A Study to Evaluate the Mode of Administration and Safety of EUR-1008 (APT-1008) in Infants 1 to 12 Months of Age

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Aptalis Pharma
ClinicalTrials.gov Identifier:
NCT01100606
First received: March 31, 2010
Last updated: March 5, 2014
Last verified: March 2014
Results First Received: March 5, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Crossover Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Intervention: Drug: EUR-1008 (APT-1008)

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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.

Reporting Groups
  Description
EUR-1008 (APT-1008) in Apple Juice First, Then in Apple Sauce EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) from open capsule, mixed with a small amount of apple juice using a syringe nurser, orally daily at dose increments of 3,000 lipase units, for 10 days in first treatment period followed by EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) from open capsule, mixed with a small amount of apple sauce using a spoon, orally daily at dose increments of 3,000 lipase units, for 10 days in second treatment period. Total dose was not to exceed 10,000 lipase units/kilogram body weight/day (lipase units/kg/day).
EUR-1008 (APT-1008) in Apple Sauce First, Then in Apple Juice EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) from open capsule, mixed with a small amount of apple sauce using a spoon, orally daily at dose increments of 3,000 lipase units, for 10 days in first treatment period followed by EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) from open capsule, mixed with a small amount of apple juice using a syringe nurser, orally daily at dose increments of 3,000 lipase units, for 10 days in second treatment period. Total dose was not to exceed 10,000 lipase units/kg/day.

Participant Flow for 2 periods

Period 1:   First Treatment Period
    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 [1]   7  
NOT COMPLETED     0     0  
[1] Due to difficulty with syringe nurser,1 participant completed study with apple sauce,not apple juice

Period 2:   Second Treatment Period
    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 [1]   7 [2]
NOT COMPLETED     0     0  
[1] Due to intolerance to apple sauce & apple juice,1 participant completed study with banana-based food
[2] Due to difficulty with syringe nurser, 1 participant completed study using apple sauce



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Safety analysis population included all participants who received at least 1 dose of study medication.

Reporting Groups
  Description
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.

Baseline Measures
    Entire Study Population  
Number of Participants  
[units: participants]
  15  
Age  
[units: months]
Mean ± Standard Deviation
  5.7  ± 3.15  
Gender  
[units: participants]
 
Female     6  
Male     9  
Average Daily Number of Stools [1]
[units: average number of stools per day]
Mean ± Standard Deviation
  2.73  ± 1.393  
Number of Stools Categorized as Per Consistency [2]
[units: average number of stools per day]
Mean ± Standard Deviation
 
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 [3]
[units: average number of stools per day]
Mean ± Standard Deviation
 
With blood     0  ± 0  
With visible oil or grease     0.11  ± 0.147  
Number of Abdominal Symptoms: Bloating [4]
[units: average number of bloating per day]
Mean ± Standard Deviation
 
Mild Bloating     0.12  ± 0.281  
Moderate Bloating     0.04  ± 0.101  
Severe Bloating     0  ± 0  
Number of Abdominal Symptoms: Flatulence [5]
[units: average number of flatulence per day]
Mean ± Standard Deviation
 
Mild Flatulence     1.00  ± 1.208  
Moderate Flatulence     0.51  ± 0.728  
Severe Flatulence     0.03  ± 0.099  
Number of Abdominal Pain Symptoms [6]
[units: average number of pain symptoms per day]
Mean ± Standard Deviation
 
Mild Abdominal Pain     0.23  ± 0.409  
Moderate Abdominal Pain     0.09  ± 0.185  
Severe Abdominal Pain     0.02  ± 0.066  
[1] 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 screening period for total participants was summarized. Number of participants evaluable for this baseline characteristic was 12.
[2] 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 screening period for total participants was summarized. Number of participants evaluable for this baseline characteristic was 12.
[3] 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 stools with signs of blood and visible oil or grease during the screening period for total participants was summarized. Number of participants evaluable for this baseline characteristic was 12.
[4] 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 screening period for total participants was summarized. Number of participants evaluable for this baseline characteristic was 12.
[5] 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 screening period for total participants was summarized. Number of participants evaluable for this baseline characteristic was 12.
[6] 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 screening period for total participants was summarized. Number of participants evaluable for this baseline characteristic was 12.



  Outcome Measures
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1.  Primary:   Treatment Difference for Acceptability of Treatment   [ Time Frame: Baseline up to end of study (Day 21) ]

2.  Primary:   Question 6 (Previous Pancreatic Enzyme Product [PEP])   [ Time Frame: Baseline ]

3.  Secondary:   Daily Number of Stools   [ Time Frame: Up to Day 10 in first and second treatment periods, end of study (Day 21) ]

4.  Secondary:   Number of Stools Categorized as Per Consistency   [ Time Frame: Up to Day 10 in first and second treatment periods, end of study (Day 21) ]

5.  Secondary:   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) ]

6.  Secondary:   Number of Abdominal Symptoms: Bloating   [ Time Frame: Up to Day 10 in first and second treatment periods, end of study (Day 21) ]

7.  Secondary:   Number of Abdominal Symptoms: Flatulence   [ Time Frame: Up to Day 10 in first and second treatment periods, end of study (Day 21) ]

8.  Secondary:   Number of Abdominal Pain Symptoms   [ Time Frame: Up to Day 10 in first and second treatment periods, end of study (Day 21) ]

9.  Secondary:   Number of Participants With Abnormal Clinical Laboratory and Vital Signs Findings   [ Time Frame: Baseline up to end of study (Day 21) ]

10.  Secondary:   Number of Participants With Abnormal Findings With Respect to Oral Mucosa   [ Time Frame: Baseline up to end of study (Day 21) ]


  Serious Adverse Events


  Other Adverse Events


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Limitations and Caveats
Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.  


Results Point of Contact:  
Name/Title: Robert Winkler, MD, VP, Clinical Development and Operations
Organization: Aptalis Pharma US, Inc.
phone: 1-800-472-2634


No publications provided


Responsible Party: Aptalis Pharma
ClinicalTrials.gov Identifier: NCT01100606     History of Changes
Other Study ID Numbers: PR-011
Study First Received: March 31, 2010
Results First Received: March 5, 2014
Last Updated: March 5, 2014
Health Authority: United States: Food and Drug Administration