Cystic FibrosisPhase 3NCT00880100
Use of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)
A Phase 3 study testing Ultrase® MT12 in people with Cystic Fibrosis and Pancreatic Insufficiency, run by Forest Laboratories at 15 sites in United States.
Trial summary
Multicenter, explorative, phase IIIb, open-label study to assess the efficacy and safety of Ultrase® MT12, in the control of steatorrhea and clinical signs and symptoms of malabsorption in CF children with pancreatic insufficiency (PI). This study is sponsored by Aptalis Pharma (formerly Axcan). Read more about this study
- Who is it for?
- Ages 2 to 6 years
- Conditions the study is for
- Cystic Fibrosis, Pancreatic Insufficiency
- What is being tested?
- Ultrase® MT12
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 49 participants
- Where
- 15 sitesUnited States
- Study dates
- Apr 2009to Nov 2009
- 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 2 to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female patients aged 2 to 6 years inclusively
- Patients with current diagnosis of CF based on one or more typical clinical features of CF or a sibling with CF or a positive newborn screening and at least either with sweat chloride test greater than or equal to 60 millimoles/liter (mmol/L) by quantitative pilocarpine iontophoresis on two separate occasions or two identifiable CF-causing mutations
- Patients with presence of PI as demonstrated by fecal elastase (FE-1) less than 100 microgram/gram (mcg/g) of stools (performed by ScheBo test) and requiring pancreatic enzyme supplementation
- Patients who are able to eat a high-fat diet calculated at a value between 2g to 4g fat/kg of body weight per day during the whole study and having a current adequate nutritional status based on the body mass index (BMI) greater than or equal to fifth percentile
- Patients receiving current treatment of PI with pancreatic enzymes
- The parent or legal guardian signed informed consent form (ICF) and is mentally able to understand and comply with the study procedures
Who cannot join
- Patients currently receiving or received an Ultrase® MT product (MT12, MT18, MT20) for PI in the last 30 days
- Patients having known contraindication, sensitivity or hypersensitivity to Ultrase® or to any porcine protein
- Patients with presence of a medical condition known to increase fecal fat loss or that could compromise study results or the study patient safety
- Patients with current diagnosis or history of complete distal intestinal obstruction syndrome (DIOS) in the past 6 months or who had 2 or more episodes of incomplete DIOS in the past year
- Patients with use of any prohibited medication or product at study entry and during the course of the study
- Patients with chronic use of narcotics
Show all 17Show fewer
- Patients with use of bowel stimulants and/or laxatives more than once a week
- Patients with presence of acute pancreatitis or exacerbation of chronic pancreatic disease
- Patients with presence of an acute infection that needed to be treated with oral or intravenous (IV) broad-spectrum antibiotics
- Patients having history of significant bowel resection; small bowel resection for meconium ileus at birth and appendectomy were accepted. Patients with Presence of dysmotility disorders
- Patients with presence of chronic or severe abdominal pain
- Patients unable to comply with diet requirement
- Patients receiving enteral tube feeding overnight at study entry or who will need to receive enteral tube feeding overnight during the course of the study
- Patients with history of or a current diagnosis of clinically significant portal hypertension
- Patients with presence of poorly controlled diabetes according to the Investigator's clinical judgment
- Patients having any condition or pre-study laboratory abnormality or history of any illness which, in the opinion of the Investigator, might have put the patient at risk, prevented the patient from completing the study, or otherwise affect the outcome of the study
- Patient with use of any investigational drug within 30 days prior to the date of signature of the ICF
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
United States15 sites
University of Michigan Health System Cystic Fibrosis Center
Ann Arbor, Michigan, 48109-0212
The Children's Hospital
Aurora, Colorado, 80045
Rainbow Babies and Children's Hospital - Cystic Fibrosis Center
Cleveland, Ohio, 44106
Children's Medical Center of Dayton
Dayton, Ohio, 45404
Duke University Medical Center
Durham, North Carolina, 27710
Helen DeVos Children's Hospital-Spectrum Health Research Department
Grand Rapids, Michigan, 40503
Pennsylvania State University and the Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
UW Hospital and Clinics
Madison, Wisconsin, 53792
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Respiratory Diseases of Children and Adolescents
Oklahoma City, Oklahoma, 73112
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15213
Virginia Commonwealth University
Richmond, Virginia, 23298
University of Utah
Salt Lake City, Utah, 84108
Sanford Children's Specialty Clinic
Sioux Falls, South Dakota, 57117-5039
SUNY Upstate Medical University
Syracuse, New York, 13203
About this study
Official title: Efficacy and Safety of Ultrase MT12 in the Control of Steatorrhea in Cystic Fibrosis (CF) and Pancreatic Insufficient (PI) Children Aged 2 to 6 Years Old
Multicenter, explorative, phase IIIb, open-label study to assess the efficacy and safety of Ultrase® MT12, in the control of steatorrhea and clinical signs and symptoms of malabsorption in CF children with pancreatic insufficiency (PI). This study is sponsored by Aptalis Pharma (formerly Axcan).
Read the detailed description
This is a multicenter, explorative, phase IIIb, open-label study in patients with CF and PI. The study consists of a screening visit (visit 1), followed by a baseline phase of 9 days (plus a 5-day window if necessary) during which the regular pancreatic enzyme will be maintained and 10 stool samples will be collected over 5 days, for baseline evaluation of steatorrhea. Afterward, a treatment phase of 19 days (plus a 5-day window if necessary) with Ultrase® MT12 will follow (the usual pancreatic enzyme will be replaced by Ultrase® MT12). Over the last 5 days of the treatment phase, 10 additional stool samples will be collected, for evaluation of steatorrhea.
- Conditions
- Cystic Fibrosis, Pancreatic Insufficiency
- Being tested
- Ultrase® MT12
- Keywords
- Steatorrhea, Malabsorption of fat, Pancreatic enzymes, Abdominal pain, Greasy stools
- Medical subject headings
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency, Steatorrhea, Abdominal Pain
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ultrase® MT12Drug
Ultrase® MT12 capsules will be given orally daily based on investigator's discretion to a maximum dose of 2,500 lipase units per kilogram (kg) body weight per meal or snack for 19 to 24 days during the treatment phase. Total maximum dose not to exceed 10,000 lipase units/kg/day.
| Study group | Role | What they receive |
|---|---|---|
| Ultrase® MT12 | Experimental | Not described Receives: Drug: Ultrase® MT12 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Patients With Control of Steatorrhea
Control of steatorrhea was defined as a less than 30 percent (%) of fat in stools as measured by nuclear magnetic resonance (NMR) spectroscopy in all stool samples which are collected at baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and during the 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12.
Measured: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Secondary outcomes (4)
Percentage of Patients With Normal Stool Frequency
Normal stool frequency was defined as having less than 4 bowel movements per day in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the Treatment Phase during which the PEP was replaced with Ultrase MT12.
Measured: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Percentage of Stools With Normal Consistency
Normal consistency of stool was defined as hard and formed or soft and formed consistency. Abnormal consistency was defined as loose and unformed stool or liquid stools and diarrhea. Percentage of stools with normal consistency of each patient was calculated from normal consistency of stools by the patient per day. Mean percentage of stools with normal consistency in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized.
Measured: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Percentage of Stools With Abnormal Characteristics
Stools of abnormal characteristics were defined as bulky/large, foul-smelling and/or oily stools. Mean percentage of stools with abnormal characteristics in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized.
Measured: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Mean Number of Days Without Abdominal Complaints
Abdominal complaints were defined as the reporting of abdominal pain and/or unusual and excessive flatulence/gas production. Mean number of days without abdominal complaints in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized.
Measured: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2009 (estimated)
- Study startApr 2009
- Primary completionNov 2009
- Study completionNov 2009
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
- 49 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
- NCT00880100
- Sponsor's protocol ID
- UMT12CF08-01
- Status last verified
- Feb 2017
- First submitted
- 9 Apr 2009
- 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 MT12 in the Control of Steatorrhea in Cystic Fibrosis (CF) and Pancreatic Insufficient (PI) Children Aged 2 to 6 Years Old
- ClinicalTrials.gov ID
- NCT00880100
- Acronym / sponsor ID
- UMT12CF08-01
- Conditions
- Cystic Fibrosis, Pancreatic Insufficiency
- Keywords
- Steatorrhea, Malabsorption of fat, Pancreatic enzymes, Abdominal pain, Greasy stools
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 49 participants (actual)
- Lead sponsor
- Forest LaboratoriesIndustry (pharma, biotech, devices)
- Study start
- Apr 2009
- Primary completion
- Nov 2009 (actual)
- Study completion
- Nov 2009 (actual)
- First posted
- 13 Apr 2009 (estimated)
- Results
- Posted 19 Feb 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Multicenter, explorative, phase IIIb, open-label study to assess the efficacy and safety of Ultrase® MT12, in the control of steatorrhea and clinical signs and symptoms of malabsorption in CF children with pancreatic insufficiency (PI). This study is sponsored by Aptalis Pharma (formerly Axcan).
Detailed description
This is a multicenter, explorative, phase IIIb, open-label study in patients with CF and PI. The study consists of a screening visit (visit 1), followed by a baseline phase of 9 days (plus a 5-day window if necessary) during which the regular pancreatic enzyme will be maintained and 10 stool samples will be collected over 5 days, for baseline evaluation of steatorrhea. Afterward, a treatment phase of 19 days (plus a 5-day window if necessary) with Ultrase® MT12 will follow (the usual pancreatic enzyme will be replaced by Ultrase® MT12). Over the last 5 days of the treatment phase, 10 additional stool samples will be collected, for evaluation of steatorrhea.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ultrase® MT12 | Experimental | Drug: Ultrase® MT12 |
Who may qualify
- Ages
- 2 to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Male or female patients aged 2 to 6 years inclusively
- Patients with current diagnosis of CF based on one or more typical clinical features of CF or a sibling with CF or a positive newborn screening and at least either with sweat chloride test greater than or equal to 60 millimoles/liter (mmol/L) by quantitative pilocarpine iontophoresis on two separate occasions or two identifiable CF-causing mutations
- Patients with presence of PI as demonstrated by fecal elastase (FE-1) less than 100 microgram/gram (mcg/g) of stools (performed by ScheBo test) and requiring pancreatic enzyme supplementation
- Patients who are able to eat a high-fat diet calculated at a value between 2g to 4g fat/kg of body weight per day during the whole study and having a current adequate nutritional status based on the body mass index (BMI) greater than or equal to fifth percentile
- Patients receiving current treatment of PI with pancreatic enzymes
Key exclusion criteria (17 listed)
- Patients currently receiving or received an Ultrase® MT product (MT12, MT18, MT20) for PI in the last 30 days
- Patients having known contraindication, sensitivity or hypersensitivity to Ultrase® or to any porcine protein
- Patients with presence of a medical condition known to increase fecal fat loss or that could compromise study results or the study patient safety
- Patients with current diagnosis or history of complete distal intestinal obstruction syndrome (DIOS) in the past 6 months or who had 2 or more episodes of incomplete DIOS in the past year
- Patients with use of any prohibited medication or product at study entry and during the course of the study
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® MT12Ultrase® MT12 capsules will be given orally daily based on investigator's discretion to a maximum dose of 2,500 lipase units per kilogram (kg) body weight per meal or snack for 19 to 24 days during the treatment phase. Total maximum dose not to exceed 10,000 lipase units/kg/day. | Drug | Ultrase® MT12 |
Primary outcome
Percentage of Patients With Control of Steatorrhea
Time frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Control of steatorrhea was defined as a less than 30 percent (%) of fat in stools as measured by nuclear magnetic resonance (NMR) spectroscopy in all stool samples which are collected at baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and during the 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12.
4 secondary outcomes
Percentage of Patients With Normal Stool Frequency
Time frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Percentage of Stools With Normal Consistency
Time frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Percentage of Stools With Abnormal Characteristics
Time frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Mean Number of Days Without Abdominal Complaints
Time frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
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
15 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® MT12 in people with Cystic Fibrosis and Pancreatic Insufficiency, run by Forest Laboratories at 15 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 6 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 15 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 19 Feb 2015). 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?
Percentage of Patients With Control of Steatorrhea (time frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)). 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.