Study to Evaluate the Safety and Efficacy of EUR-1008 (APT-1008) Pancreatic Enzyme Product in Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Aptalis Pharma
ClinicalTrials.gov Identifier:
NCT00297167
First received: February 27, 2006
Last updated: February 24, 2014
Last verified: February 2014
Results First Received: February 24, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Crossover Assignment;   Masking: Double-Blind;   Primary Purpose: Treatment
Conditions: Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Interventions: Drug: EUR-1008 (APT-1008)
Drug: Placebo

  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
Out of 34 participants who were enrolled and treated during open-label dose titration and stabilization period, 1 participant withdrew from the study before randomization to first double-blind intervention period.

Reporting Groups
  Description
Placebo First, Then EUR-1008 (APT-1008) Placebo matched to EUR-1008 (APT-1008) microtablets or minitablets contained in a capsule and was given orally daily in the first double-blind intervention period followed by EUR-1008 (APT-1008) microtablets (5000 lipase units) or minitablets (10000, 15000 or 20000 lipase units) contained in a capsule and was given orally daily in the second double-blind intervention period; at a dose based on investigator's discretion as achieved during dose titration and stabilization period (6 to 9 days). The capsules could be opened and sprinkled on acidic food and half of the established dose was used with snacks. Treatment duration for each double-blind intervention period was 2 days home treatment and 3 to 5 days hospital treatment. The stabilized dose was not to exceed 10,000 lipase units per kilogram body weight per day (lipase units/kg/day).
EUR-1008 (APT-1008) First, Then Placebo EUR-1008 (APT-1008) microtablets (5000 lipase units) or minitablets (10000, 15000 or 20000 lipase units) contained in a capsule and was given orally daily in the first double-blind intervention period followed by placebo matched to EUR-1008 (APT-1008) microtablets or minitablets contained in a capsule and was given orally daily in the second double-blind intervention period; at a dose based on investigator's discretion as achieved during dose titration and stabilization period (6 to 9 days). The capsules could be opened and sprinkled on acidic food and half of the established dose was used with snacks. Treatment duration for each double-blind intervention period was 2 days home treatment and 3 to 5 days hospital treatment. The stabilized dose was not to exceed 10,000 lipase units/kg/day.
EUR-1008 (APT-1008) (Open-label) EUR-1008 (APT-1008) microtablets (5000 lipase units) or minitablets (10000, 15000 or 20000 lipase units) contained in a capsule and was given orally daily at a fixed stabilized dose during open-label normalization period 1 (5 to 14 days) after first double-blind interventional period and during open-label normalization period 2 (7 days) after second double-blind interventional period.

Participant Flow for 4 periods

Period 1:   First Double-blind Intervention Period
    Placebo First, Then EUR-1008 (APT-1008)     EUR-1008 (APT-1008) First, Then Placebo     EUR-1008 (APT-1008) (Open-label)  
STARTED     17     16     0  
COMPLETED     17     16     0  
NOT COMPLETED     0     0     0  

Period 2:   Open-label Dose Normalization Period 1
    Placebo First, Then EUR-1008 (APT-1008)     EUR-1008 (APT-1008) First, Then Placebo     EUR-1008 (APT-1008) (Open-label)  
STARTED     0     0     33  
COMPLETED     0     0     32  
NOT COMPLETED     0     0     1  
Withdrawal by Subject                 0                 0                 1  

Period 3:   Second Double-blind Intervention Period
    Placebo First, Then EUR-1008 (APT-1008)     EUR-1008 (APT-1008) First, Then Placebo     EUR-1008 (APT-1008) (Open-label)  
STARTED     17     15     0  
COMPLETED     16     15     0  
NOT COMPLETED     1     0     0  
Protocol Violation                 1                 0                 0  

Period 4:   Open-label Dose Normalization Period 2
    Placebo First, Then EUR-1008 (APT-1008)     EUR-1008 (APT-1008) First, Then Placebo     EUR-1008 (APT-1008) (Open-label)  
STARTED     0     0     31  
COMPLETED     0     0     31  
NOT COMPLETED     0     0     0  



  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 population included all participants who received at least 1 dose of study treatment.

Reporting Groups
  Description
Entire Study Population Includes participants who received EUR-1008 (APT-1008) in open-label dose titration and stabilization phase; and EUR-1008 (APT-1008) first and placebo first after randomization to study treatment.

Baseline Measures
    Entire Study Population  
Number of Participants  
[units: participants]
  34  
Age  
[units: years]
Mean ± Standard Deviation
  14.9  ± 4.75  
Gender  
[units: participants]
 
Female     17  
Male     17  



  Outcome Measures
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1.  Primary:   Percent Coefficient of Fat Absorption (CFA%)   [ Time Frame: Day 3 up to Day 6 of hospital treatment in first and second double-blind intervention periods ]

2.  Secondary:   Percent Coefficient of Nitrogen Absorption (CNA%)   [ Time Frame: Day 3 up to Day 6 of hospital treatment in first and second double-blind intervention periods ]

3.  Secondary:   Lipid Levels   [ Time Frame: End of treatment (Day 6 during first and second double-blind intervention periods) ]

4.  Secondary:   Vitamin A Levels   [ Time Frame: End of treatment (Day 6 during first and second double-blind intervention periods) ]

5.  Secondary:   Vitamin E Levels   [ Time Frame: End of treatment (Day 6 during first and second double-blind intervention periods) ]

6.  Secondary:   Mean Daily Number of Stools   [ Time Frame: Day 3 up to Day 6 during first and second double-blind intervention periods ]

7.  Secondary:   Percentage of Stool Categorized as Per Consistency   [ Time Frame: Day 3 up to Day 6 during first and second double-blind intervention periods ]

8.  Secondary:   Mean Number of Abdominal Symptoms   [ Time Frame: Day 3 up to Day 6 during first and second double-blind intervention periods ]

9.  Other Pre-specified:   Percentage of Visible Oil or Grease in Stool   [ Time Frame: Day 3 up to Day 6 of hospital treatment in first and second double-blind intervention periods ]

10.  Other Pre-specified:   Percentage of Stools With Blood   [ Time Frame: Day 3 up to Day 6 of hospital treatment in first and second double-blind intervention periods ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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
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  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.


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 by Aptalis Pharma

Publications automatically indexed to this study:

Responsible Party: Aptalis Pharma
ClinicalTrials.gov Identifier: NCT00297167     History of Changes
Other Study ID Numbers: EUR-1008-M
Study First Received: February 27, 2006
Results First Received: February 24, 2014
Last Updated: February 24, 2014
Health Authority: United States: Food and Drug Administration