A Phase 2b Randomised, Placebo Controlled Study of OligoG in Patients With Cystic Fibrosis

• Status: Active, not recruiting
• Trial end date: 2021-07-10
• Target enrollment: 0
• Participant gender: All

Summary

A double-blind, randomised study of OligoG DPI compared to placebo DPI, both on top of standard-of-care, to assess safety, efficacy and tolerability. Adult patients with Cystic Fibrosis will be included in the study.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: AlgiPharma AS
• Collaborators: AlgiPharma Australia Pty. Ltd.; AlgiPharma Pty Ltd
• Treatments: Alginic acid

Criteria

Inclusion Criteria:

• Genotypic confirmation of CFTR mutation or clinical diagnosis of Cystic Fibrosis (CF) confirmed by a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis.

• Age 18 years or older.

• Male or female patients with any ethnicity.

• FEV1 at screening in the range of ≥40% and 90% of the predicted normal for age, sex, and height, according to the GLI equation (Eur Respir J. Dec 2012; 40(6): 1324-1343).

• History of Pseudomonas aeruginosa (PA) infection with at least one positive microbiological PA testing during the last 12 months before the Screening Visit.

• History of antibiotic treatment due to PA infection (not for eradication therapy) during the last 12 months

• Concomitant treatment with inhaled tobramycin, colistin, or aztreonam (either cycled or continuous) for at least 3 months at screening to treat PA infection. In case of cycled antibiotic treatment, the treatment should start with an active cycle at the day of randomisation (+/- 2 day) (together with the IMP intake). If taking tobramycin cycled with another antibiotic, IMP should start on the active cycle of tobramycin.

• Stable CF disease as judged by the investigator.

• Willing to remain on a stable CF medication regimen (standard of care; SOC) during the study.

• Women of child-bearing potential must have a negative urine pregnancy test at the Screening and Randomisation Visit.

• Male and female patients must use acceptable contraceptive methods for the duration of the study. Male and female patients without child-bearing potential (i.e. who are infertile, surgically sterile or post-menopausal) are exempted from the contraceptive requirements. For the purpose of this study acceptable contraception is defined as one or a combination of the following:

• oral, injected, transdermal or implanted hormonal methods of contraception; placement of an intrauterine device (IUD) or intrauterine system (IUS); barrier methods of contraception: condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository.

• Capable of inhaling dry powder.

• Willing to sign informed consent

• Willing and able to follow the study procedures.

Exclusion Criteria:

• Use of hypertonic saline more than 2 times a day. If hypertonic saline is used, OligoG inhalation should be taken at least 15 minutes after completion of hypertonic saline therapy.

• Use of CFTR modulator therapies.

• Clinically significant abnormal findings of haematology or clinical chemistry;

• Elevated gamma GT (GGT), ALT, or AST > 3x the upper normal limit of normal (ULN)

• Bilirubin >2x ULN

• Abnormal renal function, with a creatinine clearance calculated <50ml/min

• Haemoglobin <10g/dL

• History of any comorbidity that, in the opinion of the investigator, might distort the results of the study or cause an additional risk in administering study drug to the patient.

• Pulmonary exacerbation within 28 days prior to randomisation.

• Change in CF therapy within 28 days before randomisation (first dose of IMP).

• Pregnant or breastfeeding females.

• History of allergic reactions to the ingredients of the IMP according to Common Terminology Criteria for Adverse Events (CTCAE) grade 3 or 4, including lactose and milk protein.

• Patients unable to perform pulmonary function tests according to the ATS/ERS criteria.

• Uncontrolled or unstable chronic diseases (e.g. congestive heart failure, cardiac arrhythmia, or psychiatric illness/social situations) that would limit the compliance with study requirements in the opinion of the investigator.

• Any acute illness in the last 14 days

• History of, or planned organ transplantation.

• Lung infection with organisms associated with a more rapid decline in pulmonary status (including, but not limited to Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus). For subjects who have had a history of a positive culture, the following criteria will be used to determine whether the subject is free of infection with such organisms:-

• The subject has not had a respiratory tract culture positive for these organisms within the 12 months before the date of informed consent, and

• The subject has had at least 2 respiratory tract cultures negative for such organisms within the 12 months before the date of informed consent, with the first and last of these separated by at least 3 months, and the most recent one within the 6 months before the date of informed consent.

• Active allergic bronchopulmonary aspergillosis (ABPA) in the last 12 months prior to the Screening Visit, that has received pharmacological treatment for ABPA.

• Requirement for continuous (24 hour/day) oxygen supplementation.

• Patients currently receiving any other investigational treatment, or who have participated in a clinical study within 4 weeks (28 days) prior to the screening visit.

• Current malignant disease (with the exception of basal cell carcinoma and cervical neoplasia).

• Any medical or psychological condition, other than CF, which in the opinion of the investigator exposes the patient to an unacceptably high risk.

• Patients with documented or suspected, clinically significant, alcohol or drug abuse as per Investigator's discretion.