Cystic Fibrosis MedDRA version: 17.1 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Algipharma AS
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Male or female with a confirmed diagnosis of cystic fibrosis defined by: a. Clinical features consistent with the diagnosis of CF [(Rosenstein BJ and Cutting GR 1998)]; AND Sweat chloride =60 mmol/L by pilocarpine iontophoresis; OR b. Genotypic confirmation of CFTR mutation 2) Aged 18 years or older 3) Positive microbiological finding of Pseudomonas aeruginosa in expectorated sputum or cough swab documented within 24 months prior to Screening (Visit 1). 4) FEV1 must, at Screening (Visit 1), be between 40%-100% of the predicted normal value following adjustment for age, gender, and height according to the Global Lung Initiative (GLI) equation (Eur Respir J. Dec 2012; 40(6): 1324–1343)). For subjects to be included in the LCI assessment at selected sites, the FEV1 at screening should be in the range of 60-100%. 5) At Screening (Visit 1), no clinical or laboratory findings suggestive of significant pulmonary illness, other than CF, which in the opinion of the investigator would preclude participation in the study 6) Female subjects of child bearing potential and male subjects participating in the study who are sexually active must use acceptable contraception. Female subjects documented as being of non child-bearing potential (Section 4.2.9) are exempt from the contraceptive requirements. For the purpose of this study acceptable contraception is defined as: a. oral, injected transdermal or implanted hormonal methods of contraception; OR b. placement of an intrauterine device (IUD) or intrauterine system (IUS); OR c. barrier methods of contraception: condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository 7) Provision written informed consent Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 76 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 76
Exclusion criteria
Exclusion criteria: 1) Changes in underlying therapy (e.g., chest physiotherapy, bronchodilators, NSAIDs, antibiotic agents, pancreatic enzyme preparations, nutritional supplements and DNase within the 14 days prior to Day 0 (Visit 2). Subjects must be willing to remain on the same underlying stable therapy regimens for the duration of the study until the final follow-up visit at Day 98. 2) Changes in physiotherapy technique or schedule within 14 days prior to Day 0 (Visit 2). 3) Prohibited medications within 7 days prior to Day 0 (Visit 2). Prohibited medications are described in Section 5.6. 4) Pulmonary exacerbation within 28 days of Screening (Visit 1) 5) Positive microbiological finding of Burkholderia sp. in expectorated sputum or cough swab documented within 12 months prior to Screening (Visit 1). 6) Lactose intolerance/milk allergy. A skin test for milk allergy will be performed at screening. 7) On-going acute illness. Subjects must not have needed an outpatient visit, hospitalization or required any change in therapy for other pulmonary disease between Screening (Visit 1) and Day 0 (Visit 2). 8) History of, or planned organ transplantation. 9) Allergic bronchopulmonary aspergillosis (ABPA) in the last 12 months prior to Screening (Visit 1), defined as having received treatment for ABPA. 10) Requirement for continuous (24 hour/day) oxygen supplementation. 11) Diagnosed with the G551D-mutation, and currently on concomitant treatment with Ivacaftor (Kalydeco). 12) Concomitant administration of inhaled mannitol or hypertonic saline within 7 days prior to Day 0 (Visit 2). Subjects on hypertonic saline can if needed switch to isotonic saline 7 days prior to Day 0. 13) Initiation of cycled, inhaled tobramycin (TOBI) and Colistin less than 4 months prior to Screening (Visit 1). Note: Chronic TOBI and Colistin users are allowed to participate in this study, but subjects who have recently initiated chronic TOBI or Colistin should have at least 2 cycles of TOBI or Colistin respectively in the preceding 4 months before being enrolled in this study. Chronic TOBI and Colistin subjects should be starting an ‘off-TOBI’ or alternatively ‘off-Colistin’ period at Day 0 (Visit 2), to make sure that the treatment is phased in line with the antibiotic treatment. 14) Concomitant use of all other marketed antibiotic agents is permitted, providing subjects are willing to remain on the same regimens within the 21 days immediately prior to Day 0 (Visit 2) and for the entire duration of the study (until Day 98). 15) Clinically significant abnormal findings on haematology or clinical chemistry. In addition any value = 3 x the upper limit of normal will exclude the subject from participating in the study. 16) Subjects unable to perform pulmonary function tests according to the ATS/ERS criteria. 17) Pregnant or breast-feeding women. A negative urine pregnancy test must be demonstrated in females of child-bearing potential (Section 4.2.9) at Screening (Visit 1). 18) Subjects who have participated in any interventional clinical trial within the 28 days prior to Day 0 (Visit 2). 19) Subjects with documented or suspected, clinically significant, alcohol or drug abuse. The determination of clinical significance will be determined by the Investigator. 20) Current malignant disease (with the exception of basal cell carcinoma and cervical neoplasia). 21) Any serious or active medical or psychiatric illness, which in the opinion of the Investigator, would interfere
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Demonstrate efficacy of inhaled OligoG measured by FEV-1 and supported by secondary endpoints including Mucociliary Clearance, rheology, microbiology and Quality-of-Life;Secondary Objective: To demonstrate the safety and tolerability of inhaled OligoG as a dry powder for inhalation after multiple dose administration. To evaluate patient compliance with treatment.;Primary end point(s): FEV1 ;Timepoint(s) of evaluation of this end point: Baseline and day 28 of each treatment period | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1) Combined mucociliary and cough clearance at selected sites 2) Quality of Life by CFQ-R 3) Sputum rheology 4) Microbiological (culture and culture independent) measurements including P. aeruginosa density in expectorated sputum 5) Other lung function tests ;Timepoint(s) of evaluation of this end point: Baseline and day 28 of each treatment period | — |
Countries
Denmark, Germany, Sweden
Contacts
Public ContactProject Manager
SMERUD Medical Research
Outcome results
None listed