CompletedNCT00880100Updated Mar 16, 2017Results posted

Use of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)

A Phase 3 interventional study of Ultrase® MT12 in Cystic Fibrosis and Pancreatic Insufficiency, sponsored by Forest Laboratories. Completed at 15 sites in United States. Open to participants aged 2 Years to 6 Years. Per ClinicalTrials.gov, last updated 2017-03-16.

Sponsored by Forest Laboratories · Phase 3, Interventional, and Treatment

Phase: Phase 3
Study type: Interventional
Enrollment: 49
Allocation: Not applicable
Ages: 2 Years to 6 Years
Sex: All

Study 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 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 studied

Cystic Fibrosis
Pancreatic Insufficiency

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Keywords

Steatorrhea
Malabsorption of fat
Pancreatic enzymes
Abdominal pain
Greasy stools

In context

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's enrollment of 49 is above the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

Forest Laboratories is the lead sponsor of 165 studies on the registry; none are open to participants now.

Of its 6 completed or terminated interventional studies of FDA-regulated products, 6 (100%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 2 Years to 6 Years
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

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

Exclusion criteria

Exclusion Criteria:

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
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

Study design

Phase: Phase 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 49 participants (actual)

Study arms

Experimental
Ultrase® MT12
Drug: Ultrase® MT12

Interventions

DrugUltrase® MT12
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.

What researchers measure

Primary outcomes

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.
Time frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)

Secondary outcomes

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.
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
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.
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
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.
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
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.
Time frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)

Other outcomes

Total Weight of Stools
The total weight of stools in grams (g) is the total weight obtained during the stool collection period regardless of the number of stools that had been collected during this same collection period. Mean total weight of stools 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.
Time frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Percentage of Days With Abdominal Pain and Excessive Flatulence
Mean percentage of days with abdominal complaints during baseline phase (BP) and the 5-day collection period of the treatment phase for total patients was summarized. Abdominal complaints were defined as the reporting of abdominal pain and/or unusual and excessive flatulence/gas production. Mean number of days abdominal pain (AP) and excessive flatulence (EF) 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.
Time frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)

Results

Posted Feb 19, 2015

Participant flow

The enrollment started in April 2009 and was completed in November 2009. Cystic fibrosis (CF) patients with pancreatic insufficiency (PI), aged 2 to 6 years old inclusively, were enrolled from 14 CF centers located in United States of America.

Baseline Phase-Usual Pancreatic Enzymes

Participant flow — Baseline Phase-Usual Pancreatic Enzymes
Milestone	Ultrase® MT12
Started	49
Completed	48
Not completed	1
Withdrew: Protocol violation	1

Treatment Phase With Ultrase® MT12

Participant flow — Treatment Phase With Ultrase® MT12
Milestone	Ultrase® MT12
Started	48
Completed	45
Not completed	3
Withdrew: Adverse event	1
Withdrew: Protocol violation	2

Outcome measures

PrimaryPercentage 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.

Time frame:: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Reported as:: Number · percentage of patients

Percentage of Patients With Control of Steatorrhea
percentage of patients	Ultrase® MT12
Baseline phase (usual pancreatic enzymes)	46.9 (32.5 to 61.7)
Treatment phase	42.9 (28.8 to 57.8)

SecondaryPercentage 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.

Time frame:: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Reported as:: Number · percentage of patients

Percentage of Patients With Normal Stool Frequency
percentage of patients	Ultrase® MT12
Baseline phase (usual pancreatic enzymes): n=49	87.8 (75.2 to 95.4)
Treatment phase: n=45	91.1 (78.8 to 97.5)

SecondaryPercentage 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.

Time frame:: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Reported as:: Mean · percentage of stools

Percentage of Stools With Normal Consistency
percentage of stools	Ultrase® MT12
Baseline phase (usual pancreatic enzymes): n=49	77.38 ± 24.274
Treatment phase: n=45	76.03 ± 23.161

SecondaryPercentage 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.

Time frame:: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Reported as:: Mean · percentage of stools

Percentage of Stools With Abnormal Characteristics
percentage of stools	Ultrase® MT12
Baseline phase (usual pancreatic enzymes): n=49	70.37 ± 30.037
Treatment phase: n=45	61.13 ± 33.582

SecondaryMean 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.

Time frame:: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Reported as:: Mean · days

Mean Number of Days Without Abdominal Complaints
days	Ultrase® MT12
Baseline phase (usual pancreatic enzymes): n=49	2.0 ± 1.89
Treatment phase: n=45	2.3 ± 2.08

Other pre-specifiedTotal Weight of Stools

The total weight of stools in grams (g) is the total weight obtained during the stool collection period regardless of the number of stools that had been collected during this same collection period. Mean total weight of stools 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.

Time frame:: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Reported as:: Mean · gram (g)

Total Weight of Stools
gram (g)	Ultrase® MT12
Baseline phase (usual pancreatic enzymes): n=48	349.2 ± 144.40
Treatment phase: n=45	367.0 ± 155.85

Other pre-specifiedPercentage of Days With Abdominal Pain and Excessive Flatulence

Mean percentage of days with abdominal complaints during baseline phase (BP) and the 5-day collection period of the treatment phase for total patients was summarized. Abdominal complaints were defined as the reporting of abdominal pain and/or unusual and excessive flatulence/gas production. Mean number of days abdominal pain (AP) and excessive flatulence (EF) 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.

Time frame:: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Reported as:: Mean · percentage of days

Percentage of Days With Abdominal Pain and Excessive Flatulence
percentage of days	Ultrase® MT12
BP (usual pancreatic enzymes): AP (n=48)	12.50 ± 27.250
Treatment phase: AP (n=44)	9.20 ± 20.056
BP (usual pancreatic enzymes): EF (n=49)	52.04 ± 37.582
Treatment phase: EF (n=45)	45.56 ± 42.080

Post-hocPercentage of Patients With Control of Steatorrhea Based on Concomitant Use of Proton Pump Inhibitors (PPIs)

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 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.

Time frame:: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Reported as:: Number · percentage of patients

Percentage of Patients With Control of Steatorrhea Based on Concomitant Use of Proton Pump Inhibitors (PPIs)
percentage of patients	Ultrase® MT12
BP (usual pancreatic enzymes): With PPIs (n=28)	53.6 (33.9 to 72.5)
Treatment phase: With PPIs (n=26)	53.8 (33.4 to 73.4)
BP (usual pancreatic enzymes): Without PPIs(n=20)	40.0 (19.1 to 64.0)
Treatment phase: Without PPIs (n=19)	36.8 (16.3 to 61.6)

Adverse events

Collected over From signing of informed consent up to the study discharge (last study visit on Day 24 of treatment phase or early discontinuation visit). Non-serious events are listed at a 4% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Ultrase® MT12	—	0/48 (0%)	24/48 (50%)

Most frequent other events

Showing 10 of 11

Most frequent other events
Event	Ultrase® MT12
CoughRespiratory, thoracic and mediastinal disorders	5/48
VomitingGastrointestinal disorders	4/48
PyrexiaGeneral disorders	4/48
Nasal congestionRespiratory, thoracic and mediastinal disorders	4/48
ConstipationGastrointestinal disorders	3/48
Abdominal painGastrointestinal disorders	2/48
Abdominal pain upperGastrointestinal disorders	2/48
DiarrhoeaGastrointestinal disorders	2/48
Feacal fat increasedInvestigations	2/48
Decreased appetiteMetabolism and nutrition disorders	2/48

Baseline characteristics

Safety population included patients who signed an informed consent form (ICF), completed the baseline phase and started the treatment phase by taking at least one dose of the study treatment.

Age, Categorical
Age, Categorical(Participants)	Ultrase® MT12
<=18 years	48
Between 18 and 65 years	0
>=65 years	0

Age, Continuous
Age, Continuous(years)	Ultrase® MT12
Mean	3.8 ± 1.42

Sex: Female, Male
Sex: Female, Male(Participants)	Ultrase® MT12
Female	19
Male	29

Study locations

15 sites

The Children's Hospital
Aurora, Colorado 80045, United States
University of Michigan Health System Cystic Fibrosis Center
Ann Arbor, Michigan 48109-0212, United States
Helen DeVos Children's Hospital-Spectrum Health Research Department
Grand Rapids, Michigan 40503, United States
SUNY Upstate Medical University
Syracuse, New York 13203, United States
Duke University Medical Center
Durham, North Carolina 27710, United States
Rainbow Babies and Children's Hospital - Cystic Fibrosis Center
Cleveland, Ohio 44106, United States
Children's Medical Center of Dayton
Dayton, Ohio 45404, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma 73104, United States
Respiratory Diseases of Children and Adolescents
Oklahoma City, Oklahoma 73112, United States
Pennsylvania State University and the Milton S. Hershey Medical Center
Hershey, Pennsylvania 17033, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania 15213, United States
Sanford Children's Specialty Clinic
Sioux Falls, South Dakota 57117-5039, United States
University of Utah
Salt Lake City, Utah 84108, United States
Virginia Commonwealth University
Richmond, Virginia 23298, United States
UW Hospital and Clinics
Madison, Wisconsin 53792, United States

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Mar 16, 2017, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT00880100
Lead sponsor: Forest Laboratories
Responsible party: Sponsor
First posted: Apr 13, 2009
Start date: Apr 2009
Primary completion: Nov 2009
Completion: Nov 2009
Results posted: Feb 19, 2015
Last update: Mar 16, 2017

Study contacts

Aptalis Medical Information

study director · Forest Laboratories

Oversight

Data monitoring committee: Yes

View the source record on ClinicalTrials.gov ↗

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