A double-blind, randomized, placebo-controlled, cross-over study to evaluate the safety, tolerability and preliminary efficacy of alginate oligosaccharide (OligoG) administered for 28 days in subjects with Cystic Fibrosis chronically colonised with Pseudomonas aeruginosa
- Conditions
- Cystic FibrosisMedDRA version: 14.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2010-023090-19-IE
- Lead Sponsor
- AlgiPharma AS
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 24
In order to participate in the study subjects must meet all of the following inclusion criteria:
1)Male or female with a confirmed diagnosis of cystic fibrosis defined by:
a.Clinical features consistent with the diagnosis of CF AND
b.Sweat chloride =60 mmol/L by pilocarpine iontophoresis; OR
c.Genotypic confirmation of CFTR mutation
2)Aged 18 years or older
3)Ability to provide samples for microbiological evaluation throughout the study. Note: sputum samples are preferred however cough swabs may be performed on occasions where sputum cannot be collected.
4)Positive microbiological finding of Pseudomonas aeruginosa in expectorated sputum or cough swab documented within 24 months prior to Screening (Visit 1).
5)FEV1 must, at Screening (Visit 1), be between 35%-80% of the predicted normal value following adjustment for age, gender, and height according to the Knudson equation.
6)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
7)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 are exempt from the contraceptive requirements. For the purpose of this study acceptable contraception is defined as:
a.oral, injected 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
8)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 5
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 0
In order to participate in the study subjects must not meet any of the following exclusion criteria
1)Changes in underlying therapy (e.g., pulmonary massage therapy, bronchodilators, NSAIDs, antibiotic agents, pancreatic enzyme preparations, nutritional supplements and DNase within the 21 days prior to Day 1 (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 111.
2)Changes in physiotherapy technique or schedule within 14 days prior to Day 1 (Visit 2).
3)Prohibited medications within 7 days prior to Day 1 (Visit 2).
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)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 1 (Visit 2).
7)History of, or planned organ transplantation.
8)Allergic bronchopulmonary aspergillosis (ABPA) in the last 12 months prior to Screening (Visit 1), defined as having received treatment for ABPA.
9)Requirement for continuous (24 hour/day) oxygen supplementation.
10)Concomitant administration of inhaled mannitol or hypertonic saline within 7 days prior to Day 1 (Visit 2).
11)Initiation of cycled, inhaled tobramycin (TOBI) less than 4 months prior to Screening (Visit 1). Note: Chronic TOBI users are allowed to participate in this study, but subjects who have recently initiated chronic TOBI should have at least 2 cycles of TOBI in the preceding 4 months before being enrolled in this study. Chronic TOBI subjects should be starting an ‘off-TOBI’ period at Day 1 (Visit 2) so there will be no concomitant dosing of TOBI and assigned study medication.
12)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.
13)Subjects unable to perform pulmonary function tests according to the ATS/ERS criteria.
14)Pregnant or breast-feeding women. A negative urine pregnancy test must be demonstrated in females of child-bearing potential at Screening (Visit 1).
15)Subjects who have participated in any interventional clinical trial within the 28 days prior to Day 1 (Visit 2).
16)Subjects with documented or suspected, clinically significant, alcohol or drug abuse. The determination of clinical significance will be determined by the Investigator.
17)Known allergies or intolerance to alginates (e.g., foods and food additives based on seaweed extracts).
18)Current malignant disease (with the exception of basal cell carcinoma; BCC).
19)Any serious or active medical or psychiatric illness, which in the opinion of the Investigator, would interfere with subject treatment, assessment, or compliance with the protocol.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The principle research objective is:<br>To determine the safety and local tolerability of multiple dose administration of inhaled OligoG in subjects with cystic fibrosis. Particular emphasis will be put on local, clinical tolerance, lung function and adverse events related to lung function.;Secondary Objective: The secondary research objectives are:<br>The effect of multiple dose administration of inhaled OligoG on various efficacy variables will also be explored, such as break up of mucus structure, lung function, respiratory symptoms, Quality-of-Life and microbiological outcome measures.;Primary end point(s): To determine the safety and local tolerability of multiple dose administration of inhaled OligoG in CF subjects. Particular emphasis will be put on local, clinical tolerance, pulmonary function and pulmonary adverse events.;Timepoint(s) of evaluation of this end point: The primary endpoint will be evaluated at visits 1, 2, 3, 4, 5, 6, 7, 8, 9
- Secondary Outcome Measures
Name Time Method Secondary end point(s): The effect of multiple dose administration of inhaled OligoG on various efficacy variables will also be explored, such as mucolytic activity, lung function, respiratory symptoms, Quality-of-LIfe and microbiological outcome measures.;Timepoint(s) of evaluation of this end point: The secondary endpoint will be evaluated at visits 2, 3, 4, 5, 6, 7, 8, 9