Status:

TERMINATED

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

Lead Sponsor:

AlgiPharma AS

Conditions:

Cystic Fibrosis

Eligibility:

All Genders

18+ years

Phase:

PHASE2

Brief 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 includ...

Detailed Description

The alginate oligosaccharide OligoG CF-5/20 dry powder for inhalation (OligoG DPI) represents a novel therapeutic approach for patients with cystic fibrosis (CF). OligoG has been shown to release stag...

Eligibility Criteria

Inclusion

  • 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

  • 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.

Key Trial Info

Start Date :

May 16 2019

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

July 10 2021

Estimated Enrollment :

15 Patients enrolled

Trial Details

Trial ID

NCT03822455

Start Date

May 16 2019

End Date

July 10 2021

Last Update

October 28 2024

Active Locations (1)

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John Hunter Hospital

Newcastle, New South Wales, Australia