Cystic FibrosisPhase 3NCT00408317
Safety and Efficacy Study of ULTRASE® MT20 in Participants With Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (PI)
A Phase 3 study testing Ultrase® MT20 in people with Cystic Fibrosis and Exocrine Pancreatic Insufficiency, run by Forest Laboratories at 4 sites in United States.
Trial summary
The purpose of this study is to assess the safety and efficacy of Ultrase® MT20 compared to placebo for the correction of fat and protein malabsorption in participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI). This study is sponsored by Aptalis Pharma (formerly Axcan). Read more about this study
- Who is it for?
- Ages 7 years and older
- Conditions the study is for
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- What is being tested?
- Ultrase® MT20
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 4 sitesUnited States
- Study dates
- Nov 2006to Apr 2007
- 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participants or their legally authorized representative must understand the nature of the study and sign an informed consent or assent form along with a parental form
- Participants must have a confirmed diagnosis of CF based on 1 or more clinical features consistent with the CF phenotype, and one of the following:
- A genotype with 2 identifiable mutations consistent with CF
- A sweat chloride test greater than 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 must require pancreatic enzyme supplementation
- Participants must be clinically stable as evidenced by medical and medication history, baseline physical examination including vital signs and laboratory analyses
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- Participants must be 7 years and older
- Participants must have an adequate nutritional status based on the following body mass index (BMI):
- Participants 7 to 20 years old must have a BMI greater than or equal to fifth percentile
- Female participants greater than 20 years old must have a BMI greater than or equal to 16
- Male participants greater 20 years old must have a BMI greater than or equal to 16.5
- Participants must be on an optimal clinical dose of pancreatic enzymes (Ultrase® MT18 or MT20 or other pancreatic enzymes preparations including Ultrase® MT12) prior to entry in the study, and must tolerate this medication in the opinion of the investigator
- Participants must be able to swallow capsules and must be able to eat a high fat diet calculated as 2 gram (± 15%) fat per kilogram body weight per day
- Participants must be, in the opinion of the investigator, able and willing to complete this study
- Female participants must be premenarcheal, surgically sterile or postmenopausal for at least 12 consecutive months. Otherwise, the women of childbearing potential (WOCBP) must not be pregnant and must have practiced an acceptable method of contraception for at least one month prior to the study entry
Who cannot join
- Participants with a known contraindication, sensitivity or hypersensitivity to Ultrase 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 not willing to stop the prohibited medications or products at study entry and throughout the study
- Participants who are using narcotics
- Participants who are using bowel stimulants and/or laxatives on a regular basis
- Participants with acute pancreatitis or acute exacerbation of chronic pancreatic disease
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- Participants with an acute pulmonary infection
- 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 known to have 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, 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 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
4 sites in 1 country.
United States4 sites
Rainbow Babies & Children's Hospital
Cleveland, Ohio, 44106
DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Pennsylvania State University, The Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
University of Utah Health Sciences Center
Salt Lake City, Utah, 84112
About this study
Official title: A Multicenter, Randomized, Double-Blind, Crossover Study to Compare the Safety and Efficacy of Ultrase® MT20 to Placebo for the Correction of Steatorrhea in Patients With Cystic Fibrosis (CF)
The purpose of this study is to assess the safety and efficacy of Ultrase® MT20 compared to placebo for the correction of fat and protein malabsorption in participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI). This study is sponsored by Aptalis Pharma (formerly Axcan).
Read the detailed description
This is a Phase III, multicenter, randomized, double-blind, two-period cross-over, placebo-controlled study designed to compare the efficacy and safety of Ultrase® MT20 to placebo in participants with CF and pancreatic insufficiency. The study consists of a screening period (up to 11 days) and two treatment periods (6-7 days). During screening period participants will be treated with open-label Ultrase® MT18 or MT20. Each treatment period will be preceded by a stabilization period (4 days) and the two treatment periods are separated by a break period (3-6 days). A safety follow-up visit will be performed 7-10 days after discharge from the last treatment period.
- Conditions
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- Being tested
- Ultrase® MT20, Placebo
- Keywords
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency, Ultrase® MT20
- Medical subject headings
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ultrase® MT20Drug
Also called Pancreatic enzyme product
Ultrase® MT 20 capsules containing enteric-coated minitablets orally daily at a dose stabilized during the first stabilization period (4 days), as per investigator's discretion, for 6 to 7 days in either first intervention period or second intervention period.
PlaceboDrug
Placebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in either first intervention period or second intervention period.
| Study group | Role | What they receive |
|---|---|---|
| Ultrase® MT20 | Experimental | Not described Receives: Drug: Ultrase® MT20 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
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 which could extend to 96 hours during both intervention periods. Mean CFA percent was calculated for 72-hour/96-hour period during Day 3 to Day 7 in the first and second intervention periods.
Measured: Day 3 to Day 7 in first intervention period and second intervention period
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 which could extend to 96 hours during both intervention periods. Nitrogen intake was calculated as protein intake/6.25. Mean percent CNA was calculated for 72-hour/96-hour period during Day 3 to Day 7 in the first and second intervention periods.
Measured: Day 3 to Day 7 in first intervention period and second intervention period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2006 (estimated)
- Study startNov 2006
- Primary completionApr 2007
- Study completionApr 2007
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
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 36 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
- NCT00408317
- Sponsor's protocol ID
- UMT20CF05-01
- Status last verified
- Feb 2017
- First submitted
- 4 Dec 2006
- 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
- A Multicenter, Randomized, Double-Blind, Crossover Study to Compare the Safety and Efficacy of Ultrase® MT20 to Placebo for the Correction of Steatorrhea in Patients With Cystic Fibrosis (CF)
- ClinicalTrials.gov ID
- NCT00408317
- Acronym / sponsor ID
- UMT20CF05-01
- Conditions
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- Keywords
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency, Ultrase® MT20
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Forest LaboratoriesIndustry (pharma, biotech, devices)
- Study start
- Nov 2006
- Primary completion
- Apr 2007 (actual)
- Study completion
- Apr 2007 (actual)
- First posted
- 6 Dec 2006 (estimated)
- Results
- Posted 12 Mar 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the safety and efficacy of Ultrase® MT20 compared to placebo for the correction of fat and protein malabsorption in participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI). This study is sponsored by Aptalis Pharma (formerly Axcan).
Detailed description
This is a Phase III, multicenter, randomized, double-blind, two-period cross-over, placebo-controlled study designed to compare the efficacy and safety of Ultrase® MT20 to placebo in participants with CF and pancreatic insufficiency. The study consists of a screening period (up to 11 days) and two treatment periods (6-7 days). During screening period participants will be treated with open-label Ultrase® MT18 or MT20. Each treatment period will be preceded by a stabilization period (4 days) and the two treatment periods are separated by a break period (3-6 days). A safety follow-up visit will be performed 7-10 days after discharge from the last treatment period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ultrase® MT20 | Experimental | Drug: Ultrase® MT20 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 7 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (15 listed)
- Participants or their legally authorized representative must understand the nature of the study and sign an informed consent or assent form along with a parental form
- Participants must have a confirmed diagnosis of CF based on 1 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 must require pancreatic enzyme supplementation
- Participants must be clinically stable as evidenced by medical and medication history, baseline physical examination including vital signs and laboratory analyses
- Participants must be 7 years and older
Key exclusion criteria (18 listed)
- Participants with a known contraindication, sensitivity or hypersensitivity to Ultrase 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 not willing to stop the prohibited medications or products at study entry and throughout the study
- Participants who are using narcotics
- Participants who are using bowel stimulants and/or laxatives on a regular basis
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® MT20Also: Pancreatic enzyme productUltrase® MT 20 capsules containing enteric-coated minitablets orally daily at a dose stabilized during the first stabilization period (4 days), as per investigator's discretion, for 6 to 7 days in either first intervention period or second intervention period. | Drug | Ultrase® MT20 |
| PlaceboPlacebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in either first intervention period or second intervention period. | Drug | Placebo |
Primary outcome
Percent Coefficient of Fat Absorption (CFA)
Time frame: Day 3 to Day 7 in first intervention period and second intervention period
Percent (%) CFA was calculated as ([fat intake - fat excretion]/fat intake)*100, determined by the stools collected during the 72-hour period which could extend to 96 hours during both intervention periods. Mean CFA percent was calculated for 72-hour/96-hour period during Day 3 to Day 7 in the first and second intervention periods.
1 secondary outcome
Percent Coefficient of Nitrogen Absorption (CNA)
Time frame: Day 3 to Day 7 in first intervention period and second intervention period
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
4 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 Exocrine Pancreatic Insufficiency, run by Forest Laboratories at 4 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 15 inclusion and 18 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 4 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. Some participants in this study may receive a placebo. 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 12 Mar 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 7 in first intervention period and second intervention period). 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.