Clinical trials: Cystic Fibrosis
Cystic Fibrosis
Rollover Study of VX-770 in Cystic Fibrosis Subjects
The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF).
- Trial name
- NCT01117012
- Testing
- Ivacaftor
- Where
- 58 sites in United States, Australia and 6 more
- Volunteers
- 192 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis (CF)
Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of SION-719 When Added to Trikafta
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of SION-719 when given to people with CF who are already taking Trikafta.
- Trial name
- PreciSION CF (NCT07108153)
- Testing
- SION-719
- Where
- 13 sites in United States, Australia
- Volunteers
- 15 people
- Sponsor
- Sionna Therapeutics Inc.
Cystic Fibrosis
Cystic Fibrosis (CF) Flow Rates Study
We wish to measure the inspiratory flow and volumes generated by the subjects inhaling from a spirometer with a high resistance dry powder inhaler in series in subjects with cystic fibrosis.
- Trial name
- NCT00779077
- Volunteers
- 25 people
- Sponsor
- Syntara
Cystic Fibrosis
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation
The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 6 to 11 years who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)…
- Trial name
- ENVISION (NCT00909727)
- Testing
- Ivacaftor
- Where
- 29 sites in United States, Australia and 5 more
- Volunteers
- 52 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Study to Assess the Safety and PK of Oral and IV Xenleta in Adults With Cystic Fibrosis
This study is intended to assess the pharmacokinetic (PK) and safety of a single dose of IV and oral formulations of lefamulin in adults with cystic fibrosis (CF).
- Trial name
- NCT05225805
- Testing
- Lefamulin
- Where
- 6 sites in United States
- Volunteers
- 13 people
- Sponsor
- Nabriva Therapeutics AG
Cystic Fibrosis
A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del
This study will explore the impact of lumacaftor/ivacaftor (LUM/IVA) on disease progression in subjects aged 2 through 5 years with cystic fibrosis (CF), homozygous for F508del (F/F).
- Trial name
- NCT03625466
- Testing
- LUM/IVA
- Where
- 5 sites in Germany
- Volunteers
- 51 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Mannitol Dose Response Study in Cystic Fibrosis
Many cystic fibrosis patients die of lung failure caused by repeated lung infections from thick, sticky mucus. Past studies have shown Bronchitol inhalation may help to facilitate the clearance of mucus by altering its rheology…
- Trial name
- NCT00251056
- Testing
- mannitol
- Where
- 12 sites in Argentina, Canada
- Volunteers
- 48 people
- Sponsor
- Syntara
Cystic Fibrosis
A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
This study will evaluate the efficacy, safety and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for…
- Trial name
- NCT04058353
- Testing
- ELX/TEZ/IVA, IVA, TEZ/IVA
- Where
- 93 sites in United States, Australia and 11 more
- Volunteers
- 271 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis, Mucociliary Clearance
A Randomized, Double-Blind, Placebo-Controlled Multiple Dose Trial of GS-9411 in Healthy Volunteers
The purpose of this study is to evaluate the safety and tolerability of repeated doses of GS-9411 in healthy volunteers. GS-9411 is a sodium channel inhibitor, that may restore airway hydration and mucociliary clearance in the…
- Conditions
- Cystic Fibrosis, Mucociliary Clearance, Airway Hydration
- Trial name
- NCT00999531
- Testing
- GS-9411
- Where
- Australia
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Gilead Sciences
Cystic Fibrosis
A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR Mutation
This is a Phase 3, multicenter, open-label, 3-part rollover study in subjects with CF who are homozygous or heterozygous for the F508del-CFTR mutation and who participated in studies VX13-661-103 (Study 103, NCT02070744)…
- Trial name
- NCT02565914
- Testing
- TEZ/IVA, IVA
- Where
- 154 sites in United States, Australia and 14 more
- Volunteers
- 1,131 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Exocrine Pancreatic Insufficiency, Pancreatic Diseases
Special Investigation of LipaCreon on Long-term Use in Patients With Pancreatic Exocrine Insufficiency
This study aims at collecting the information related to the safety and effectiveness in the pancreatic exocrine insufficiency patients receiving the treatment with LipaCreon for a long term in order to evaluate the effective and…
- Conditions
- Exocrine Pancreatic Insufficiency, Pancreatic Diseases, Digestive System Diseases
- Trial name
- NCT01427725
- Testing
- Lipacreon
- Where
- 189 sites in Japan
- Volunteers
- 579 people
- Sponsor
- Mylan Inc.
Cystic Fibrosis
A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF
This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.
- Trial name
- NCT06237335
- Testing
- RCT2100, Ivacaftor
- Where
- 23 sites in United States, France and 3 more
- Volunteers
- 128 people, healthy volunteers welcome
- Sponsor
- ReCode Therapeutics
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Cystic Fibrosis
Pro-resolving Effect of MAG-DHA in Cystic Fibrosis (PREMDIC)
Monoglyceride of DHA (DHA-MAG) is a lipid compound for which intestinal absorption would increase the ratio DHA / arachidonic acid (AA) and promote the synthesis of specific metabolites involved in the resolution of inflammation.
- Trial name
- PREMDIC (NCT02518672)
- Testing
- MAG-DHA
- Where
- Canada
- Volunteers
- 11 people
- Sponsor
- SCF Pharma
Non-cystic Fibrosis Bronchiectasis
Dose-finding Study to Assess the Efficacy, Safety and Tolerability of Tobramycin Inhalation Powder in Patients With Non-Cystic Fibrosis Bronchiectasis and Pulmonary P. Aeruginosa Infection
The purpose of this study was to support the selection of a safe and tolerable tobramycin inhalation powder (TIP) dose, and regimen that exhibits effective bacterial reduction of P.
- Trial name
- iBEST-1 (NCT02712983)
- Testing
- TIP
- Where
- 34 sites in Belgium, France and 4 more
- Volunteers
- 107 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis, CFTR Gene Mutation
Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis
ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy or are not taking CFTR modulators due to drug intolerance, poor…
- Trial name
- LunairCF (NCT06747858)
- Testing
- ARCT-032
- Where
- 13 sites in United States
- Volunteers
- 33 people
- Sponsor
- Arcturus Therapeutics, Inc.
Chronic Rhinosinusitis, Cystic Fibrosis
Efficacy of Nasal Irrigation With Respimer® Netiflow® vs Saline Among Patients With Cystic Fibrosis and Chronic Rhinosinusitis
Prospective, randomized, cross-over, multicenter, trial comparing the efficacy and tolerability of nasal irrigation with Respimer® mineral salts solution versus saline solution both administered with Respimer® Netiflow® medical…
- Trial name
- Respire (NCT03145051)
- Testing
- Saline solution, Respimer Netiflow
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Laboratoire de la Mer
Cystic Fibrosis
A Study Looking at the Safety, Tolerability and Efficacy of the Combination of the Study Drugs GLPG2451 and GLPG2222 With or Without GLPG2737 in Patients With Cystic Fibrosis.
This is a Phase Ib, multi-center, open-label, nonrandomized multiple cohorts study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses of a combination treatment of GLPG2451 and GLPG2222…
- Trial name
- FALCON (NCT03540524)
- Testing
- GLPG2451 dose regimen A, GLPG2451 dose regimen B, GLPG2222
- Where
- 20 sites in Belgium, Bulgaria and 6 more
- Volunteers
- 10 people
- Sponsor
- Lakefront Biotherapeutics NV
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency
The purpose of this study is to evaluate the efficacy in patients with cystic fibrosis and pancreatic insufficiency following treatment with BSSL
- Trial name
- NCT00743483
- Testing
- rhBSSL
- Where
- 4 sites in Netherlands, Poland
- Volunteers
- 15 people
- Sponsor
- Swedish Orphan Biovitrum
Cystic Fibrosis
A Study to Determine the Variability of a 6-Minute Walk Test in Cystic Fibrosis Subjects With Advanced Lung Disease
This is an observational study to be conducted at approximately 25 sites in the United States. Approximately 25 subjects with severe obstructive CF lung disease (FVC <40% predicted) will be enrolled.
- Trial name
- NCT00311506
- Volunteers
- 25 people
- Sponsor
- Genentech, Inc.