Trial Outcomes & Findings for Special Investigation (All Cases) of LipaCreon in Patients With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis (NCT NCT01427712)
NCT ID: NCT01427712
Last Updated: 2022-06-10
Results Overview
An adverse event (AE) was defined as any unfavourable or unintended disease, or symptom or sign of such a disease, or abnormal laboratory finding that occurred in a patient who received Lipacreon, whether or not considered related to the medicinal product. Also, an AE for which the relationship with Lipacreon could not be ruled out was regarded as an adverse drug reaction (ADR). 1. Related : There is a temporal relationship between the use of the medicinal product and the onset of an AE, or a relapse with readministration,where other factors are less likely to be involved. 2. Relationship cannot be ruled out : There are other potential factors although there is a temporal relationship between the use of the medicinal product and the onset of an AE
COMPLETED
24 participants
From the start of Lipacreon treatment to the end of the observation period or discontinuation. Approximately 7 years in the study completers (From date of dosage start, up to 7 years).
2022-06-10
Participant Flow
For this survey, the contract for the conduct of the survey was concluded with 21 departments of 21 facilities during the period from August 2011 to March 2018. Among these, 24 patients were registered at 21 departments of 21 facilities (patient registration period: August 2011 to March 2017).
Participant milestones
| Measure |
LipaCreon
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Overall Study
STARTED
|
24
|
|
Overall Study
Collected CRFs
|
21
|
|
Overall Study
Safety Analysis Set
|
21
|
|
Overall Study
COMPLETED
|
17
|
|
Overall Study
NOT COMPLETED
|
7
|
Reasons for withdrawal
| Measure |
LipaCreon
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Overall Study
Transfer to a different hospital
|
2
|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
Aggravation of cystic fibrosis
|
3
|
|
Overall Study
Failure to visit
|
1
|
Baseline Characteristics
Special Investigation (All Cases) of LipaCreon in Patients With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Age, Continuous
|
8.7 years
STANDARD_DEVIATION 9.4 • n=5 Participants
|
|
Age, Customized
< 6 years
|
12 Participants
n=5 Participants
|
|
Age, Customized
>= 6 years and < 12 years
|
4 Participants
n=5 Participants
|
|
Age, Customized
>= 12 years and < 18 years
|
0 Participants
n=5 Participants
|
|
Age, Customized
>= 18 years
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
21 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Japan
|
21 participants
n=5 Participants
|
|
Body Mass Index (BMI)
< 18.5 kg/m^2
|
16 Participants
n=5 Participants
|
|
Body Mass Index (BMI)
>= 18.5 kg/m^2 and < 25 kg/m^2
|
2 Participants
n=5 Participants
|
|
Body Mass Index (BMI)
>= 25 kg/m^2
|
0 Participants
n=5 Participants
|
|
Body Mass Index (BMI)
unknown
|
3 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From the start of Lipacreon treatment to the end of the observation period or discontinuation. Approximately 7 years in the study completers (From date of dosage start, up to 7 years).Population: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients.
An adverse event (AE) was defined as any unfavourable or unintended disease, or symptom or sign of such a disease, or abnormal laboratory finding that occurred in a patient who received Lipacreon, whether or not considered related to the medicinal product. Also, an AE for which the relationship with Lipacreon could not be ruled out was regarded as an adverse drug reaction (ADR). 1. Related : There is a temporal relationship between the use of the medicinal product and the onset of an AE, or a relapse with readministration,where other factors are less likely to be involved. 2. Relationship cannot be ruled out : There are other potential factors although there is a temporal relationship between the use of the medicinal product and the onset of an AE
Outcome measures
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Number of Patients With Adverse Drug Reaction
|
1 participants
|
SECONDARY outcome
Timeframe: Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 yearsPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients.
The following nutritional endpoints were measured prior to the start of Lipacreon treatment, at 4, 8, and 24 weeks after the start of treatment, at the completion of the 52-week observation period, at 1-year intervals during the follow-up period, and at the completion of follow-up in March 2018. •BMI (height \[only prior to the start of Lipacreon treatment\] and weight)
Outcome measures
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Nutritional Endpoints - BMI
Baseline
|
15.093 kg/m^2
Standard Deviation 1.955
|
|
Nutritional Endpoints - BMI
4 weeks
|
15.583 kg/m^2
Standard Deviation 1.511
|
|
Nutritional Endpoints - BMI
8 weeks
|
15.649 kg/m^2
Standard Deviation 1.667
|
|
Nutritional Endpoints - BMI
24 weeks
|
15.604 kg/m^2
Standard Deviation 1.350
|
|
Nutritional Endpoints - BMI
52 weeks
|
15.698 kg/m^2
Standard Deviation 1.239
|
|
Nutritional Endpoints - BMI
2 years
|
15.766 kg/m^2
Standard Deviation 1.438
|
|
Nutritional Endpoints - BMI
3 years
|
15.152 kg/m^2
Standard Deviation 1.241
|
|
Nutritional Endpoints - BMI
4 years
|
15.479 kg/m^2
Standard Deviation 1.757
|
|
Nutritional Endpoints - BMI
5 years
|
15.584 kg/m^2
Standard Deviation 2.608
|
|
Nutritional Endpoints - BMI
6 years
|
15.819 kg/m^2
Standard Deviation 2.467
|
|
Nutritional Endpoints - BMI
7 years
|
11.806 kg/m^2
|
SECONDARY outcome
Timeframe: Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 yearsPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients.
The following nutritional endpoints were measured prior to the start of Lipacreon treatment, at 4, 8, and 24 weeks after the start of treatment, at the completion of the 52-week observation period, at 1-year intervals during the follow-up period, and at the completion of follow-up in March 2018. •Serum total protein
Outcome measures
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Nutritional Endpoints - Serum Total Protein
Baseline
|
7.282 g/dL
Standard Deviation 1.105
|
|
Nutritional Endpoints - Serum Total Protein
4 weeks
|
6.925 g/dL
Standard Deviation 0.770
|
|
Nutritional Endpoints - Serum Total Protein
8 weeks
|
6.700 g/dL
Standard Deviation 0.441
|
|
Nutritional Endpoints - Serum Total Protein
24 weeks
|
7.350 g/dL
Standard Deviation 1.049
|
|
Nutritional Endpoints - Serum Total Protein
52 weeks
|
7.421 g/dL
Standard Deviation 0.877
|
|
Nutritional Endpoints - Serum Total Protein
2 years
|
7.563 g/dL
Standard Deviation 1.058
|
|
Nutritional Endpoints - Serum Total Protein
3 years
|
8.029 g/dL
Standard Deviation 1.164
|
|
Nutritional Endpoints - Serum Total Protein
4 years
|
8.127 g/dL
Standard Deviation 1.301
|
|
Nutritional Endpoints - Serum Total Protein
5 years
|
7.390 g/dL
Standard Deviation 1.086
|
|
Nutritional Endpoints - Serum Total Protein
6 years
|
7.200 g/dL
Standard Deviation 0.557
|
|
Nutritional Endpoints - Serum Total Protein
7 years
|
6.100 g/dL
|
SECONDARY outcome
Timeframe: Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 yearsPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients.
The following nutritional endpoints were measured prior to the start of Lipacreon treatment, at 4, 8, and 24 weeks after the start of treatment, at the completion of the 52-week observation period, at 1-year intervals during the follow-up period, and at the completion of follow-up in March 2018. •Albumin
Outcome measures
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Nutrition Endpoints - Albumin
24 weeks
|
4.130 g/dL
Standard Deviation 0.267
|
|
Nutrition Endpoints - Albumin
Baseline
|
3.872 g/dL
Standard Deviation 0.641
|
|
Nutrition Endpoints - Albumin
4 weeks
|
4.054 g/dL
Standard Deviation 0.657
|
|
Nutrition Endpoints - Albumin
8 weeks
|
4.067 g/dL
Standard Deviation 0.339
|
|
Nutrition Endpoints - Albumin
52 weeks
|
4.232 g/dL
Standard Deviation 0.549
|
|
Nutrition Endpoints - Albumin
2 years
|
4.390 g/dL
Standard Deviation 0.304
|
|
Nutrition Endpoints - Albumin
3 years
|
4.054 g/dL
Standard Deviation 0.323
|
|
Nutrition Endpoints - Albumin
4 years
|
4.040 g/dL
Standard Deviation 0.844
|
|
Nutrition Endpoints - Albumin
5 years
|
3.613 g/dL
Standard Deviation 0.931
|
|
Nutrition Endpoints - Albumin
6 years
|
4.233 g/dL
Standard Deviation 0.115
|
|
Nutrition Endpoints - Albumin
7 years
|
3.700 g/dL
|
SECONDARY outcome
Timeframe: Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 yearsPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients.
The following nutritional endpoints were measured prior to the start of Lipacreon treatment, at 4, 8, and 24 weeks after the start of treatment, at the completion of the 52-week observation period, at 1-year intervals during the follow-up period, and at the completion of follow-up in March 2018. •Total cholesterol
Outcome measures
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Nutrition Endpoints - Total Cholesterol
Baseline
|
124.94 mg/dL
Standard Deviation 31.04
|
|
Nutrition Endpoints - Total Cholesterol
4 weeks
|
126.10 mg/dL
Standard Deviation 34.94
|
|
Nutrition Endpoints - Total Cholesterol
8 weeks
|
128.00 mg/dL
Standard Deviation 35.96
|
|
Nutrition Endpoints - Total Cholesterol
24 weeks
|
139.13 mg/dL
Standard Deviation 40.88
|
|
Nutrition Endpoints - Total Cholesterol
52 weeks
|
123.11 mg/dL
Standard Deviation 29.40
|
|
Nutrition Endpoints - Total Cholesterol
2 years
|
148.17 mg/dL
Standard Deviation 26.12
|
|
Nutrition Endpoints - Total Cholesterol
3 years
|
139.00 mg/dL
Standard Deviation 39.72
|
|
Nutrition Endpoints - Total Cholesterol
4 years
|
136.50 mg/dL
Standard Deviation 30.88
|
|
Nutrition Endpoints - Total Cholesterol
5 years
|
114.00 mg/dL
Standard Deviation 62.23
|
|
Nutrition Endpoints - Total Cholesterol
6 years
|
157.50 mg/dL
Standard Deviation 57.28
|
|
Nutrition Endpoints - Total Cholesterol
7 years
|
167.00 mg/dL
|
SECONDARY outcome
Timeframe: Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 yearsPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients.
The following nutritional endpoints were measured prior to the start of Lipacreon treatment, at 4, 8, and 24 weeks after the start of treatment, at the completion of the 52-week observation period, at 1-year intervals during the follow-up period, and at the completion of follow-up in March 2018. •Triglycerides
Outcome measures
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Nutrition Endpoints - Triglycerides
Baseline
|
81.6 mg/dL
Standard Deviation 41.4
|
|
Nutrition Endpoints - Triglycerides
4 weeks
|
126.1 mg/dL
Standard Deviation 115.2
|
|
Nutrition Endpoints - Triglycerides
8 weeks
|
73.2 mg/dL
Standard Deviation 35.4
|
|
Nutrition Endpoints - Triglycerides
24 weeks
|
80.5 mg/dL
Standard Deviation 39.6
|
|
Nutrition Endpoints - Triglycerides
52 weeks
|
117.1 mg/dL
Standard Deviation 51.1
|
|
Nutrition Endpoints - Triglycerides
2 years
|
83.6 mg/dL
Standard Deviation 69.9
|
|
Nutrition Endpoints - Triglycerides
3 years
|
89.8 mg/dL
Standard Deviation 34.8
|
|
Nutrition Endpoints - Triglycerides
4 years
|
100.0 mg/dL
Standard Deviation 49.2
|
|
Nutrition Endpoints - Triglycerides
5 years
|
89.0 mg/dL
Standard Deviation 28.3
|
|
Nutrition Endpoints - Triglycerides
6 years
|
124.5 mg/dL
Standard Deviation 38.9
|
|
Nutrition Endpoints - Triglycerides
7 years
|
160.0 mg/dL
|
SECONDARY outcome
Timeframe: Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 yearsPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients.
The following nutritional endpoints were measured prior to the start of Lipacreon treatment, at 4, 8, and 24 weeks after the start of treatment, at the completion of the 52-week observation period, at 1-year intervals during the follow-up period, and at the completion of follow-up in March 2018. •Haemoglobin
Outcome measures
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Nutrition Endpoints - Haemoglobin
Baseline
|
12.02 g/dL
Standard Deviation 1.84
|
|
Nutrition Endpoints - Haemoglobin
4 weeks
|
12.38 g/dL
Standard Deviation 1.65
|
|
Nutrition Endpoints - Haemoglobin
8 weeks
|
12.07 g/dL
Standard Deviation 1.94
|
|
Nutrition Endpoints - Haemoglobin
24 weeks
|
12.82 g/dL
Standard Deviation 1.65
|
|
Nutrition Endpoints - Haemoglobin
52 weeks
|
12.89 g/dL
Standard Deviation 1.88
|
|
Nutrition Endpoints - Haemoglobin
2 years
|
13.41 g/dL
Standard Deviation 1.18
|
|
Nutrition Endpoints - Haemoglobin
3 years
|
13.18 g/dL
Standard Deviation 0.81
|
|
Nutrition Endpoints - Haemoglobin
4 years
|
13.38 g/dL
Standard Deviation 2.25
|
|
Nutrition Endpoints - Haemoglobin
5 years
|
11.65 g/dL
Standard Deviation 2.11
|
|
Nutrition Endpoints - Haemoglobin
6 years
|
12.50 g/dL
Standard Deviation 3.22
|
|
Nutrition Endpoints - Haemoglobin
7 years
|
12.20 g/dL
|
SECONDARY outcome
Timeframe: Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 yearsPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients.
The following symptoms were assessed as those related to exocrine pancreatic insufficiency prior to the start of Lipacreon treatment, at 4, 8, and 24 weeks after the start of treatment, at the completion of the 52-week observation period, at 1-year intervals during the follow-up period, and at the completion of follow-up in March 2018. •Steatorrhoea (Yes/No)
Outcome measures
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
Baseline, Yes
|
14 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
Baseline, No
|
4 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
4 weeks, Yes
|
4 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
4 weeks, No
|
8 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
8 weeks, Yes
|
2 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
8 weeks, No
|
7 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
24 weeks, Yes
|
4 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
24 weeks, No
|
10 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
52 weeks, Yes
|
3 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
52 weeks, No
|
9 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
2 years, Yes
|
3 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
2 years, No
|
7 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
3 years, Yes
|
2 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
3 Years, No
|
6 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
4 years, Yes
|
2 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
4 years, No
|
6 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
5 years, Yes
|
2 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
5 years, No
|
4 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
6 years, Yes
|
2 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
6 years, No
|
2 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
7 years, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea
7 years, No
|
1 participants
|
SECONDARY outcome
Timeframe: Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 yearsPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients.
The following symptoms were assessed as those related to exocrine pancreatic insufficiency prior to the start of Lipacreon treatment, at 4, 8, and 24 weeks after the start of treatment, at the completion of the 52-week observation period, at 1-year intervals during the follow-up period, and at the completion of follow-up in March 2018. •Frequency of bowel movements (times/day)
Outcome measures
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Frequency of Bowel Movements
Baseline
|
2.40 times/day
Standard Deviation 1.76
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Frequency of Bowel Movements
4 weeks
|
1.90 times/day
Standard Deviation 0.74
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Frequency of Bowel Movements
8 weeks
|
1.86 times/day
Standard Deviation 0.69
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Frequency of Bowel Movements
24 weeks
|
1.83 times/day
Standard Deviation 0.83
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Frequency of Bowel Movements
52 weeks
|
1.82 times/day
Standard Deviation 0.75
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Frequency of Bowel Movements
2 years
|
1.75 times/day
Standard Deviation 1.16
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Frequency of Bowel Movements
3 years
|
1.63 times/day
Standard Deviation 0.74
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Frequency of Bowel Movements
4 years
|
2.29 times/day
Standard Deviation 1.11
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Frequency of Bowel Movements
5 years
|
1.60 times/day
Standard Deviation 0.89
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Frequency of Bowel Movements
6 years
|
1.33 times/day
Standard Deviation 0.58
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Frequency of Bowel Movements
7 years
|
1.00 times/day
|
SECONDARY outcome
Timeframe: Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 yearsPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients.
The following symptoms were assessed as those related to exocrine pancreatic insufficiency prior to the start of Lipacreon treatment, at 4, 8, and 24 weeks after the start of treatment, at the completion of the 52-week observation period, at 1-year intervals during the follow-up period, and at the completion of follow-up in March 2018. •Diarrhoea (Yes/No)
Outcome measures
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
8 weeks, No
|
10 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
24 weeks, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
Baseline, Yes
|
6 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
Baseline, No
|
13 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
4 weeks, Yes
|
1 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
4 weeks, No
|
12 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
8 weeks, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
24 weeks, No
|
16 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
52 weeks, Yes
|
1 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
52 weeks, No
|
12 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
2 years, Yes
|
1 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
2 years, No
|
10 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
3 years, Yes
|
1 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
3 Years, No
|
7 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
4 years, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
4 years, No
|
9 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
5 years, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
5 years, No
|
7 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
6 years, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
6 years, No
|
4 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
7 years, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea
7 years, No
|
1 participants
|
SECONDARY outcome
Timeframe: Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 yearsPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients.
The following symptoms were assessed as those related to exocrine pancreatic insufficiency prior to the start of Lipacreon treatment, at 4, 8, and 24 weeks after the start of treatment, at the completion of the 52-week observation period, at 1-year intervals during the follow-up period, and at the completion of follow-up in March 2018. •Foul stool odour (Yes/No)
Outcome measures
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
Baseline, Yes
|
6 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
Baseline, No
|
13 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
4 weeks, Yes
|
1 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
4 weeks, No
|
12 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
8 weeks, Yes
|
2 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
8 weeks, No
|
8 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
24 weeks, Yes
|
2 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
24 weeks, No
|
13 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
52 weeks, Yes
|
3 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
52 weeks, No
|
9 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
2 years, Yes
|
2 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
2 years, No
|
8 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
3 years, Yes
|
3 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
3 Years, No
|
5 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
4 years, Yes
|
1 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
4 years, No
|
7 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
5 years, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
5 years, No
|
7 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
6 years, Yes
|
1 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
6 years, No
|
3 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
7 years, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour
7 years, No
|
1 participants
|
SECONDARY outcome
Timeframe: Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 yearsPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients.
The following symptoms were assessed as those related to exocrine pancreatic insufficiency prior to the start of Lipacreon treatment, at 4, 8, and 24 weeks after the start of treatment, at the completion of the 52-week observation period, at 1-year intervals during the follow-up period, and at the completion of follow-up in March 2018. •Inappetence (Yes/No)
Outcome measures
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
Baseline, Yes
|
7 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
Baseline, No
|
13 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
4 weeks, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
4 weeks, No
|
13 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
8 weeks, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
8 weeks, No
|
10 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
24 weeks, Yes
|
4 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
24 weeks, No
|
12 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
52 weeks, Yes
|
2 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
52 weeks, No
|
11 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
2 years, Yes
|
3 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
2 years, No
|
8 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
3 years, Yes
|
2 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
3 Years, No
|
6 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
4 years, Yes
|
3 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
4 years, No
|
6 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
5 years, Yes
|
3 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
5 years, No
|
4 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
6 years, Yes
|
1 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
6 years, No
|
3 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
7 years, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence
7 years, No
|
1 participants
|
SECONDARY outcome
Timeframe: Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 yearsPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients.
The following symptoms were assessed as those related to exocrine pancreatic insufficiency prior to the start of Lipacreon treatment, at 4, 8, and 24 weeks after the start of treatment, at the completion of the 52-week observation period, at 1-year intervals during the follow-up period, and at the completion of follow-up in March 2018. •Abdominal distension (Yes/No)
Outcome measures
| Measure |
LipaCreon
n=21 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
Baseline, Yes
|
5 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
Baseline, No
|
15 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
4 weeks, Yes
|
1 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
4 weeks, No
|
12 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
8 weeks, Yes
|
2 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
8 weeks, No
|
8 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
24 weeks, Yes
|
2 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
24 weeks, No
|
14 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
52 weeks, Yes
|
1 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
52 weeks, No
|
12 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
2 years, Yes
|
3 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
2 years, No
|
8 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
3 years, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
3 Years, No
|
8 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
4 years, Yes
|
1 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
4 years, No
|
8 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
5 years, Yes
|
3 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
5 years, No
|
4 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
6 years, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
6 years, No
|
4 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
7 years, Yes
|
0 participants
|
|
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension
7 years, No
|
1 participants
|
SECONDARY outcome
Timeframe: At 24 weeksPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients. Of these 21 patients, 17 patients were obtained with data on Degree of General Improvement at 24 weeks.
Degree of general improvement was assessed at 24 weeks after the start of Lipacreon treatment and at the completion of the 52-week observation period, using the following 4 grades: Improved, unchanged, exacerbated, unassessable
Outcome measures
| Measure |
LipaCreon
n=17 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Degree of General Improvement
Improved
|
10 Participants
|
|
Degree of General Improvement
Unchanged
|
7 Participants
|
|
Degree of General Improvement
Exacerbated
|
0 Participants
|
|
Degree of General Improvement
Unassessable
|
0 Participants
|
SECONDARY outcome
Timeframe: At 52 weeksPopulation: Among the 21 patients from whom the survey forms were collected, no patient was excluded from the safety analysis or efficacy analysis, and both the safety analysis set and the efficacy analysis set included 21 patients. Of these 21 patients, 14 patients were obtained with data on Degree of General Improvement at 52 weeks.
Degree of general improvement was assessed at 24 weeks after the start of Lipacreon treatment and at the completion of the 52-week observation period, using the following 4 grades: Improved, unchanged, exacerbated, unassessable
Outcome measures
| Measure |
LipaCreon
n=14 Participants
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Degree of General Improvement
Improved
|
9 Participants
|
|
Degree of General Improvement
Unchanged
|
5 Participants
|
|
Degree of General Improvement
Exacerbated
|
0 Participants
|
|
Degree of General Improvement
Unassessable
|
0 Participants
|
Adverse Events
LipaCreon
Serious adverse events
| Measure |
LipaCreon
n=21 participants at risk
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Infections and infestations
Pharyngitis
|
4.8%
1/21 • Number of events 1 • From the start of Lipacreon treatment to the end of the observation period or discontinuation. Approximately 7 years in the study completers (From date of dosage start, up to 7 years).
|
|
Infections and infestations
Pneumonia
|
4.8%
1/21 • Number of events 1 • From the start of Lipacreon treatment to the end of the observation period or discontinuation. Approximately 7 years in the study completers (From date of dosage start, up to 7 years).
|
|
Infections and infestations
Enteritis infectious
|
4.8%
1/21 • Number of events 2 • From the start of Lipacreon treatment to the end of the observation period or discontinuation. Approximately 7 years in the study completers (From date of dosage start, up to 7 years).
|
|
Metabolism and nutrition disorders
Dehydration
|
4.8%
1/21 • Number of events 1 • From the start of Lipacreon treatment to the end of the observation period or discontinuation. Approximately 7 years in the study completers (From date of dosage start, up to 7 years).
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
4.8%
1/21 • Number of events 1 • From the start of Lipacreon treatment to the end of the observation period or discontinuation. Approximately 7 years in the study completers (From date of dosage start, up to 7 years).
|
|
Gastrointestinal disorders
Haematochezia
|
4.8%
1/21 • Number of events 1 • From the start of Lipacreon treatment to the end of the observation period or discontinuation. Approximately 7 years in the study completers (From date of dosage start, up to 7 years).
|
|
Skin and subcutaneous tissue disorders
Erythema multiforme
|
4.8%
1/21 • Number of events 1 • From the start of Lipacreon treatment to the end of the observation period or discontinuation. Approximately 7 years in the study completers (From date of dosage start, up to 7 years).
|
|
Renal and urinary disorders
Renal impairment
|
4.8%
1/21 • Number of events 1 • From the start of Lipacreon treatment to the end of the observation period or discontinuation. Approximately 7 years in the study completers (From date of dosage start, up to 7 years).
|
|
Investigations
C-Reactive Protein Increased
|
4.8%
1/21 • Number of events 2 • From the start of Lipacreon treatment to the end of the observation period or discontinuation. Approximately 7 years in the study completers (From date of dosage start, up to 7 years).
|
Other adverse events
| Measure |
LipaCreon
n=21 participants at risk
In general, pancrelipase 600 mg/dose was orally administered immediately after a meal, 3 times a day.
Also, the dose was adjusted appropriately according to the patient's condition.
Pancrelipase
|
|---|---|
|
Infections and infestations
Bronchitis
|
9.5%
2/21 • Number of events 6 • From the start of Lipacreon treatment to the end of the observation period or discontinuation. Approximately 7 years in the study completers (From date of dosage start, up to 7 years).
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
9.5%
2/21 • Number of events 6 • From the start of Lipacreon treatment to the end of the observation period or discontinuation. Approximately 7 years in the study completers (From date of dosage start, up to 7 years).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place