HOPE-1 Study: Hydration for Optimal Pulmonary Effectiveness
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled Phase II Study to Evaluate the Efficacy and Safety of SPX-101 Inhalation Solution in Subjects with Cystic Fibrosis
IRAS ID
225743
Contact name
Damian Downey
Contact email
Sponsor organisation
Spyryx Biosciences, Inc.
Eudract number
2016-005230-30
Duration of Study in the UK
1 years, 5 months, 0 days
Research summary
Cystic fibrosis (CF) is a genetic disorder affecting over 10,000 individuals in the UK. It is caused by defects in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Patients with CF have repeated infections that damage the lungs and ultimately cause them to fail. The majority of deaths in CF are due to lung disease
The loss of CFTR function leads to chronic lung disease and the abnormal increase of cell receptors of ENaC (epithelial sodium channel) can result in thickened sputum that can be difficult to expectorate. This retention can cause lung damage and is a source of chronic infection. Current therapies for CF focus on antibiotic treatment of airway infections, nebulised therapies, airway clearance techniques such as chest physiotherapy and more recently CFTR modulators.
It has been discovered that a natural inhibitor of ENaC, short palate lung and nasal epithelial clone 1 (SPLUNC1), works by increasing mucus transportation, resulting in the reduced effect of ENaC’s activity in the airways. However, the effects of SPLUNC1 rely on the change in pH (a measure of the acidity or alkalinity of a solution) and are lost in the CF airway which is acidic. SPX-101 was developed to address this shortcoming of SPLUNC1.
This study will investigate how safe and effective the study medication, SPX-101, is in patients with CF. SPX-101/Placebo will be administered via inhalation by a nebulised aerosol (PARI eFlow® nebuliser with eTrack®). eTrack will assess study medication compliance via Bluetooth. Patients will follow their usual routine of medication and chest physiotherapy, unchanged, throughout the study
SPX-101 can be effectively delivered to the CF lung and as it acts through ENaC instead of CFTR, SPX-101 may be effective for all patients with CF, regardless of genetic mutation.
REC name
HSC REC A
REC reference
17/NI/0096
Date of REC Opinion
3 Jul 2017
REC opinion
Further Information Favourable Opinion