenpep® 5,000 (pancrelipase) from open capsule mixed with a small amount of apple sauce in the screening period for 10 days.
Participants by arm
| Arm | Count |
|---|---|
| Entire Study Population Includes all enrolled participants who received EUR-1008 (APT-1008) (Zenpep® \[pancrelipase\] delayed release capsule) mixed with a small amount of apple juice using a syringe nurser first and EUR-1008 (APT-1008) (Zenpep® \[pancrelipase\] delayed release capsule) mixed with a small amount of apple sauce using a spoon first. | 15 |
| Total | 15 |
Baseline characteristics
| Characteristic | Entire Study Population |
|---|---|
| Age, Continuous | 5.7 months STANDARD_DEVIATION 3.15 |
| Average Daily Number of Stools | 2.73 average number of stools per day STANDARD_DEVIATION 1.393 |
| Number of Abdominal Pain Symptoms Mild Abdominal Pain | 0.23 average number of pain symptoms per day STANDARD_DEVIATION 0.409 |
| Number of Abdominal Pain Symptoms Moderate Abdominal Pain | 0.09 average number of pain symptoms per day STANDARD_DEVIATION 0.185 |
| Number of Abdominal Pain Symptoms Severe Abdominal Pain | 0.02 average number of pain symptoms per day STANDARD_DEVIATION 0.066 |
| Number of Abdominal Symptoms: Bloating Mild Bloating | 0.12 average number of bloating per day STANDARD_DEVIATION 0.281 |
| Number of Abdominal Symptoms: Bloating Moderate Bloating | 0.04 average number of bloating per day STANDARD_DEVIATION 0.101 |
| Number of Abdominal Symptoms: Bloating Severe Bloating | 0 average number of bloating per day STANDARD_DEVIATION 0 |
| Number of Abdominal Symptoms: Flatulence Mild Flatulence | 1.00 average number of flatulence per day STANDARD_DEVIATION 1.208 |
| Number of Abdominal Symptoms: Flatulence Moderate Flatulence | 0.51 average number of flatulence per day STANDARD_DEVIATION 0.728 |
| Number of Abdominal Symptoms: Flatulence Severe Flatulence | 0.03 average number of flatulence per day STANDARD_DEVIATION 0.099 |
| Number of Stools Categorized as Per Consistency Diarrhea | 0.48 average number of stools per day STANDARD_DEVIATION 0.801 |
| Number of Stools Categorized as Per Consistency Hard | 0.11 average number of stools per day STANDARD_DEVIATION 0.255 |
| Number of Stools Categorized as Per Consistency Normal | 1.18 average number of stools per day STANDARD_DEVIATION 1.129 |
| Number of Stools Categorized as Per Consistency Soft | 0.91 average number of stools per day STANDARD_DEVIATION 0.777 |
| Number of Stools With Signs of Blood and Visible Oil or Grease With blood | 0 average number of stools per day STANDARD_DEVIATION 0 |
| Number of Stools With Signs of Blood and Visible Oil or Grease With visible oil or grease | 0.11 average number of stools per day STANDARD_DEVIATION 0.147 |
| Sex: Female, Male Female | 6 Participants |
| Sex: Female, Male Male | 9 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 11 / 15 | 10 / 12 | 14 / 15 |
| serious Total, serious adverse events | 0 / 15 | 0 / 12 | 0 / 15 |
Outcome results
Primary
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
Population: Safety analysis population included all participants who received at least 1 dose of study medication.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| EUR-1008 (APT-1008) in Apple Juice | Question 6 (Previous Pancreatic Enzyme Product [PEP]) | Zenpep® | 4 participants |
| EUR-1008 (APT-1008) in Apple Juice | Question 6 (Previous Pancreatic Enzyme Product [PEP]) | Creon® | 9 participants |
| EUR-1008 (APT-1008) in Apple Juice | Question 6 (Previous Pancreatic Enzyme Product [PEP]) | Ultrase® | 1 participants |
| EUR-1008 (APT-1008) in Apple Juice | Question 6 (Previous Pancreatic Enzyme Product [PEP]) | Pancrecarb® | 1 participants |
Primary
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)
Population: Intention-to-treat (ITT) population included all participants who received at least 1 dose of study medication, had data (partial or complete) on acceptability questionnaire and clinical signs and symptoms of exocrine pancreatic insufficiency (EPI). Here, 'N' (number of participants analyzed) =participants who were evaluable for this measure.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| EUR-1008 (APT-1008) in Apple Juice | Treatment Difference for Acceptability of Treatment | Overall Satisfaction | 1.0 units on a scale |
| EUR-1008 (APT-1008) in Apple Juice | Treatment Difference for Acceptability of Treatment | Overall Infant Acceptance | 3.5 units on a scale |
| EUR-1008 (APT-1008) in Apple Juice | Treatment Difference for Acceptability of Treatment | Comparative Ease of Administration | 2.0 units on a scale |
| EUR-1008 (APT-1008) in Apple Juice | Treatment Difference for Acceptability of Treatment | Time of Administration | 2.5 units on a scale |
| EUR-1008 (APT-1008) in Apple Juice | Treatment Difference for Acceptability of Treatment | Comparative Infant Acceptance | 2.0 units on a scale |
| EUR-1008 (APT-1008) in Apple Juice | Treatment Difference for Acceptability of Treatment | Dispensing Instructions | 3.0 units on a scale |
| EUR-1008 (APT-1008) in Apple Juice | Treatment Difference for Acceptability of Treatment | Comparative Overall Satisfaction | 1.0 units on a scale |
| EUR-1008 (APT-1008) in Apple Juice | Treatment Difference for Acceptability of Treatment | Ease of Administration | 1.5 units on a scale |
| EUR-1008 (APT-1008) in Apple Sauce | Treatment Difference for Acceptability of Treatment | Comparative Overall Satisfaction | 3.0 units on a scale |
| EUR-1008 (APT-1008) in Apple Sauce | Treatment Difference for Acceptability of Treatment | Time of Administration | 1.0 units on a scale |
| EUR-1008 (APT-1008) in Apple Sauce | Treatment Difference for Acceptability of Treatment | Overall Infant Acceptance | 1.5 units on a scale |
| EUR-1008 (APT-1008) in Apple Sauce | Treatment Difference for Acceptability of Treatment | Dispensing Instructions | 4.5 units on a scale |
| EUR-1008 (APT-1008) in Apple Sauce | Treatment Difference for Acceptability of Treatment | Overall Satisfaction | 3.0 units on a scale |
| EUR-1008 (APT-1008) in Apple Sauce | Treatment Difference for Acceptability of Treatment | Comparative Ease of Administration | 3.0 units on a scale |
| EUR-1008 (APT-1008) in Apple Sauce | Treatment Difference for Acceptability of Treatment | Comparative Infant Acceptance | 3.0 units on a scale |
| EUR-1008 (APT-1008) in Apple Sauce | Treatment Difference for Acceptability of Treatment | Ease of Administration | 3.5 units on a scale |
Secondary
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)
Population: ITT population included all participants who received at least 1 dose of study medication, had data (partial or complete) on acceptability questionnaire on and clinical signs and symptoms of EPI. Here, 'N' (number of participants analyzed) =participants who were evaluable for this measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| EUR-1008 (APT-1008) in Apple Juice | Daily Number of Stools | 2.76 average number of stools per day | Standard Deviation 0.771 |
| EUR-1008 (APT-1008) in Apple Sauce | Daily Number of Stools | 2.44 average number of stools per day | Standard Deviation 0.766 |
Secondary
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)
Population: ITT population included all participants who received at least 1 dose of study medication, had data (partial or complete) on acceptability questionnaire and clinical signs and symptoms of EPI. Here, 'N' (number of participants analyzed) =participants who were evaluable for this measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| EUR-1008 (APT-1008) in Apple Juice | Number of Abdominal Pain Symptoms | Mild Abdominal Pain | 0.10 average number of pain symptoms per day | Standard Deviation 0.259 |
| EUR-1008 (APT-1008) in Apple Juice | Number of Abdominal Pain Symptoms | Moderate Abdominal Pain | 0.09 average number of pain symptoms per day | Standard Deviation 0.238 |
| EUR-1008 (APT-1008) in Apple Juice | Number of Abdominal Pain Symptoms | Severe Abdominal Pain | 0 average number of pain symptoms per day | Standard Deviation 0 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Abdominal Pain Symptoms | Mild Abdominal Pain | 0.13 average number of pain symptoms per day | Standard Deviation 0.343 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Abdominal Pain Symptoms | Moderate Abdominal Pain | 0.07 average number of pain symptoms per day | Standard Deviation 0.231 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Abdominal Pain Symptoms | Severe Abdominal Pain | 0 average number of pain symptoms per day | Standard Deviation 0 |
Secondary
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)
Population: ITT population included all participants who received at least 1 dose of study medication, had data (partial or complete) on acceptability questionnaire and clinical signs and symptoms of EPI. Here, 'N' (number of participants analyzed) =participants who were evaluable for this measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| EUR-1008 (APT-1008) in Apple Juice | Number of Abdominal Symptoms: Bloating | Mild Bloating | 0.22 average number of bloating per day | Standard Deviation 0.632 |
| EUR-1008 (APT-1008) in Apple Juice | Number of Abdominal Symptoms: Bloating | Moderate Bloating | 0 average number of bloating per day | Standard Deviation 0 |
| EUR-1008 (APT-1008) in Apple Juice | Number of Abdominal Symptoms: Bloating | Severe Bloating | 0 average number of bloating per day | Standard Deviation 0 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Abdominal Symptoms: Bloating | Mild Bloating | 0.26 average number of bloating per day | Standard Deviation 0.857 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Abdominal Symptoms: Bloating | Moderate Bloating | 0 average number of bloating per day | Standard Deviation 0 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Abdominal Symptoms: Bloating | Severe Bloating | 0 average number of bloating per day | Standard Deviation 0 |
Secondary
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)
Population: ITT population included all participants who received at least 1 dose of study medication, had data (partial or complete) on acceptability questionnaire and clinical signs and symptoms of EPI. Here, 'N' (number of participants analyzed) =participants who were evaluable for this measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| EUR-1008 (APT-1008) in Apple Juice | Number of Abdominal Symptoms: Flatulence | Severe Flatulence | 0.04 average number of flatulence per day | Standard Deviation 0.144 |
| EUR-1008 (APT-1008) in Apple Juice | Number of Abdominal Symptoms: Flatulence | Mild Flatulence | 1.0 average number of flatulence per day | Standard Deviation 1.583 |
| EUR-1008 (APT-1008) in Apple Juice | Number of Abdominal Symptoms: Flatulence | Moderate Flatulence | 0.31 average number of flatulence per day | Standard Deviation 0.559 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Abdominal Symptoms: Flatulence | Moderate Flatulence | 0.32 average number of flatulence per day | Standard Deviation 0.67 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Abdominal Symptoms: Flatulence | Severe Flatulence | 0 average number of flatulence per day | Standard Deviation 0 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Abdominal Symptoms: Flatulence | Mild Flatulence | 0.62 average number of flatulence per day | Standard Deviation 0.869 |
Secondary
Number of Participants With Abnormal Clinical Laboratory and Vital Signs Findings
Time frame: Baseline up to end of study (Day 21)
Population: Data was reported in individual participant listings but not statistically summarized for analysis as planned.
Secondary
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)
Population: Safety population included all participants who received at least 1 dose of study medication.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| EUR-1008 (APT-1008) in Apple Juice | Number of Participants With Abnormal Findings With Respect to Oral Mucosa | 0 participants |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Participants With Abnormal Findings With Respect to Oral Mucosa | 0 participants |
Secondary
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)
Population: ITT population included all participants who received at least 1 dose of study medication, had data (partial or complete) on acceptability questionnaire and clinical signs and symptoms of EPI. Here, 'N' (number of participants analyzed) =participants who were evaluable for this measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| EUR-1008 (APT-1008) in Apple Juice | Number of Stools Categorized as Per Consistency | Hard | 0.04 average number of stools per day | Standard Deviation 0.089 |
| EUR-1008 (APT-1008) in Apple Juice | Number of Stools Categorized as Per Consistency | Normal | 1.77 average number of stools per day | Standard Deviation 0.864 |
| EUR-1008 (APT-1008) in Apple Juice | Number of Stools Categorized as Per Consistency | Soft | 0.62 average number of stools per day | Standard Deviation 0.71 |
| EUR-1008 (APT-1008) in Apple Juice | Number of Stools Categorized as Per Consistency | Diarrhea | 0.24 average number of stools per day | Standard Deviation 0.527 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Stools Categorized as Per Consistency | Diarrhea | 0.07 average number of stools per day | Standard Deviation 0.135 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Stools Categorized as Per Consistency | Hard | 0.14 average number of stools per day | Standard Deviation 0.184 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Stools Categorized as Per Consistency | Soft | 0.83 average number of stools per day | Standard Deviation 0.813 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Stools Categorized as Per Consistency | Normal | 1.37 average number of stools per day | Standard Deviation 0.77 |
Secondary
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)
Population: ITT population included all participants who received at least 1 dose of study medication, had data (partial or complete) on acceptability questionnaire and clinical signs and symptoms of EPI. Here, 'N' (number of participants analyzed) =participants who were evaluable for this measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| EUR-1008 (APT-1008) in Apple Juice | Number of Stools With Signs of Blood and Visible Oil or Grease | With blood | 0 average number of stools per day | Standard Deviation 0 |
| EUR-1008 (APT-1008) in Apple Juice | Number of Stools With Signs of Blood and Visible Oil or Grease | With visible oil or grease | 0.09 average number of stools per day | Standard Deviation 0.191 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Stools With Signs of Blood and Visible Oil or Grease | With blood | 0.01 average number of stools per day | Standard Deviation 0.047 |
| EUR-1008 (APT-1008) in Apple Sauce | Number of Stools With Signs of Blood and Visible Oil or Grease | With visible oil or grease | 0.17 average number of stools per day | Standard Deviation 0.368 |