M19-530: Ph2 Cystic Fibrosis ABBV-3067 & ABBV-2222 in F508del
Research type
Research Study
Full title
A Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in Cystic Fibrosis Subjects Who Are Homozygous for the F508del Mutation
IRAS ID
268446
Contact name
Daniel Peckham
Contact email
Sponsor organisation
AbbVie Ltd.
Eudract number
2019-000750-63
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 7 months, 25 days
Research summary
Cystic Fibrosis (CF) is a genetic condition that affects many parts of the body causing reduced functions in organs like lungs and pancreas. Disease symptoms include breathing difficulties, infections, digestive problems and diabetes. People with CF often have short life spans with reduced quality of life.\n\nCF is caused by mutations in Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene that affect production and function of CFTR protein. CFTR works on cell surface by transporting chemicals in and out of the cell. When CFTR is not produced correctly, it affects the salt and fluid balance inside and outside of the cell. CFTR modulators can help cells produce more CFTR on the surface by helping the protein form the correct shape (“correctors”) or helping CFTR work better (“potentiators”). \n\nIn this study, patients will be given ABBV-3067 (“potentiator”) and ABBV-2222 (“corrector”) to see if treatment improves patients’ lung function. Different groups of patients will receive different doses, and some will receive a dummy drug (“placebo”). This study evaluates the safety and efficacy (how well the drugs work) of the drugs and the best dose of each drug to be used in future studies.\n\nThis is a Phase 2, double-blinded (treatment type is unknown to patients and investigators) study in CF patients who have two copies of the F508del mutation (the most common CFTR gene mutation). \n\nPatients go through a screening process (up to a month) and receive study treatment for up to a month, followed by a review, a month later. Tests done for the study include lung function tests, sweat chloride tests, blood and urine tests, and ECG. Side effects are monitored through the course of the study.\n\nApproximately 189 adult patients (male and female) will take part in approximately 59 sites worldwide. AbbVie is funding this study.
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
19/YH/0301
Date of REC Opinion
31 Oct 2019
REC opinion
Further Information Favourable Opinion