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

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.

Study Overview

• Status: Completed
• Conditions: Digestive System Diseases; Pancreatic Diseases; Cystic Fibrosis; Exocrine Pancreatic Insufficiency
• Intervention / Treatment: Drug: Pancrelipase

• Study Type: Observational
• Enrollment (Actual): 24

Contacts and Locations

Study Locations

• Japan
  • Aichi, Japan, 462-8508
    • Site Reference ID/Investigator# 73813
  • Aichi, Japan, 466-8650
    • Site reference ID/Investigator # 96698
  • Aichi, Japan, 467-8602
    • Research facility ID ORG-000141
  • Aichi, Japan, 470-0224
    • Site reference ID/Investigator no. ORG-000842
  • Aichi, Japan, 474-8710
    • Site reference ID/Investigator # 93736
  • Ehime, Japan, 794-0006
    • Research facility ORG-000972
  • Hyōgo, Japan, 660-8550
    • Mylan investigational site A
  • Ibaraki, Japan, 305-8520
    • Site reference ID/Investigator # 93735
  • Ibaraki, Japan, 305-8558
    • Site Reference ID/Investigator# 88673
  • Ishikawa, Japan, 920-8641
    • Site Reference ID/Investigator# 65529
  • Kagawa, Japan, 760-0017
    • Site Reference ID/Investigator# 65530
  • Kagoshima, Japan, 890-8520
    • Site Reference ID/Investigator# 65527
  • Kanagawa, Japan, 232-8555
    • Mylan investigational site C
  • Miyagi, Japan, 980-0872
    • Site Reference ID/Investigator# 65528
  • Miyagi, Japan, 989-3126
    • Mylan investigational site B
  • Oita, Japan, 874-0011
    • Research facility ID ORG-001138
  • Osaka, Japan, 534-0021
    • Research facility ID ORG-000594
  • Shizuoka, Japan, 438-8550
    • Research facility ORG-000971
  • Tochigi, Japan, 329-0431
    • Site reference ID/Investigator# 117495
  • Tokyo, Japan, 108-8329
    • Research facility no. ORG-001309
  • Tokyo, Japan, 157-8535
    • Site Reference ID/Investigator# 67122

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 99 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients who receive LipaCreon for the replacement of pancreatic digestive enzymes in pancreatic exocrine insufficiency

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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.	Drug: Pancrelipase; Other Names: Lipacreon

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).; 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.; 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	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).

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Mylan Inc.
• Investigators: Study Director: Shigenori Haruna, Mylan EPD G.K.

Study record dates

Study Major Dates

• Study Start (Actual): August 1, 2011
• Primary Completion (Actual): March 1, 2018
• Study Completion (Actual): March 1, 2018

Study Registration Dates

• First Submitted: August 31, 2011
• First Submitted That Met QC Criteria: August 31, 2011
• First Posted (Estimate): September 2, 2011

Study Record Updates

• Last Update Posted (Actual): June 10, 2022
• Last Update Submitted That Met QC Criteria: March 9, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Keywords: Cystic Fibrosis; Digestive System Diseases; Therapeutic Uses; Exocrine Pancreatic Insufficiency; Pancreatic Diseases; Pancrelipase; Gastrointestinal Agents
• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Fibrosis; Gastrointestinal Diseases; Digestive System Diseases; Cystic Fibrosis; Pancreatic Diseases; Exocrine Pancreatic Insufficiency; Gastrointestinal Agents; Pancrelipase; Pancreatin
• Other Study ID Numbers: P12-893

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: We will consider after completion of CSR