Clinical trials: Cystic Fibrosis
Cystic Fibrosis
Impact of Discontinuing Chronic Therapies in People With Cystic Fibrosis on Highly Effective CFTR Modulator Therapy
Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies in treating CF, it is still largely unknown whether or not other chronic therapies can be safely stopped.
- Trial name
- SIMPLIFY (NCT04378153)
- Where
- 80 sites in United States
- Volunteers
- 987 people
- Sponsor
- Nicole Hamblett
Cystic Fibrosis
Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years
This study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination therapy in CF subjects 2 through 5 years of age.
- Trial name
- NCT04537793
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 22 sites in United States, Australia and 3 more
- Volunteers
- 83 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Evaluation of VX-659/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-659, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF)…
- Trial name
- NCT03633526
- Testing
- VX-659/TEZ/IVA, IVA
- Where
- 6 sites in United States
- Volunteers
- 18 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Tobramycin Inhalation Powder (TIP) Administered Once Daily Continuously Versus TIP Administered BID in 28 Day on / 28 Day Off Cycles
To provide efficacy and safety data comparing two dosing schedules of Tobramycin Inhalation Powder (TIP) for the treatment of pulmonary Pseudomonas aeruginosa in patients with cystic fibrosis.
- Trial name
- NCT02015663
- Testing
- Tobramycin Inhalation Powder
- Where
- 21 sites in United States
- Volunteers
- 32 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygotes (for Cystic Fibrosis)
The purpose of this research study is to test a new combination of medicines, Phenylbutyrate and Genistein, to determine if they could be used to treat cystic fibrosis (CF).
- Trial name
- NCT00590538
- Testing
- Sodium 4-Phenylbutyrate, Genistein (Unconjugated Isoflavones 100)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Children's Hospital of Philadelphia
Cystic Fibrosis
Comparison of the Airway Clearance Efficacy of Electro Flo 5000 and the G5 Flimm-Fighter in Patients With CF
It is the goal of the proposed study to compare the efficacy, as assessed primarily by sputum weight, of these two different devices (the Electroflo 500 and the G5 Flimm-Fighter) for airway clearance (AC) in CF patients with mild…
- Trial name
- NCT02423447
- Testing
- Electro-Flo Intervention, G5 Intervention
- Volunteers
- 25 people
- Sponsor
- Stanford University
Cystic Fibrosis, Prediabetes
Prevention of Cystic Fibrosis Diabetes
Acute systemic hyperglycemia causes oxidative stress and a pro-inflammatory response. The pro-inflammatory cytokines induced by hyperglycemia are toxic to islet insulin producing cells, and thus worsen glucose intolerance.
- Trial name
- Prevent (NCT00967798)
- Testing
- Sitagliptin
- Where
- 3 sites in United States
- Volunteers
- 33 people
- Sponsor
- Emory University
Sinusitis, Cystic Fibrosis
Nasally Delivered Pulmozyme for Sinusitis in Cystic Fibrosis
Chronic sinusitis is a frequent complication in cystic fibrosis. The aim of this study is to determine whether Pulmozyme(dornase alfa) would maintain sinus health (compared to placebo) in patients with cystic fibrosis who have…
- Trial name
- NCT00416182
- Testing
- Pulmozyme (dornase alfa)
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of Vermont
Cystic Fibrosis
Inhaled Xylitol Versus Saline in Stable Subjects With Cystic Fibrosis
Cystic fibrosis (CF) lung disease is characterized by chronic bacterial colonization and recurrent infection of the airways. Lowering the airway surface liquid (ASL) salt concentration has been shown to increase activity of salt…
- Trial name
- NCT01355796
- Testing
- Xylitol, Hypertonic saline
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Joseph Zabner
Cystic Fibrosis
Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation
Exploratory proof of concept study to determine whether intranasal administration of QR-010 in subjects with cystic fibrosis, homozygous or compound heterozygous for the ΔF508 mutation, can increase the function of Cystic…
- Trial name
- NCT02564354
- Testing
- QR-010
- Where
- 5 sites in United States, Belgium and 1 more
- Volunteers
- 18 people
- Sponsor
- ProQR Therapeutics
Cystic Fibrosis
A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis
This is a Phase 2, randomized, double-blind, placebo- and tezacaftor/ivacaftor (TEZ/IVA)-controlled, parallel-group, 3-part, multicenter study designed to evaluate the safety and efficacy of VX-659 in triple combination (TC) with…
- Trial name
- NCT03224351
- Testing
- VX-659, TEZ/IVA, IVA
- Where
- 47 sites in United States, Ireland and 2 more
- Volunteers
- 124 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis, Cystic Fibrosis-related Diabetes
Semaglutide in CFRD
This open label, single arm pilot study, will examine the safety and tolerability of GLP-1RA semaglutide as an add-on therapy to insulin for overweight/obese adult patients with cystic fibrosis related diabetes (CFRD).
- Trial name
- NCT05788965
- Testing
- Semaglutide
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Minnesota
Cystic Fibrosis
A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)
The purpose of this study evaluates the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who were heterozygous for F508del and a minimal function mutation (F/MF participants).
- Trial name
- NCT05033080
- Testing
- VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA
- Where
- 139 sites in United States, Australia and 10 more
- Volunteers
- 435 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation
The purpose of this study is to evaluate the effect of ivacaftor (VX-770) on lung clearance index (LCI) in subjects aged 6 years and older with cystic fibrosis (CF) who have the G551D-CFTR mutation on at least 1 allele.
- Trial name
- NCT01262352
- Testing
- Ivacaftor
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 21 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109…
- Trial name
- NCT02544451
- Testing
- LUM/IVA
- Where
- 60 sites in United States, Australia and 7 more
- Volunteers
- 246 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in participants 1 to less than 2 years of age with cystic fibrosis (CF), homozygous for F508del (F/F).
- Trial name
- NCT03601637
- Testing
- LUM, IVA
- Where
- 27 sites in United States, Canada
- Volunteers
- 61 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
STeP IT UP CF: STimulating ImProved Health And Well-being In CysTic Fibrosis
STeP IT UP CF: STimulating ImProved Health And Well-being In CysTic Fibrosis Using Integration Of Fitness Technology and Port CF.
- Trial name
- NCT04018495
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Medical College of Wisconsin
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
Control of Steatorrhea in Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency
This study by Aptalis (formerly Axcan) assesses the efficacy and safety of Panzytrat® 25,000 compared to Kreon® 25,000 in the control of steatorrhea in participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency…
- Trial name
- NCT01327703
- Testing
- Panzytrat® 25,000, Kreon® 25,000
- Where
- 13 sites in Germany, Poland
- Volunteers
- 87 people
- Sponsor
- Forest Laboratories
Cystic Fibrosis
Phase 3 Study of Aztreonam for Inhalation Solution (AZLI) in a Continuous Alternating Therapy Regimen for the Treatment of Chronic Pseudomonas Aeruginosa Infection in Patients With CF
The primary objective of this study is to evaluate the safety and efficacy of a CAT regimen with aztreonam for inhalation solution (AZLI) and tobramycin inhalation solution (TIS) in adult and pediatric subjects with cystic…
- Trial name
- AZLI CAT (NCT01641822)
- Testing
- AZLI, Tobramycin inhalation solution
- Where
- 71 sites in United States
- Volunteers
- 107 people
- Sponsor
- Gilead Sciences
Cystic Fibrosis
Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations
The purpose of this study is to explore the combination of Ataluren and ivacaftor as a treatment for patients with a specific cystic fibrosis mutation
- Trial name
- NCT03256799
- Testing
- Ivacaftor/Ataluren
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Alabama at Birmingham