Clinical trials are listed below.
Mucociliary ClearanceCompleted with results Location: USA
Phase 3 study of inhaled mannitol in adults with cystic fibrosis , protocol number DPM-CF-303 NCT02134353
This study is taking place at multiple care centers across the U.S. It will look at the safety and effectiveness of the inhaled drug mannitol and will use a placebo control.
Age:
18 Years and Older
Mutation(s):
No Mutation Requirements
FEV1% Predicted:
40 to 90%
Number of Visits:
5
Length of Participation:
6
Restore CFTR FunctionEnrolling Location: Australia - QLD
A Study to Evaluate Safety, PK and PD of FDL169 in Cystic Fibrosis Subjects , protocol number FDL169-2015-04 NCT03093714
A Randomized, Double-Blind, Placebo-Controlled, Parallel Study to Evaluate Safety, Pharmacokinetics (PK) and Pharmacodynamics(PD) of FDL169 in Cystic Fibrosis (CF) Subjects Homozygous for the F508del-CFTR Mutation
18 Years to 85 Years
Homozygous for the F508del-CFTR mutation.
(FEV1) >40% of predicted normal for age, sex and height.
Not specified
Not Specified
OtherEnrolling Location: Multicentre
Saline Hypertonic in pre-schoolers + CT (SHIP-CT) , protocol number SHP002 NCT02950883
The purpose of this study is to assess whether inhalation of 7% hypertonic saline (HS) twice daily for 48 weeks reduces structural lung disease as assessed by computed tomography (CT) in comparison with inhalation of 0.9% isotonic saline (IS) in preschool children (ages 3 to 6) with cystic fibrosis.
3-5 years
A documented genotype with two disease-causing mutations in the CFTR gene
Restore CFTR FunctionEnrolling Location: Perth Linear Clinical Research, Contact: Jennifer Seabourne Email: greenteam@linear.org.au • Brisbane Q-Pharm Contact: Sharon Rankine Phone: 0439 664 900 Email: s.rankine@qpharm.com.au
Study to Assess the Safety, Tolerability and PK Profile of FDL176 in Healthy and CF Participants. , protocol number FDL176-2016-01 NCT03173573
Study to Assess the Safety, Tolerability and PK Profile of FDL176 in Healthy and CF Participants , protocol number FDL176-2016-01 NCT03173573 This is a 5-part study. FDL176 is being tested on healthy male and female participants in parts 1-4 Part 5 is a single dose, open-label study in male and female participants with CF to determine the PK profile of FDL176. • Eligible participants will be reimbursed for their time. • Study sites are located in Perth and Brisbane. • Further information can be obtained at: https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=6602&isClinicalTrial=True
18 Years or older
Mutation(s): 2 CF causing mutations or diagnosis of CF defined as a sweat chloride value of >/= 60mmol/L by quantitative pilocarpine iontopheresis
FEV1% Predicted: >/= 40%
Screening, A visit that involves 3 overnight stays and a Follow up visit
Approximately 4 weeks.
Restore CFTR FunctionClosed to enrollment Location: USA & Australia
Phase 3 study of Ataluren in people with CF who have a nonsense mutation and are not taking aminoglycosides. , protocol number PTC124-GD-021-CF NCT02139306
This study is taking place at multiple care centers across the U.S. It looked at the effectiveness and safety of the oral drug ataluren in people with CF who have a nonsense mutation of cystic fibrosis. This study used a placebo control.
6 Years and Older
One Copy F508del or No Copies F508del
60 to 90%
10
56 weeks
Mucociliary ClearanceEnrolling Location: Multiple care centres across the US
Cohort 3 of a study to evaluate inhaled QBW276 in adults with CF , protocol number Novartis QBW276 - Cohort 3
This randomized, placebo-controlledstudy is taking place at multiple care centers across the US. This study will look at the safety, effectiveness and tolerability of the inhaled drug QBW276.
18 years and older
Two copies of F508del
40-100%
15
3 months
Restore CFTR FunctionEnrolling Location: Multiple care centres across the US & Australia
Phase 1/2 Study of VX-445 combination drug in healthy adults and then in people with cystic fibrosis. , protocol number NCT03227471 VX16-445-001
This study is taking place at multiple care centres across the US and Australia. It will look at the safety and effectiveness of the drug VX-445.
No mutation requirement
40-90%
12 weeks
A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis Who Have an F508del-CFTR Mutation , protocol number NCT02565914 VX14-661-110
This is a Phase 3, multicenter, open-label, 2-part rollover study in subjects with CF who are homozygous or heterozygous for the F508del-CFTR mutation and who participated in Studies NCT02070744, NCT02347657, NCT02516410, NCT02392234, NCT02412111, and NCT02508207. The study is designed to evaluate the safety and efficacy of long term treatment of VX-661 in combination with ivacaftor.
12 years and older
OtherEnrolling Location: NSW - Australia
A Phase 3 multi-centre randomised placebo-controlled study of azithromycin in the primary prevention of radiologically-defined bronchiectasis in infants with cystic fibrosis (COMBATCF). , protocol number ACTRN12610001072000 NCT01270074
The general aim of this project is to conduct a randomized double-blind, placebo-controlled clinical trial of azithromycin to determine whether treatment from infancy is safe and will prevent the onset of bronchiectasis. One hundred and thirty infants will be recruited from CF clinics in Australia and New Zealand and treated from 3 months to three years of age. The primary outcome will be the proportion with radiologically-defined bronchiectasis at 3 years of age. Safety and mechanistic evaluations will also be undertaken.
6 Weeks to 6 Months
0
Restore CFTR FunctionClosed to enrollment Location: Multicentre
A Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate GLPG2222 in Ivacaftor-treated Subjects With Cystic Fibrosis Harbouring One F508del CFTR Mutation and a Second Gating (Class III) Mutation , protocol number GLPG2222-CL-201 NCT03045523
This clinical study is a phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel group study to evaluate two doses of orally administered GLPG2222 in adult subjects with a confirmed diagnosis of CF harbouring one F508del CFTR mutation and a second gating (class III) mutation and on stable treatment with ivacaftor. Up to 35 evaluable subjects are planned to be included in the study. Eligible subjects must be on stable treatment with physician prescribed ivacaftor (Kalydeco®) for at least 28 days at the baseline visit. They will be randomized in a 2:2:1 ratio to receive one of two active doses of GLPG2222 (150 mg q.d. or 300 mg q.d.) or placebo q.d. administered for 29 days. Subjects will be in the study for a minimum of 6 weeks and a maximum of 10 weeks, from screening until the follow-up visit.
One F508del mutation on one allele in the CFTR gene, a gating (class III) mutation (one of the following: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R) on the 2nd allele in the
(FEV1) ≥ 40% of predicted normal for age, gender and height at screening