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Safety and Efficacy Study of ULTRASE® MT20 in Participants With Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (PI)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00408317
Recruitment Status : Completed
First Posted : December 6, 2006
Results First Posted : March 12, 2014
Last Update Posted : March 16, 2017
Sponsor:
Information provided by (Responsible Party):
Forest Laboratories

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Conditions Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Interventions Drug: Ultrase® MT20
Drug: Placebo
Enrollment 36
Recruitment Details Participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI) were recruited from centers with CF specialists.
Pre-assignment Details Out of 36 participants who entered the screening period (11 days), and treated with Ultrase® MT20, 31 were randomized to first intervention period.
Arm/Group Title Ultrase® MT20 First, Then Placebo Placebo First, Then Ultrase®MT20
Hide Arm/Group Description 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 the first intervention period followed by placebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in the second intervention period. Break period of 3 to 6 days and fixed dose second stabilization period of 4 days was maintained after first intervention period. Placebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in the first intervention period followed by 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 the second intervention period. Break period of 3 to 6 days and fixed dose second stabilization period of 4 days was maintained after first intervention period.
Period Title: First Intervention Period
Started 14 17
Completed 14 16
Not Completed 0 1
Reason Not Completed
Adverse Event             0             1
Period Title: Second Intervention Period
Started 14 16
Completed 12 14
Not Completed 2 2
Reason Not Completed
Adverse Event             2             0
Protocol Violation             0             1
Withdrawal of consent             0             1
Arm/Group Title Entire Study Population
Hide Arm/Group Description Includes all participants who received Ultrase® MT20 first and placebo first.
Overall Number of Baseline Participants 31
Hide Baseline Analysis Population Description
Intent-to-treat (ITT) population included all randomized participants.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 31 participants
19.6  (6.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants
Female
11
  35.5%
Male
20
  64.5%
1.Primary Outcome
Title Percent Coefficient of Fat Absorption (CFA)
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all randomized participants. Here, 'N' (number of participants analyzed) signifies those participants who were evaluated for this outcome measure.
Arm/Group Title Ultrase® MT20 Placebo
Hide Arm/Group Description:
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.
Placebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in either first intervention period or second intervention period.
Overall Number of Participants Analyzed 25 27
Mean (Standard Deviation)
Unit of Measure: Percent CFA
88.55  (4.94) 55.61  (25.10)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ultrase® MT20, Placebo
Comments P-values are from a semi-parametric mixed model on ranked CFA% values including sequence, period, and treatment group as fixed effects; participant identification (ID) as random effect.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 34.74
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Percent Coefficient of Nitrogen Absorption (CNA)
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all randomized participants. Here, 'N' (number of participants analyzed) signifies those participants who were evaluated for this outcome measure.
Arm/Group Title Ultrase® MT20 Placebo
Hide Arm/Group Description:
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.
Placebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in either first intervention period or second intervention period.
Overall Number of Participants Analyzed 25 27
Mean (Standard Deviation)
Unit of Measure: Percent CNA
84.05  (7.24) 58.78  (20.57)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ultrase® MT20, Placebo
Comments P-values are from a semi-parametric mixed model on ranked CNA% values including sequence, period, and treatment group as fixed effects, and participant ID as random effect.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 25.68
Estimation Comments [Not Specified]
3.Other Pre-specified Outcome
Title Number of Bowel Movements
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all randomized participants. Here, 'N' (number of participants analyzed) signifies those participants who were evaluated for this outcome measure.
Arm/Group Title Ultrase® MT20 Placebo
Hide Arm/Group Description:
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.
Placebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in either first intervention period or second intervention period.
Overall Number of Participants Analyzed 29 30
Mean (Standard Deviation)
Unit of Measure: bowel movements
1.5  (1.0) 3.1  (1.8)
4.Other Pre-specified Outcome
Title Percentage of Stool Categorized by Consistency
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all randomized participants. Here, 'N' (number of participants analyzed) signifies those participants who were evaluated for this outcome measure.
Arm/Group Title Ultrase® MT20 Placebo
Hide Arm/Group Description:
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.
Placebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in either first intervention period or second intervention period.
Overall Number of Participants Analyzed 28 28
Mean (Standard Deviation)
Unit of Measure: percentage of stools
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)
Time Frame Day 1 of first stabilization period up to Day 7 of second intervention period
Adverse Event Reporting Description Adverse event (AE) was defined as any untoward medical occurrence regardless of causal relationship to study medication. Serious adverse event (SAE) was any event that resulted in death, life threatening, required or prolonged in-patient hospitalization, significant disability/incapacity,or was a congenital anomaly/birth defect.
 
Arm/Group Title Ultrase® MT20 Placebo
Hide Arm/Group Description 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. Placebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in either first intervention period or second intervention period.
All-Cause Mortality
Ultrase® MT20 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Hide Serious Adverse Events
Ultrase® MT20 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/30 (0.00%)   1/31 (3.23%) 
General disorders     
General physical health deterioration * 1 [1]  0/30 (0.00%)  1/31 (3.23%) 
Pyrexia * 1  0/30 (0.00%)  1/31 (3.23%) 
Investigations     
Breath sounds abnormal * 1  0/30 (0.00%)  1/31 (3.23%) 
Pulmonary function test decreased * 1  0/30 (0.00%)  1/31 (3.23%) 
Respiratory, thoracic and mediastinal disorders     
Cough * 1  0/30 (0.00%)  1/31 (3.23%) 
Dyspnoea * 1  0/30 (0.00%)  1/31 (3.23%) 
Productive cough * 1  0/30 (0.00%)  1/31 (3.23%) 
Rales * 1  0/30 (0.00%)  1/31 (3.23%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA 9.0
[1]
All SAEs reported in this table were unrelated to study drug and all occurred in the same subject.
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Ultrase® MT20 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   17/30 (56.67%)   27/31 (87.10%) 
Gastrointestinal disorders     
Abdominal pain * 1  2/30 (6.67%)  12/31 (38.71%) 
Abdominal pain upper * 1  4/30 (13.33%)  6/31 (19.35%) 
Constipation * 1  2/30 (6.67%)  2/31 (6.45%) 
Flatulence * 1  1/30 (3.33%)  5/31 (16.13%) 
Abdominal tenderness * 1  0/30 (0.00%)  2/31 (6.45%) 
Diarrhoea * 1  0/30 (0.00%)  3/31 (9.68%) 
Nausea * 1  0/30 (0.00%)  5/31 (16.13%) 
Vomiting * 1  0/30 (0.00%)  3/31 (9.68%) 
Investigations     
Alaninie aminotransferase increased * 1  1/30 (3.33%)  2/31 (6.45%) 
Fecal fat increased * 1  4/30 (13.33%)  9/31 (29.03%) 
Weight decreased * 1  0/30 (0.00%)  3/31 (9.68%) 
Blood glucose increased * 1  0/30 (0.00%)  2/31 (6.45%) 
Laboratory test abnormal * 1  3/30 (10.00%)  7/31 (22.58%) 
Metabolism and nutrition disorders     
Decreased appetite * 1  0/30 (0.00%)  2/31 (6.45%) 
Nervous system disorders     
Headache * 1  2/30 (6.67%)  1/31 (3.23%) 
Respiratory, thoracic and mediastinal disorders     
Pharyngolaryngeal pain * 1  2/30 (6.67%)  1/31 (3.23%) 
Rales * 1  2/30 (6.67%)  1/31 (3.23%) 
Rhinorrhoea * 1  1/30 (3.33%)  3/31 (9.68%) 
Vascular disorders     
Epistaxis * 1  2/30 (6.67%)  0/31 (0.00%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA 9.0
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Restrictions may vary in accordance with each agreement that is negotiated with individual investigators. Sponsor will allow publication after multi-center publication has been published or after an agreed period of time if no such multi-center publication is submitted for publication. Sponsor can ask that Sponsor’s confidential information be removed from any publication and defer publication for period of time to allow Sponsor to obtain patent or other intellectual property right protection.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Robert Winkler, MD, VP, Clinical Development and Operations
Organization: Aptalis Pharma US, Inc.
Phone: 1-800-472-2634
Layout table for additonal information
Responsible Party: Forest Laboratories
ClinicalTrials.gov Identifier: NCT00408317    
Other Study ID Numbers: UMT20CF05-01
First Submitted: December 4, 2006
First Posted: December 6, 2006
Results First Submitted: January 27, 2014
Results First Posted: March 12, 2014
Last Update Posted: March 16, 2017