Cystic Fibrosis
Conditions
Keywords
Oligosaccharide
Brief summary
Randomized, double blind, placebo controlled study. The study has two parts: Dose-finding part, followed by longer term follow-up (6 months)
Detailed description
Part 1: Randomized, double blind, placebo controlled dose-finding. Patients will be assigned to 1 of 3 doses OligoG, or to placebo, on top of Standard of Care. Patients will be treated for 12 weeks, followed by 4 weeks washout.The primary endpoint is relative change in % predicted FEV1. Secondary endpoints include additional spirometry parameters, exacerbation rate, Quality of Life, sputum rheology and microbiology, safety laboratory tests and adverse event reporting. Part 2: Randomized double-blind 6 -month study, for longer term follow-up of the dose identified in Part 1. New patients will be recruited in part 2, in addition to patients who received placebo in Part 1. In addition to the endpoints studied in Part 1, Part 2 will include Lung Clearance Index (LCI), chest imaging by MRI or CT, and pharmaco-economic parameters.
Interventions
DRUGOligoG Dry powder for inhalation
10 capsules Dry Powder for Inhalation, BID
Sponsors
Imperial College London
Cystic Fibrosis Foundation
European Cystic Fibrosis Society
University Hospital of Cologne
Cystic Fibrosis Europe
AlgiPharma AS
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Placebo medication with identical appearance to the active drug
Intervention model description
Randomised, double blind, dose-finding
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Confirmed diagnosis of CF * FEV1 at screening \>40 and \< 90% of predicted normal * History of PA infection in last 12 m * History of antibiotic treatment due tp PA infection (not for eradication) during last 12 m * Concomitant inhaled tobramycin, colistin, aztreonam, or levoflaxin (cycled or continuous) * Stable CF disease * Willing to remain on stable CF medication (Standard of care) * Women of child-bearing potential must habe negative urine pregnany test * Males & females must use acceptable contraception * Capable of inhaling dry powder * willing to sign informed consent * willing and able to follow study procedures
Exclusion criteria
* Use of hypertonic saline \> twice daily * Clinically significant abnormal lab findings, except CRP. In case of high GGT values, case will be discussed by experts before deciding * History of comorbidity that may distort results or cause additional risk * pulmonary exacerbation within 28 days prior to randomisation * Change in CF therapy within 28 days prior to randomisation * Burkholderia spp. finding within 12 m prior to screening * pregnant or breast feeding females * History of allergic reactions to IMP ingredients, incl milk protein * Inability to perform lung function tests according to ATS/ ERS criteria * Uncontrolled or unstable diseases that might limit compliance * History of, or planned organ transplantation * Allergic ABPA in the last 12 months prior to the screening visit * Requirement for continuous oxygen supplementation * Current participation in another clinical study * medical condition, other than CFwhich exposes the patient to an unacceptably high risk * Concurrent mlignant disease, except BCC and cervical neoplasia * Clinically significant alcohol or drug abuse
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Forced Expiratory Volume in 1 second (FEV1) | Change from before to after 12 weeks treatment (Part 1) and 26 weeks (Part 2) | the amount of air a person can forcefully exhale in one second |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Lung Clearance Index (LCI) (Part two only) | Change from before to after 26 weeks treatment | Lung clearance index (LCI) measured by multiple breath washout is a sensitive measure of ventilation inhomogeneity. |
| Sputum culture microbiology | Change from before to after 12 weeks treatment (Part 1) and 26 weeks (Part 2) | Reduction in Pseudomonas Aeruginosa CFU Counts and total counts |
| Pulmonary Exacerbations | Change from before to after 12 weeks treatment (Part 1) and 26 weeks (Part 2) | Acute pulmonary exacerbations |
Countries
Germany
Outcome results
None listed