Combination Therapy effect on Exercise Tolerance - Cystic Fibrosis
Research type
Research Study
Full title
A Phase 4, Randomized, Double-Blind, Placebo-controlled, Parallel-Design Study of the effect of Lumacaftor/Ivacaftor Combination Therapy on Exercise tolerance in subjects aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
IRAS ID
202462
Contact name
Don Urquhart
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2016-000066-34
Duration of Study in the UK
1 years, 4 months, 31 days
Research summary
Cystic fibrosis (CF) is a recessive disease with serious, chronically debilitating effects and high early death rate, and at present, there is no cure. Cystic fibrosis affects approximately 70,000 individuals worldwide, with approximately 30,000 individuals in the US, 36,000 individuals in the European Union (EU), 3,800 individuals in Canada, and 3,000 individuals in Australia. Despite progress in the treatment of CF, the predicted age of survival for a person with CF is approximately 40 years. Although the disease affects multiple organs, progressive loss of lung function is the leading cause of mortality.
This study is designed to look at the effect of lumacaftor/ivacaftor (LUM/IVA) combination therapy on exercise tolerance in subjects with cystic fibrosis (CF). Patients with CF exhibit exercise difficulties, and although pulmonary symptoms are partially responsible, heart or muscle factors are believed to be the major limitation in patients with milder disease.
Patients who are found to be eligible for the study will be consented and they will follow an treatment phase of approximately 8 months with either lumacaftor/ivacaftor (LUM/IVA) combination therapy or a Placebo.
Participants will be randomised to receive either lumacaftor/ivacaftor (LUM/IVA) combination therapy or a Placebo. in a blinded manner. Patients will receive the drug they will require at the study visits on Day 1 and week 12 once they are randomised onto the study and instructed to take one tablet twice per day orally within 30 minutes of consumption of fat-containing food with a glass of water each morning.
This is a multicentre, multinational, randomised, double-blind study comparing the efficacy and safety of lumacaftor/ivacaftor (LUM/IVA) combination therapy to a placebo in patients with CF
Study Sites: This study is to be conducted at approximately 13 study sites in the UK and Australia.REC name
East of Scotland Research Ethics Service REC 2
REC reference
16/ES/0067
Date of REC Opinion
24 Jun 2016
REC opinion
Further Information Favourable Opinion