Cystic FibrosisPhase 3NCT00513682
Efficacy and Safety of Ultrase® MT20 in Improving the Coefficient of Fat Absorption (CFA) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI)
A Phase 3 study testing Ultrase® MT20 in people with Cystic Fibrosis and Pancreatic Insufficiency, run by Forest Laboratories at 3 sites in United States.
Trial summary
This is a Phase III, multicenter, open-label study, that will evaluate the improvement of nutrient absorption when participants will receive Ultrase® MT20. This study is sponsored by Aptalis (formerly Axcan). This study is performed in children from 7 to 11 years old. Read more about this study
- Who is it for?
- Ages 7 to 11 years
- Conditions the study is for
- Cystic Fibrosis, Pancreatic Insufficiency
- What is being tested?
- Ultrase® MT20
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 3 sitesUnited States
- Study dates
- Jul 2007to Apr 2008
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- After a full explanation of the study, participants or their legally authorized representative must have signed the informed consent or assent form along with a parental form
- Participants must have a confirmed diagnosis of CF based on one or more clinical features consistent with the CF phenotype, and one of the following:
- A genotype with two identifiable mutations consistent with CF
- A sweat chloride test greater that 60 millimole per liter (mmol/L) by quantitative pilocarpine iontophoresis
- Participants must have PI as demonstrated by a fecal elastase-1 (FE-1) concentration less than 100 microgram per gram (mcg/g) of stools (ScheBo test) and requiring pancreatic enzyme supplementation
- Participants must be clinically stable according to the physician's judgment by:
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- Medical and medication history
- Baseline physical examination including vital signs and laboratory analyses
- Participants must be 7 to 11 years of age
- Participants must have an adequate nutritional status based on body mass index (BMI) greater than or equal to fifth percentile
- Participants must be on an optimal clinical dose of pancreatic enzymes (Ultrase® MT12 , MT18 or MT20 or other pancreatic enzyme preparations) prior to entry in the study and must tolerate this medication in the opinion of the investigator
- Participants must be able to be able to swallow capsules and eat a high-fat diet calculated as 2 gram fat per kilogram (g fat/kg) of body weight per day
- Participants must be, in the opinion of the investigator, able and willing to complete the study
- Female participants should be premenarcheal. Otherwise, a female participant of childbearing potential (WOCBP) must not be pregnant and must have practiced an acceptable method of contraception for at least 1 month prior to the study entry
Who cannot join
- Participants with a known contraindication, sensitivity or hypersensitivity to Ultrase® MT20 or any porcine protein
- Participants with a known allergy to the food, drug and cosmetic (FD\&C) Blue No. 2 dye indicator (stool marker)
- Participants who are not willing to stop the prohibited medications or products at study entry and throughout the study
- Participants who use narcotics chronically and bowel stimulants and/or laxatives on a regular basis
- Participants with acute pancreatitis or acute exacerbation of chronic pancreatic disease
- Participants with an acute pulmonary infection
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- Participants with a history of bowel resection
- Participants suffering from any dysmotility disorders
- Participants with chronic or severe abdominal pain
- Participants receiving enteral tube feeding and not willing to stop during the course of the study
- Participants with a significant medical disease that would compromise their welfare or confound the study results
- Participants with a history of or a current diagnosis of clinically significant portal hypertension
- Participants who have a condition known to increase fecal fat loss including celiac's disease, biliary cancer, biliary stricture, cholelithiasis, Crohn's disease, ulcerative colitis, pancreas cancer, radiation enteritis, tropical sprue, Whipple's disease, lactose intolerance, pseudomembranous colitis
- Participants with a current diagnosis or a history of complete distal intestinal obstruction syndrome (DIOS) in the past 6 months; or, participants who had 2 or more episodes of DIOS in the past year
- Participants with poorly controlled diabetes according to the investigator's opinion
- Female participants who are pregnant or lactating
- Participants who received an investigational drug within 30 days prior to entry into the study
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of Michigan Health System Cystic Fibrosis Center
Ann Arbor, Michigan, 48109-0212
Cystic Fibrosis Center Rainbow Babies and Children's HospitalDivision
Cleveland, Ohio, 44106
Pennsylvania State University And the Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
About this study
Official title: Efficacy and Safety of Ultrase® MT20 in Improving the Coefficient of Fat Absorption (CFA%) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI)
This is a Phase III, multicenter, open-label study, that will evaluate the improvement of nutrient absorption when participants will receive Ultrase® MT20. This study is sponsored by Aptalis (formerly Axcan). This study is performed in children from 7 to 11 years old.
Read the detailed description
This is a Phase III, multicenter, open-label study, which will quantify the improvement of nutrient absorption when participants are receiving Ultrase® MT20. The improvement will be demonstrated by comparing the CFA percent (%) and CNA% obtained during a washout of enzyme with the CFA% and CNA% obtained during a period of treatment with Ultrase® MT20. The study is also designed to obtain safety data in CF children suffering also from PI taking Ultrase® MT20. The total duration for the participation of children in this study will be approximately up to 38 days and will include 3 phases: screening phase, the washout phase and treatment phase.
Screening phase: this phase will last 15 days and all participants will take Ultrase® MT20 as per investigator's discretion during this period. During the last 4 days, participants will be stabilized on a high fat diet and with Ultrase® MT20. The individual 'stabilized dose' of Ultrase® MT20 capsules will be determined for each participant based on the average number of capsules of Ultrase® MT20 taken during last 4 days.
Washout phase: this phase will last 6 to 7 days. The participants will continue the high-fat diet but will refrain from taking Ultrase® MT20 or any other enzymes. A 72-hour stool collection will be performed and all food consumed by the participants will be recorded to assess the CFA% and CNA%.
Treatment phase: this phase will last 7 to 11 days. The participants will continue the high-fat diet and will take the 'stabilized dose' of Ultrase® MT20 established during screening. Another 72-hour stool collection will be performed and all food consumed by the participants will be recorded to assess the CFA% and CNA%.
- Conditions
- Cystic Fibrosis, Pancreatic Insufficiency
- Being tested
- Ultrase® MT20
- Keywords
- Cystic Fibrosis, Pancreatic insufficiency, Steatorrhea, Children 7 to 11 years, Pancreatic enzyme
- Medical subject headings
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency, Steatorrhea
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ultrase® MT20Drug
Ultrase® MT20 capsules will be administered 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 will receive high-fat diet and Ultrase® MT20 dose will be adjusted depending on symptoms of steatorrhea. This will be followed by a washout phase of 6 to 7 days, in which participants will receive only high-fat diet; then stabilized dose of Ultrase® MT20 capsule (as identified during screening phase) will be administered orally for 7 to 11 days during treatment phase. The stabilized dose should not to exceed 2500 lipase units per kilogram body weight per meal (lipase units/kg/meal).
| Study group | Role | What they receive |
|---|---|---|
| Ultrase® MT20 | Experimental | Not described Receives: Drug: Ultrase® MT20 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Coefficient of Fat Absorption (CFA)
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.
Measured: Day 3 to Day 5 or Day 6 during washout phase and treatment phase
Secondary outcomes (1)
Percent Coefficient of Nitrogen Absorption (CNA)
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.
Measured: Day 3 to Day 5 or Day 6 during washout phase and treatment phase
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2007 (estimated)
- Study startJul 2007
- Primary completionMar 2008
- Study completionApr 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Forest Laboratories
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Aptalis Medical Information, Study director, Forest Laboratories
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00513682
- Sponsor's protocol ID
- UMT20CF07-01
- Status last verified
- Feb 2017
- First submitted
- 7 Aug 2007
- Last updated
- 16 Mar 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Ultrase® MT20 in Improving the Coefficient of Fat Absorption (CFA%) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI)
- ClinicalTrials.gov ID
- NCT00513682
- Acronym / sponsor ID
- UMT20CF07-01
- Conditions
- Cystic Fibrosis, Pancreatic Insufficiency
- Keywords
- Cystic Fibrosis, Pancreatic insufficiency, Steatorrhea, Children 7 to 11 years, Pancreatic enzyme
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Forest LaboratoriesIndustry (pharma, biotech, devices)
- Study start
- Jul 2007
- Primary completion
- Mar 2008 (actual)
- Study completion
- Apr 2008 (actual)
- First posted
- 9 Aug 2007 (estimated)
- Results
- Posted 10 Apr 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase III, multicenter, open-label study, that will evaluate the improvement of nutrient absorption when participants will receive Ultrase® MT20. This study is sponsored by Aptalis (formerly Axcan). This study is performed in children from 7 to 11 years old.
Detailed description
This is a Phase III, multicenter, open-label study, which will quantify the improvement of nutrient absorption when participants are receiving Ultrase® MT20. The improvement will be demonstrated by comparing the CFA percent (%) and CNA% obtained during a washout of enzyme with the CFA% and CNA% obtained during a period of treatment with Ultrase® MT20. The study is also designed to obtain safety data in CF children suffering also from PI taking Ultrase® MT20. The total duration for the participation of children in this study will be approximately up to 38 days and will include 3 phases: screening phase, the washout phase and treatment phase.
Screening phase: this phase will last 15 days and all participants will take Ultrase® MT20 as per investigator's discretion during this period. During the last 4 days, participants will be stabilized on a high fat diet and with Ultrase® MT20. The individual 'stabilized dose' of Ultrase® MT20 capsules will be determined for each participant based on the average number of capsules of Ultrase® MT20 taken during last 4 days.
Washout phase: this phase will last 6 to 7 days. The participants will continue the high-fat diet but will refrain from taking Ultrase® MT20 or any other enzymes. A 72-hour stool collection will be performed and all food consumed by the participants will be recorded to assess the CFA% and CNA%.
Treatment phase: this phase will last 7 to 11 days. The participants will continue the high-fat diet and will take the 'stabilized dose' of Ultrase® MT20 established during screening. Another 72-hour stool collection will be performed and all food consumed by the participants will be recorded to assess the CFA% and CNA%.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ultrase® MT20 | Experimental | Drug: Ultrase® MT20 |
Who may qualify
- Ages
- 7 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (14 listed)
- After a full explanation of the study, participants or their legally authorized representative must have signed the informed consent or assent form along with a parental form
- Participants must have a confirmed diagnosis of CF based on one or more clinical features consistent with the CF phenotype, and one of the following:
- Participants must have PI as demonstrated by a fecal elastase-1 (FE-1) concentration less than 100 microgram per gram (mcg/g) of stools (ScheBo test) and requiring pancreatic enzyme supplementation
- Participants must be clinically stable according to the physician's judgment by:
- Participants must be 7 to 11 years of age
Key exclusion criteria (17 listed)
- Participants with a known contraindication, sensitivity or hypersensitivity to Ultrase® MT20 or any porcine protein
- Participants with a known allergy to the food, drug and cosmetic (FD\&C) Blue No. 2 dye indicator (stool marker)
- Participants who are not willing to stop the prohibited medications or products at study entry and throughout the study
- Participants who use narcotics chronically and bowel stimulants and/or laxatives on a regular basis
- Participants with acute pancreatitis or acute exacerbation of chronic pancreatic disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ultrase® MT20Ultrase® MT20 capsules will be administered 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 will receive high-fat diet and Ultrase® MT20 dose will be adjusted depending on symptoms of steatorrhea. This will be followed by a washout phase of 6 to 7 days, in which participants will receive only high-fat diet; then stabilized dose of Ultrase® MT20 capsule (as identified during screening phase) will be administered orally for 7 to 11 days during treatment phase. The stabilized dose should not to exceed 2500 lipase units per kilogram body weight per meal (lipase units/kg/meal). | Drug | Ultrase® MT20 |
Primary outcome
Percent Coefficient of Fat Absorption (CFA)
Time frame: Day 3 to Day 5 or Day 6 during washout phase and treatment phase
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.
1 secondary outcome
Percent Coefficient of Nitrogen Absorption (CNA)
Time frame: Day 3 to Day 5 or Day 6 during washout phase and treatment phase
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Aptalis Medical Information study director
Forest Laboratories
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ultrase® MT20 in people with Cystic Fibrosis and Pancreatic Insufficiency, run by Forest Laboratories at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Apr 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Coefficient of Fat Absorption (CFA) (time frame: Day 3 to Day 5 or Day 6 during washout phase and treatment phase). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Mar 2017.