A Phase 3 interventional study of Ultrase® MT20 and Placebo in Cystic Fibrosis and Exocrine Pancreatic Insufficiency, sponsored by Forest Laboratories. Completed at 4 sites in United States. Open to participants aged 7 Years and older. Per ClinicalTrials.gov, last updated 2017-03-16.
Sponsored by Forest Laboratories · Phase 3, Interventional, and Treatment
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).
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.
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's enrollment of 36 is close to the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →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.
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 an adequate nutritional status based on the following body mass index (BMI):
Exclusion Criteria:
Drug: Ultrase® MT20
Drug: Placebo
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.
Also known as: Pancreatic enzyme product
Placebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in either first intervention period or second intervention period.
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.
Time frame: Day 3 to Day 7 in first intervention period and second intervention period
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.
Time frame: Day 3 to Day 7 in first intervention period and second intervention period
Number of Bowel Movements
Number of bowel movements of each participant was calculated from frequency of stools by the participant per day. Mean daily number of bowel movements on Day 3 for the first treatment period and second treatment period was summarized.
Time frame: Day 3 on first intervention period and second intervention period
Percentage of Stool Categorized by Consistency
Stool consistency was categorized as hard, formed/normal, soft or watery stool. Percentage of stools of a specific consistency of each participant was calculated as the number of stools with a specific consistency relative to the total number of stools during the collection period. Mean percentage of stool with specific consistency on Day 4 for the first treatment period and second treatment period period for total participants was summarized.
Time frame: Day 4 on first intervention period and second intervention period
Participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI) were recruited from centers with CF specialists.
| Milestone | Ultrase® MT20 First, Then Placebo | Placebo First, Then Ultrase®MT20 |
|---|---|---|
| Started | 14 | 17 |
| Completed | 14 | 16 |
| Not completed | 0 | 1 |
| Withdrew: Adverse event | 0 | 1 |
| Milestone | Ultrase® MT20 First, Then Placebo | Placebo First, Then Ultrase®MT20 |
|---|---|---|
| Started | 14 | 16 |
| Completed | 12 | 14 |
| Not completed | 2 | 2 |
| Withdrew: Adverse event | 2 | 0 |
| Withdrew: Protocol violation | 0 | 1 |
| Withdrew: Withdrawal of consent | 0 | 1 |
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.
| Percent CFA | Ultrase® MT20 | Placebo |
|---|---|---|
| Percent Coefficient of Fat Absorption (CFA) | 88.55 ± 4.94 | 55.61 ± 25.10 |
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.
| Percent CNA | Ultrase® MT20 | Placebo |
|---|---|---|
| Percent Coefficient of Nitrogen Absorption (CNA) | 84.05 ± 7.24 | 58.78 ± 20.57 |
Number of bowel movements of each participant was calculated from frequency of stools by the participant per day. Mean daily number of bowel movements on Day 3 for the first treatment period and second treatment period was summarized.
| bowel movements | Ultrase® MT20 | Placebo |
|---|---|---|
| Number of Bowel Movements | 1.5 ± 1.0 | 3.1 ± 1.8 |
Stool consistency was categorized as hard, formed/normal, soft or watery stool. Percentage of stools of a specific consistency of each participant was calculated as the number of stools with a specific consistency relative to the total number of stools during the collection period. Mean percentage of stool with specific consistency on Day 4 for the first treatment period and second treatment period period for total participants was summarized.
| percentage of stools | Ultrase® MT20 | Placebo |
|---|---|---|
| Hard Stools | 11.31 ± 27.98 | 3.57 ± 18.90 |
| Formed/Normal Stools | 76.19 ± 39.13 | 25.71 ± 43.84 |
| Soft Stools | 12.50 ± 29.27 | 66.73 ± 45.08 |
| Watery Stools | 0 ± 0 | 3.99 ± 13.65 |
Collected over Day 1 of first stabilization period up to Day 7 of second intervention period. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Ultrase® MT20 | — | 0/30 (0%) | 17/30 (56.7%) |
| Placebo | — | 1/31 (3.2%) | 27/31 (87.1%) |
| Event | Ultrase® MT20 | Placebo |
|---|---|---|
| General physical health deteriorationGeneral disorders | 0/30 | 1/31 |
| PyrexiaGeneral disorders | 0/30 | 1/31 |
| Breath sounds abnormalInvestigations | 0/30 | 1/31 |
| Pulmonary function test decreasedInvestigations | 0/30 | 1/31 |
| CoughRespiratory, thoracic and mediastinal disorders | 0/30 | 1/31 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 0/30 | 1/31 |
| Productive coughRespiratory, thoracic and mediastinal disorders | 0/30 | 1/31 |
| RalesRespiratory, thoracic and mediastinal disorders | 0/30 | 1/31 |
| Event | Ultrase® MT20 | Placebo |
|---|---|---|
| Abdominal painGastrointestinal disorders | 2/30 | 12/31 |
| Fecal fat increasedInvestigations | 4/30 | 9/31 |
| Laboratory test abnormalInvestigations | 3/30 | 7/31 |
| Abdominal pain upperGastrointestinal disorders | 4/30 | 6/31 |
| FlatulenceGastrointestinal disorders | 1/30 | 5/31 |
| NauseaGastrointestinal disorders | 0/30 | 5/31 |
| Weight decreasedInvestigations | 0/30 | 3/31 |
| DiarrhoeaGastrointestinal disorders | 0/30 | 3/31 |
| VomitingGastrointestinal disorders | 0/30 | 3/31 |
| RhinorrhoeaRespiratory, thoracic and mediastinal disorders | 1/30 | 3/31 |
Intent-to-treat (ITT) population included all randomized participants.
| Age, Continuous(years) | Entire Study Population |
|---|---|
| Mean | 19.6 ± 6.6 |
| Sex: Female, Male(Participants) | Entire Study Population |
|---|---|
| Female | 11 |
| Male | 20 |
This study is completed, as verified in Feb 2017. You cannot join it, but the record below documents what was studied.
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