ALX- 009 in cystic fibrosis and non-CF bronchiectasis
Research type
Research Study
Full title
Randomized, double-blind, placebo-controlled study of the safety, tolerability and pharmacokinetics after single ascending doses or multiple ascending doses of hypothiocyanite (OSCN-), / bovine Lactoferrin (bLF), or their combination (ALX- 009) in healthy male volunteers and patients suffering from cystic fibrosis (CF) and non-CF bronchiectasis (NCFBE).
IRAS ID
265365
Contact name
Joseph Elborn
Contact email
Sponsor organisation
ALAXIA SAS
Eudract number
2014-002401-38
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 7 months, 30 days
Research summary
This is a phase I, multicenter, double-blind, placebo-controlled, randomised, parallel-group, multiple ascending doses study in healthy male volunteers and patients suffering from Cystic fibrosis and non-CF bronchiectasis.
The test products are: ALX-009, Bovine Lactoferrin (bLF) and OSCN- solution
There are four parts in this study.
103 volunteers, aged from 18 to 50 years will be included in the study:
- Part I: 42 healthy male subjects - Completed
- Part II: 16 healthy male subjects - Completed
- Part III: 7 CF patients (completed) and 14 healthy male subjects
- Part IV: 12 healthy male subjects and 12 patients (CF and/or NCFBE).
UK will only participate in the Part IV patients’ cohorts. No healthy volunteers will be recruited in the UK.
All patients will receive multiple nebulized doses of combination of the test product (ALX-009) or placebo twice a day during 7 days at 3 different doses.
ALX-009 presented as a solution for inhalation, is composed of 2 endogenous substances (OSCN-/Lactoferrin) with antimicrobial properties mimicking the innate immune system.
Lactoferrin is a glycoprotein extracted from cow milk, that inhibits bacterial growth mainly through iron deprivation but also via specific binding to receptors on the surface of some microorganisms.
The hypothiocyanite anion OSCN- kills bacteria by targeting active thiol residues of key enzymes involved in microbial metabolism. When combined, in vitro studies evidenced that OSCN- immediately, i.e. within the first 2 hours of exposure, kills the sensitive bacteria, while LF allows for maintaining this bactericidal effect, resulting in a prolonged effect.
In Part IV only ALX-009 will be administered.
This study is being conducted to evaluate the safety and tolerability of the study drugs and their combination after multiple administrations at different doses of the selected ratios.REC name
East of England - Cambridge East Research Ethics Committee
REC reference
19/EE/0321
Date of REC Opinion
3 Jan 2020
REC opinion
Further Information Favourable Opinion