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Control of Steatorrhea 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:
NCT01327703
First received: March 28, 2011
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: Exocrine Pancreatic Insufficiency
Cystic Fibrosis
Interventions: Drug: Panzytrat® 25,000
Drug: Kreon® 25,000

  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
No text entered.

Reporting Groups
  Description
Panzytrat® First, Then Kreon® Panzytrat® 25,000 capsule orally daily at a stabilized dose, as per investigator's discretion, for 14 days in first treatment period followed by Kreon® 25,000 capsule orally daily at a stabilized dose, as per investigator's discretion, for 14 days in second treatment period. Stabilized dose for a participant was the optimal dose determined during a qualification phase that preceded the first treatment period and was based upon the participant's usual lipase and lipid intake and total dose was not to exceed 10,000 European Pharmacopoeia (Ph.Eur.) units lipase/kilogram (kg) body weight/day.
Kreon® First, Then Panzytrat® Kreon® 25,000 capsule orally daily at a stabilized dose, as per investigator's discretion, for 14 days in first treatment period followed by Panzytrat® 25,000 capsule orally daily at a stabilized dose, as per investigator's discretion, for 14 days in second treatment period. Stabilized dose for a participant was the optimal dose determined during a qualification phase that preceded the first treatment period and was based upon the participant's usual lipase and lipid intake and total dose was not to exceed 10,000 Ph.Eur. units lipase/kg body weight/day.

Participant Flow for 2 periods

Period 1:   First Treatment Period
    Panzytrat® First, Then Kreon®     Kreon® First, Then Panzytrat®  
STARTED     42     45  
COMPLETED     42     45  
NOT COMPLETED     0     0  

Period 2:   Second Treatment Period
    Panzytrat® First, Then Kreon®     Kreon® First, Then Panzytrat®  
STARTED     40 [1]   44 [2]
COMPLETED     39     42  
NOT COMPLETED     1     2  
Adverse Event                 1                 1  
Protocol Violation                 0                 1  
[1] Two participants discontinued between first and second treatment periods due to adverse event.
[2] One participant discontinued between first and second treatment periods due to adverse event.



  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.
Intent-to-treat (ITT) population included all randomized participants.

Reporting Groups
  Description
Entire Study Population Includes randomized participants who received Panzytrat® 25,000 first and Kreon® 25,000 first.

Baseline Measures
    Entire Study Population  
Number of Participants  
[units: participants]
  87  
Age  
[units: years]
Mean ± Standard Deviation
  13.6  ± 6.14  
Gender  
[units: participants]
 
Female     34  
Male     53  
Nutritional status as assessed by Vitamin A and E level [1]
[units: micromole/literĀ (mcmol/L)]
Mean ± Standard Deviation
 
Vitamin A (n=87)     1.403  ± 0.5329  
Vitamin E (Alpha-Tocopherol) (n=87)     18.554  ± 8.9713  
Vitamin E (Beta-Gamma-Tocopherol) (n=83)     1.231  ± 2.0781  
Nutritional status as assessed by Vitamin D level [2]
[units: nanomole/LĀ (nmol/L)]
Mean ± Standard Deviation
  55.4  ± 33.24  
[1] Nutritional status was assessed by determining Vitamin A and E (Alpha-Tocopherol and Beta-Gamma-Tocopherol) level. Here, 'n' signifies number of participants who were evaluable for the specific categories of this baseline characteristic.
[2] Nutritional status was assessed by determining Vitamin D level. Number of participants who were evaluable for Vitamin D level was 86.



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Percent Coefficient of Fat Absorption (CFA)   [ Time Frame: Day 12 up to Day 15 in first and second treatment periods ]

2.  Secondary:   Mean Daily Number of Stools   [ Time Frame: Day 12 up to Day 15 in first and second treatment periods ]

3.  Secondary:   Percentage of Stools With Normal Consistency   [ Time Frame: Day 12 up to Day 15 in first and second treatment periods ]

4.  Secondary:   Total Weight of Stools   [ Time Frame: Day 12 up to Day 15 in first and second treatment periods ]

5.  Secondary:   Mean Weight Per Stool Sample   [ Time Frame: Day 12 up to Day 15 in first and second treatment periods ]

6.  Secondary:   Relative Frequency of Days With Abdominal Symptoms   [ Time Frame: Day 1 up to Day 15 in first and second treatment periods ]

7.  Secondary:   Percentage of Participants With Abdominal Distension   [ Time Frame: Day 1 up to Day 15 in first and second treatment periods ]

8.  Secondary:   Percent Coefficient of Fat Absorption (CFA) Based on Concomitant Use of Proton Pump Inhibitors (PPIs)   [ Time Frame: Day 12 up to Day 15 in first and second treatment periods ]

9.  Secondary:   Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs)   [ Time Frame: Baseline up to 30 days after last dose ]

10.  Secondary:   Nutritional Status as Assessed by Body Weight   [ Time Frame: Baseline, end of treatment (within 3 days after Day 15 of first and second treatment periods) or early discontinuation ]

11.  Secondary:   Nutritional Status as Assessed by Body Mass Index (BMI)   [ Time Frame: Baseline, end of treatment (within 3 days after Day 15 of first and second treatment periods) or early discontinuation ]

12.  Secondary:   Nutritional Status as Assessed by Electrolytes Level   [ Time Frame: Baseline, end of treatment (within 3 days after Day 15 of first and second treatment periods) or early discontinuation ]

13.  Secondary:   Nutritional Status as Assessed by Albumin, Serum Transferrin and Hemoglobin Level   [ Time Frame: Baseline, end of treatment (within 3 days after Day 15 of first and second treatment periods) or early discontinuation ]

14.  Secondary:   Nutritional Status as Assessed by Hematocrit Level   [ Time Frame: Baseline, end of treatment (within 3 days after Day 15 of first and second treatment periods) or early discontinuation ]


  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


Responsible Party: Aptalis Pharma
ClinicalTrials.gov Identifier: NCT01327703     History of Changes
Other Study ID Numbers: MA-PA25CF10-01, 2010-019267-11
Study First Received: March 28, 2011
Results First Received: March 5, 2014
Last Updated: March 5, 2014
Health Authority: Germany: Federal Institute for Drugs and Medical Devices
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products