Trial Outcomes & Findings for Efficacy and Safety of Ultrase® MT20 in Improving the Coefficient of Fat Absorption (CFA) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI) (NCT NCT00513682)
NCT ID: NCT00513682
Last Updated: 2017-03-16
Results Overview
Percent CFA was calculated as (\[fat intake - fat excretion\]/fat intake)\*100, determined by the stools collected during the 72-hour period in either washout phase or treatment phase. Mean percent CFA was calculated for Day 3 to Day 5 or Day 6 of the respective phase.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
9 participants
Primary outcome timeframe
Day 3 to Day 5 or Day 6 during washout phase and treatment phase
Results posted on
2017-03-16
Participant Flow
Participant milestones
| Measure |
Ultrase® MT20
Ultrase® MT20 capsules orally with each meal during Day 1 to 15 in screening phase at a dose based on investigator's discretion. During Day 12 to 15, participants received high-fat diet and Ultrase® MT20 dose was adjusted depending on symptoms of steatorrhea. This was followed by a washout phase of 6 to 7 days, in which participants received only high-fat diet. Then the treatment phase was started which consisted of a stabilized dose of Ultrase® MT20 capsule (as identified during screening phase) orally for 7 to 11 days. The stabilized dose not to exceed 2500 lipase units per kilogram body weight per meal (lipase units/kg/meal).
|
|---|---|
|
Screening Phase
STARTED
|
9
|
|
Screening Phase
COMPLETED
|
9
|
|
Screening Phase
NOT COMPLETED
|
0
|
|
Washout Phase
STARTED
|
9
|
|
Washout Phase
COMPLETED
|
7
|
|
Washout Phase
NOT COMPLETED
|
2
|
|
Treatment Phase
STARTED
|
7
|
|
Treatment Phase
COMPLETED
|
7
|
|
Treatment Phase
NOT COMPLETED
|
0
|
Reasons for withdrawal
| Measure |
Ultrase® MT20
Ultrase® MT20 capsules orally with each meal during Day 1 to 15 in screening phase at a dose based on investigator's discretion. During Day 12 to 15, participants received high-fat diet and Ultrase® MT20 dose was adjusted depending on symptoms of steatorrhea. This was followed by a washout phase of 6 to 7 days, in which participants received only high-fat diet. Then the treatment phase was started which consisted of a stabilized dose of Ultrase® MT20 capsule (as identified during screening phase) orally for 7 to 11 days. The stabilized dose not to exceed 2500 lipase units per kilogram body weight per meal (lipase units/kg/meal).
|
|---|---|
|
Washout Phase
Adverse Event
|
2
|
Baseline Characteristics
Efficacy and Safety of Ultrase® MT20 in Improving the Coefficient of Fat Absorption (CFA) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI)
Baseline characteristics by cohort
| Measure |
Ultrase® MT20
n=9 Participants
Ultrase® MT20 capsules orally with each meal during Day 1 to 15 in screening phase at a dose based on investigator's discretion. During Day 12 to 15, participants received high-fat diet and Ultrase® MT20 dose was adjusted depending on symptoms of steatorrhea. This was followed by a washout phase of 6 to 7 days, in which participants received only high-fat diet. Then the treatment phase was started which consisted of a stabilized dose of Ultrase® MT20 capsule (as identified during screening phase) orally for 7 to 11 days. The stabilized dose not to exceed 2500 lipase units per kilogram body weight per meal (lipase units/kg/meal).
|
|---|---|
|
Age, Continuous
|
9.8 years
STANDARD_DEVIATION 1.64 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 3 to Day 5 or Day 6 during washout phase and treatment phasePopulation: Intent-to-treat (ITT) population included all the participants who took at least 1 dose of Ultrase® MT20 and had completed at least 1 phase (washout or treatment phase) evaluating primary and secondary efficacy parameters.
Percent CFA was calculated as (\[fat intake - fat excretion\]/fat intake)\*100, determined by the stools collected during the 72-hour period in either washout phase or treatment phase. Mean percent CFA was calculated for Day 3 to Day 5 or Day 6 of the respective phase.
Outcome measures
| Measure |
Ultrase® MT20 Washout Phase
n=7 Participants
Ultrase® MT20 capsules orally with each meal during Day 1 to 15 in screening phase at a dose based on investigator's discretion. During Day 12 to 15, participants received high-fat diet and Ultrase® MT20 dose was adjusted depending on symptoms of steatorrhea. This was followed by a washout phase of 6 to 7 days, in which participants received only high-fat diet.
|
Ultrase® MT20 Treatment Phase
n=7 Participants
Participants who received Ultrase® MT20 capsules orally in screening phase and underwent washout phase, received stabilized dose of Ultrase® MT20 capsule (as identified during screening phase) orally for 7 to 11 days during treatment phase. The stabilized dose not to exceed 2500 lipase units per kilogram body weight per meal (unit/kg/meal).
|
|---|---|---|
|
Percent Coefficient of Fat Absorption (CFA)
|
34.5 Percent CFA
Standard Deviation 21.35
|
82.7 Percent CFA
Standard Deviation 13.25
|
SECONDARY outcome
Timeframe: Day 3 to Day 5 or Day 6 during washout phase and treatment phasePopulation: Intent-to-treat (ITT) population included all the participants who took at least 1 dose of Ultrase® MT20 and had completed at least 1 phase (washout or treatment phase) evaluating primary and secondary efficacy parameters.
Percent CNA was calculated as \[(nitrogen intake-nitrogen excretion)/nitrogen intake\]\*100, determined by the stools collected during the 72- hour period in either washout phase or treatment phase. Nitrogen intake was calculated as protein intake/6.25. Nitrogen excretion was measured as total fecal nitrogen. Mean percent CNA was calculated for Day 3 to Day 5 or Day 6 of the respective phase.
Outcome measures
| Measure |
Ultrase® MT20 Washout Phase
n=7 Participants
Ultrase® MT20 capsules orally with each meal during Day 1 to 15 in screening phase at a dose based on investigator's discretion. During Day 12 to 15, participants received high-fat diet and Ultrase® MT20 dose was adjusted depending on symptoms of steatorrhea. This was followed by a washout phase of 6 to 7 days, in which participants received only high-fat diet.
|
Ultrase® MT20 Treatment Phase
n=7 Participants
Participants who received Ultrase® MT20 capsules orally in screening phase and underwent washout phase, received stabilized dose of Ultrase® MT20 capsule (as identified during screening phase) orally for 7 to 11 days during treatment phase. The stabilized dose not to exceed 2500 lipase units per kilogram body weight per meal (unit/kg/meal).
|
|---|---|---|
|
Percent Coefficient of Nitrogen Absorption (CNA)
|
33.4 Percent CNA
Standard Deviation 20.06
|
79.5 Percent CNA
Standard Deviation 10.56
|
Adverse Events
Ultrase® MT20 Screening Phase
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Ultrase® MT20 Washout Phase
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Ultrase® MT20 Treatment Phase
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Ultrase® MT20 Screening Phase
n=9 participants at risk
Ultrase® MT20 capsules orally with each meal during Day 1 to 15 in screening phase at a dose based on investigator's discretion. During Day 12 to 15, participants received high-fat diet and Ultrase® MT20 dose was adjusted depending on symptoms of steatorrhea.
|
Ultrase® MT20 Washout Phase
n=9 participants at risk
Participants who received Ultrase® MT20 capsules orally in screening phase and underwent a washout phase, of 6 to 7 days, in which participants received only high-fat diet and refrained from taking Ultrase® MT20.
|
Ultrase® MT20 Treatment Phase
n=7 participants at risk
Participants who received Ultrase® MT20 capsules orally in screening phase and underwent washout phase, received stabilized dose of Ultrase® MT20 capsule (as identified during screening phase) orally for 7 to 11 days during treatment phase. The stabilized dose not to exceed 2500 lipase units per kilogram body weight per meal (unit/kg/meal).
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal discomfort
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
11.1%
1/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
33.3%
3/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
11.1%
1/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Gastrointestinal disorders
Abnormal faeces
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
22.2%
2/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
11.1%
1/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
11.1%
1/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Investigations
Weight decreased
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
11.1%
1/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
14.3%
1/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Nervous system disorders
Headache
|
11.1%
1/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
11.1%
1/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
11.1%
1/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
14.3%
1/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal disorder
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
14.3%
1/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
11.1%
1/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
11.1%
1/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
11.1%
1/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Ear and labyrinth disorders
Ear pain
|
11.1%
1/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
|
Infections and infestations
Beta haemolytic streptococcal infection
|
11.1%
1/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/9 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
0.00%
0/7 • Day 1 of screening phase up to follow up (7 to 10 days post-treatment phase)
Safety population included all participants who signed the informed consent form and took at least 1 dose of the study medication, including medication dispensed for the screening phase. Number of participants evaluable for each reporting group were 9 and 7 respectively.
|
Additional Information
Robert Winkler, MD, VP, Clinical Development and Operations
Aptalis Pharma US, Inc.
Phone: 1-800-472-2634
Results disclosure agreements
- Principal investigator is a sponsor employee Restrictions may vary in accordance with each agreement that is negotiated with individual investigators. Sponsor will allow publication after 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 defer publication for period of time to allow Sponsor to obtain patent or other intellectual property right protection.
- Publication restrictions are in place
Restriction type: OTHER