Orkambi in Patients with Cystic Fibrosis and Severe Liver Disease

Phase 1

Recruiting

• Conditions: Cystic Fibrosis; Cystic Fibrosis Related Liver Disease; Respiratory - Other respiratory disorders / diseases; Oral and Gastrointestinal - Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon; Human Genetics and Inherited Disorders - Cystic fibrosis

• Registration Number: ACTRN12619001347156
• Lead Sponsor: Dr Adeline Lim

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-10-01
• Last Update Posted: 13 February 2023

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

Phase 1
1. Children between 6 years old and 18 years of age homozygous for Phe508del-CFTR
2. Severe cystic fibrosis related liver disease

Phase 2
1. Children between 2 years old and 18 years of age homozygous for Phe508del-CFTR
2. Severe cystic fibrosis related liver disease

Patients without severe cystic fibrosis related liver disease
1. Children between 2 years old and 18 years of age homozygous for Phe508del-CFTR
2. On full dose Orkambi for at least two weeks prior to enrolment to the study

Exclusion Criteria

Cystic fibrosis without severe liver disease
Exclusion criteria - presence of cirrhosis and portal hypertension

These patients must already be receiving full dose Orkambi for at least two weeks prior to enrolment to this group.

Study & Design

• Study Type: Interventional
• Study Design: Not specified