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PR-018: An Open-Label, Safety Extension of Study PR-011

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ClinicalTrials.gov Identifier: NCT01131507
Recruitment Status : Completed
First Posted : May 27, 2010
Results First Posted : March 7, 2014
Last Update Posted : March 21, 2017
Sponsor:
Information provided by (Responsible Party):
Forest Laboratories

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Intervention Drug: EUR-1008 (APT-1008)
Enrollment 15
Recruitment Details Participants who completed previous study PR-011 (NCT01100606) and consented to continue treatment with EUR-1008 (APT-1008) 3,000 lipase units were enrolled in this study.
Pre-assignment Details  
Arm/Group Title EUR-1008 (APT-1008)
Hide Arm/Group Description EUR-1008 (APT-1008) (Zenpep® [pancrelipase] 3,000 lipase units delayed release capsules) from open capsules mixed with a small amount of apple juice or apple sauce orally starting at the same dose as administered at the end of study PR-011 (NCT01100606), with dose increments of 3,000 lipase units. The dose was adjusted based on participants' age and body weight. Total dose not to exceed 10,000 lipase units per kilogram (kg) of body weight per day unless clinically indicated. Total duration of study treatment was up to 12 months.
Period Title: Overall Study
Started 15
Completed 12
Not Completed 3
Reason Not Completed
Adverse Event             1
Lost to Follow-up             1
Withdrawal by Subject             1
Arm/Group Title EUR-1008 (APT-1008)
Hide Arm/Group Description EUR-1008 (APT-1008) (Zenpep® [pancrelipase] 3,000 lipase units delayed release capsules) from open capsules mixed with a small amount of apple juice or apple sauce orally starting at the same dose as administered at the end of study PR-011 (NCT01100606), with dose increments of 3,000 lipase units. The dose was adjusted based on participants' age and body weight. Total dose not to exceed 10,000 lipase units per kg body weight per day unless clinically indicated. Total duration of study treatment was up to 12 months.
Overall Number of Baseline Participants 15
Hide Baseline Analysis Population Description
Safety population included all participants who received at least 1 dose of study drug.
Age, Continuous  
Median (Full Range)
Unit of measure:  Months
Number Analyzed 15 participants
9.0
(3 to 14)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants
Female
6
  40.0%
Male
9
  60.0%
Weight  
Median (Full Range)
Unit of measure:  Kilogram
Number Analyzed 15 participants
7.80
(6.0 to 10.6)
Length   [1] 
Median (Full Range)
Unit of measure:  Centimeter
Number Analyzed 15 participants
68.5
(59 to 79)
[1]
Measure Description: Number of participants evaluable for this baseline characteristic = 14
1.Primary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Hide Description TEAE was any event not present prior to exposure to study drug or any event already present that worsened in either intensity or frequency following exposure to test drug. Serious AE (SAE) was any event that resulted in death, immediately life threatening, hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or a congenital anomaly/birth defect. Number of participants with TEAEs, SAEs, TEAE's relationship to study drug (unrelated, possible and probable) and on the basis of severity (mild [minimal/no treatment and did not interfere with daily activities], moderate [resulted in a low level of inconvenience or concern with the therapeutic measures and may have caused some interference with functioning] and severe [interrupted participant's usual daily activity, may have required systemic drug therapy or other treatment and were usually incapacitating]) with a frequency threshold of above 5% were reported.
Time Frame Up to Month 12 or early termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population included all participants who received at least 1 dose of study drug.
Arm/Group Title EUR-1008 (APT-1008)
Hide Arm/Group Description:
EUR-1008 (APT-1008) (Zenpep® [pancrelipase] 3,000 lipase units delayed release capsules) from open capsules mixed with a small amount of apple juice or apple sauce orally starting at the same dose as administered at the end of study PR-011 (NCT01100606), with dose increments of 3,000 lipase units. The dose was adjusted based on participants' age and body weight. Total dose not to exceed 10,000 lipase units per kg body weight per day unless clinically indicated. Total duration of study treatment was up to 12 months.
Overall Number of Participants Analyzed 15
Measure Type: Number
Unit of Measure: participants
All TEAEs 15
SAEs 3
Unrelated TEAEs 10
Possibly related TEAEs 4
Probably related TEAEs 1
Mild TEAEs 5
Moderate TEAEs 8
Severe TEAEs 2
2.Secondary Outcome
Title Change From Baseline in Growth Percentiles at Month 3, 6, 9 and 12
Hide Description Participant's ability to thrive was evaluated through growth percentiles for weight-for-age, length-for-age and weight-for-length recorded on Center for Disease Control and Prevention (CDC) growth charts during each treatment visit.
Time Frame Baseline, Month 3, 6, 9 and 12
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population included all participants who received at least 1 dose of study drug. Here, 'n' specifies number of participants who were evaluable for various categories at each time point.
Arm/Group Title EUR-1008 (APT-1008)
Hide Arm/Group Description:
EUR-1008 (APT-1008) (Zenpep® [pancrelipase] 3,000 lipase units delayed release capsules) from open capsules mixed with a small amount of apple juice or apple sauce orally starting at the same dose as administered at the end of study PR-011 (NCT01100606), with dose increments of 3,000 lipase units. The dose was adjusted based on participants' age and body weight. Total dose not to exceed 10,000 lipase units per kg body weight per day unless clinically indicated. Total duration of study treatment was up to 12 months.
Overall Number of Participants Analyzed 15
Median (Full Range)
Unit of Measure: growth percentile
Weight-for-age: Baseline (n=15)
22.0
(2 to 91)
Weight-for-age: Change at Month 3 (n=15)
2.0
(-18 to 34)
Weight-for-age: Change at Month 6 (n=13)
6.0
(-35 to 34)
Weight-for-age: Change at Month 9 (n=12)
4.0
(-42 to 34)
Weight-for-age: Change at Month 12 (n=12)
5.5
(-45 to 36)
Length-for-age: Baseline (n=14)
36.5
(1 to 74)
Length-for-age: Change at Month 3 (n=14)
2.0
(-22 to 30)
Length-for-age: Change at Month 6 (n=12)
2.0
(-23 to 28)
Length-for-age: Change at Month 9 (n=12)
11.0
(-24 to 34)
Length-for-age: Change at Month 12 (n=12)
0.5
(-27 to 53)
Weight-for-length: Baseline (n=14)
41.5
(8 to 98)
Weight-for-length: Change at Month 3 (n=14)
0.5
(-12 to 41)
Weight-for-length: Change at Month 6 (n=12)
13.5
(-17 to 47)
Weight-for-length: Change at Month 9 (n=12)
9.0
(-54 to 31)
Weight-for-length: Change at Month 12 (n=12)
10.5
(-57 to 48)
Time Frame Up to Month 12 or early termination
Adverse Event Reporting Description Adverse event (AE) was defined as any untoward medical occurrence regardless of causal relationship to study medication. Serious AE was any event that resulted in death, life threatening, required or prolonged in-patient hospitalization, significant disability/incapacity, or was a congenital anomaly/birth defect.
 
Arm/Group Title EUR-1008 (APT-1008)
Hide Arm/Group Description EUR-1008 (APT-1008) (Zenpep® [pancrelipase] 3,000 lipase units delayed release capsules) from open capsules mixed with a small amount of apple juice or apple sauce will be administered orally starting at the same dose as administered at the end of study PR-011 (NCT01100606), with dose increments of 3,000 lipase units. The dose was adjusted based on participants' age and body weight. Total dose not to exceed 10,000 lipase units per kg body weight per day unless clinically indicated. Total duration of study treatment was up to 12 months.
All-Cause Mortality
EUR-1008 (APT-1008)
Affected / at Risk (%)
Total   --/-- 
Hide Serious Adverse Events
EUR-1008 (APT-1008)
Affected / at Risk (%)
Total   3/15 (20.00%) 
Immune system disorders   
Anaphylactic shock * 1 [1]  1/15 (6.67%) 
Infections and infestations   
Bronchopneumonia * 1  1/15 (6.67%) 
Viral infection * 1  1/15 (6.67%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (13.0)
[1]
This AE was an acute reaction to peanuts and not related to study drug.
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
EUR-1008 (APT-1008)
Affected / at Risk (%)
Total   15/15 (100.00%) 
Cardiac disorders   
Tachycardia * 1  1/15 (6.67%) 
Congenital, familial and genetic disorders   
Cystic fibrosis lung * 1  3/15 (20.00%) 
Ear and labyrinth disorders   
Cerumen impaction * 1  1/15 (6.67%) 
Eye disorders   
Eyelid margin crusting * 1  1/15 (6.67%) 
Gastrointestinal disorders   
Diarrhoea * 1  5/15 (33.33%) 
Vomiting * 1  5/15 (33.33%) 
Steatorrhoea * 1  4/15 (26.67%) 
Abnormal faeces * 1  3/15 (20.00%) 
Constipation * 1  3/15 (20.00%) 
Abdominal distension * 1  2/15 (13.33%) 
Faecal volume increased * 1  2/15 (13.33%) 
Flatulence * 1  2/15 (13.33%) 
Post-tussive vomiting * 1  2/15 (13.33%) 
Teething * 1  2/15 (13.33%) 
Faeces hard * 1  1/15 (6.67%) 
Frequent bowel movements * 1  1/15 (6.67%) 
Haematochezia * 1  1/15 (6.67%) 
Malabsorption * 1  1/15 (6.67%) 
Salivary hypersecretion * 1  1/15 (6.67%) 
General disorders   
Pyrexia * 1  10/15 (66.67%) 
Fatigue * 1  3/15 (20.00%) 
Immune system disorders   
Drug hypersensitivity * 1  1/15 (6.67%) 
Infections and infestations   
Upper respiratory tract infection * 1  6/15 (40.00%) 
Otitis media * 1  5/15 (33.33%) 
Bronchitis * 1  2/15 (13.33%) 
Pseudomonas infection * 1  2/15 (13.33%) 
Rhinitis * 1  2/15 (13.33%) 
Sinusitis * 1  2/15 (13.33%) 
Acute sinusitis * 1  1/15 (6.67%) 
Croup infectious * 1  1/15 (6.67%) 
Ear infection * 1  1/15 (6.67%) 
Gastroenteritis * 1  1/15 (6.67%) 
Influenza * 1  1/15 (6.67%) 
Nasopharyngitis * 1  1/15 (6.67%) 
Oral candidiasis * 1  1/15 (6.67%) 
Pharyngitis streptococcal * 1  1/15 (6.67%) 
Pneumonia * 1  1/15 (6.67%) 
Respiratory tract infection * 1  1/15 (6.67%) 
Staphylococcal infection * 1  1/15 (6.67%) 
Investigations   
Weight decreased * 1  2/15 (13.33%) 
Band neutrophil count increased * 1  1/15 (6.67%) 
Pseudomonas test positive * 1  1/15 (6.67%) 
Metabolism and nutrition disorders   
Decreased appetite * 1  2/15 (13.33%) 
Vitamin D deficiency * 1  2/15 (13.33%) 
Weight gain poor * 1  1/15 (6.67%) 
Respiratory, thoracic and mediastinal disorders   
Rhinorrhoea * 1  12/15 (80.00%) 
Cough * 1  11/15 (73.33%) 
Wheezing * 1  8/15 (53.33%) 
Nasal congestion * 1  7/15 (46.67%) 
Productive cough * 1  4/15 (26.67%) 
Respiratory tract congestion * 1  2/15 (13.33%) 
Rhonchi * 1  2/15 (13.33%) 
Asthma * 1  1/15 (6.67%) 
Dyspnoea * 1  1/15 (6.67%) 
Haemoptysis * 1  1/15 (6.67%) 
Oropharyngeal pain * 1  1/15 (6.67%) 
Rales * 1  1/15 (6.67%) 
Sputum increased * 1  1/15 (6.67%) 
Tonsillar hypertrophy * 1  1/15 (6.67%) 
Skin and subcutaneous tissue disorders   
Dermatitis diaper * 1  2/15 (13.33%) 
Rash * 1  1/15 (6.67%) 
Rash generalised * 1  1/15 (6.67%) 
Rash macular * 1  1/15 (6.67%) 
Seborrhoea * 1  1/15 (6.67%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (13.0)
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.
Restrictions vary in accordance with each agreement with the individual investigators. Sponsor will allow publication after a multi-center publication has been published or after an agreed period of time if no such multi-center publication is submitted for publication. Sponsor can ask that Sponsor's confidential information be removed from any publication and can defer publication for a period of time to allow for Sponsor to obtain patent or other intellectual property right protection.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Robert Winkler, MD, VP, Clinical Development and Operations
Organization: Aptalis Pharma US, Inc.
Phone: 1-800-472-2634
Layout table for additonal information
Responsible Party: Forest Laboratories
ClinicalTrials.gov Identifier: NCT01131507    
Other Study ID Numbers: PR-018
First Submitted: May 25, 2010
First Posted: May 27, 2010
Results First Submitted: January 27, 2014
Results First Posted: March 7, 2014
Last Update Posted: March 21, 2017