Special Investigation (All Cases) of LipaCreon in Patients With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT01427712

Enrollment

Registered

2011-09-02

Start date

2011-08-31

Completion date

2018-03-31

Last updated

2022-06-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis, Digestive System Diseases, Exocrine Pancreatic Insufficiency, Pancreatic Diseases

Keywords

Digestive System Diseases, Cystic Fibrosis, Therapeutic Uses, Gastrointestinal Agents, Pancreatic Diseases, Pancrelipase, Exocrine Pancreatic Insufficiency

Brief summary

This study aims at collecting the information related to the safety and effectiveness in the pancreatic exocrine insufficiency patients due to cystic fibrosis receiving the treatment with LipaCreon in order to evaluate the effective and safe use of LipaCreon.

Interventions

DRUGPancrelipase

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

1 Years to 99 Years

Healthy volunteers

Inclusion criteria

* Patients who receive LipaCreon for the replacement of pancreatic digestive enzymes in pancreatic exocrine insufficiency due to cystic fibrosis

Exclusion criteria

* Patients with a history of hypersensitivity to the ingredient of LipaCreon. * Patients with a history of hypersensitivity to porcine protein

Design outcomes

Primary

Measure	Time frame	Description
Number of Patients With Adverse Drug Reaction	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).	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

Secondary

Measure	Time frame	Description
Nutritional Endpoints - Serum Total Protein	Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 years	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
Nutrition Endpoints - Albumin	Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 years	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
Nutrition Endpoints - Total Cholesterol	Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 years	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
Nutrition Endpoints - Triglycerides	Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 years	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
Nutrition Endpoints - Haemoglobin	Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 years	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
Symptoms Related to Exocrine Pancreatic Insufficiency - Steatorrhoea	Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 years	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)
Nutritional Endpoints - BMI	Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 years	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)
Symptoms Related to Exocrine Pancreatic Insufficiency - Diarrhoea	Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 years	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)
Symptoms Related to Exocrine Pancreatic Insufficiency - Foul Stool Odour	Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 years	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)
Symptoms Related to Exocrine Pancreatic Insufficiency - Inappetence	Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 years	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)
Symptoms Related to Exocrine Pancreatic Insufficiency - Abdominal Distension	Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 years	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)
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
Symptoms Related to Exocrine Pancreatic Insufficiency - Frequency of Bowel Movements	Baseline, 4 weeks, 8 weeks, 24 weeks, 52 weeks, 2 years, 3 years, 4 years, 5 years, 6 years, and 7 years	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)

Countries

Japan

Participant flow

Recruitment details

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).

Participants by arm

Arm	Count
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	21
Total	21

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Aggravation of cystic fibrosis	3
Overall Study	Failure to visit	1
Overall Study	Lost to Follow-up	1
Overall Study	Transfer to a different hospital	2

Baseline characteristics

Characteristic	LipaCreon
Age, Continuous	8.7 years STANDARD_DEVIATION 9.4
Age, Customized >= 12 years and < 18 years	0 Participants
Age, Customized >= 18 years	5 Participants
Age, Customized < 6 years	12 Participants
Age, Customized >= 6 years and < 12 years	4 Participants
Body Mass Index (BMI) < 18.5 kg/m^2	16 Participants
Body Mass Index (BMI) >= 18.5 kg/m^2 and < 25 kg/m^2	2 Participants
Body Mass Index (BMI) >= 25 kg/m^2	0 Participants
Body Mass Index (BMI) unknown	3 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants
Race (NIH/OMB) Asian	21 Participants
Race (NIH/OMB) Black or African American	0 Participants
Race (NIH/OMB) More than one race	0 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants
Race (NIH/OMB) White	0 Participants
Region of Enrollment Japan	21 participants
Sex: Female, Male Female	11 Participants
Sex: Female, Male Male	10 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	3 / 21
other Total, other adverse events	4 / 21
serious Total, serious adverse events	4 / 21

Outcome results

Primary

Number of Patients With Adverse Drug Reaction

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

Time frame: 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).

Arm	Measure	Value (NUMBER)
LipaCreon	Number of Patients With Adverse Drug Reaction	1 participants

Secondary

Degree of General Improvement

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

Time frame: At 52 weeks

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. Of these 21 patients, 14 patients were obtained with data on Degree of General Improvement at 52 weeks.

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
LipaCreon	Degree of General Improvement	Improved	9 Participants
LipaCreon	Degree of General Improvement	Unchanged	5 Participants
LipaCreon	Degree of General Improvement	Exacerbated	0 Participants
LipaCreon	Degree of General Improvement	Unassessable	0 Participants

Secondary

Degree of General Improvement

Time frame: At 24 weeks

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. Of these 21 patients, 17 patients were obtained with data on Degree of General Improvement at 24 weeks.

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
LipaCreon	Degree of General Improvement	Improved	10 Participants
LipaCreon	Degree of General Improvement	Unchanged	7 Participants
LipaCreon	Degree of General Improvement	Exacerbated	0 Participants
LipaCreon	Degree of General Improvement	Unassessable	0 Participants

Secondary

Nutritional Endpoints - BMI

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)