Clinical trials: Cystic Fibrosis (CF)
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
A Study to Evaluate the Mode of Administration and Safety of EUR-1008 (APT-1008) in Infants 1 to 12 Months of Age
A study to determine the safety, effectiveness, and acceptability of 2 methods of administration of EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) 3,000 lipase units capsule, a pancreatic enzyme product…
- Trial name
- NCT01100606
- Testing
- EUR-1008 (APT-1008)
- Where
- 6 sites in United States
- Volunteers
- 15 people
- Sponsor
- Forest Laboratories
Cystic Fibrosis
Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation
The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis (CF) who have a non-G551D cystic fibrosis transmembrane regulator (CFTR) gating mutation (any one of the following…
- Trial name
- KONNECTION (NCT01614470)
- Testing
- Ivacaftor
- Where
- 12 sites in United States, Belgium and 1 more
- Volunteers
- 39 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Pulmonary Infections, Pseudomonas Aeruginosa
Second Open Label Extension to Bridging Study CTBM100C2303
This was an open-label, single arm (uncontrolled) study in participants suffering from cystic fibrosis, who have completed their study participation in CTBM100C2303 and extension study one CTBM100C2303E1 (all visits), who were…
- Trial name
- NCT01069705
- Testing
- Tobramycin inhalation powder
- Where
- 17 sites in Bulgaria, Estonia and 5 more
- Volunteers
- 49 people
- Sponsor
- Novartis Pharmaceuticals
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 Lung, Respiratory Infections, Recurrent, Chronic
A Double-Blind, Active-Controlled, Multiple-Ascending Dose Study of Aerosolized RSP-1502 in Subjects With CF and Chronic PA Lung Infection
A double-blind, active-controlled, multiple-ascending dose, safety study of aerosolized RSP-1502 in subjects with cystic fibrosis Pseudomonas aeruginosa lung infection.
- Conditions
- Cystic Fibrosis Lung, Respiratory Infections, Recurrent, Chronic, Pseudomonas Aeruginosa
- Trial name
- NCT06016088
- Testing
- RSP-1502, Tobramycin inhalation solution
- Where
- 22 sites in United States, Australia
- Volunteers
- 71 people
- Sponsor
- Respirion Pharmaceuticals Pty Ltd
Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis Patients
Randomized, Controlled Study of CF Patients Between 3 Months and Less Than 7 Years
This study investigated the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis.
- Trial name
- EARLY (NCT01082367)
- Testing
- TOBI
- Where
- 19 sites in Canada, Egypt and 8 more
- Volunteers
- 50 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
PR-018: An Open-Label, Safety Extension of Study PR-011
A study to evaluate long term safety and effect on ability to thrive of EUR-1008 (APT-1008) 3,000 lipase units (Zenpep® [pancrelipase] delayed release capsules) in infants with exocrine pancreatic insufficiency (EPI) due to…
- Trial name
- NCT01131507
- Testing
- EUR-1008 (APT-1008)
- Where
- 6 sites in United States
- Volunteers
- 15 people
- Sponsor
- Forest Laboratories
Cystic Fibrosis
A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F).
- Trial name
- NCT03460990
- Testing
- VX-659/TEZ/IVA, TEZ/IVA, IVA
- Where
- 46 sites in United States, Australia and 4 more
- Volunteers
- 116 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis, Pancreatic Insufficiency
Efficacy and Safety of Ultrase® MT20 in Improving the Coefficient of Fat Absorption (CFA) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI)
This is a Phase III, multicenter, open-label study, that will evaluate the improvement of nutrient absorption when participants will receive Ultrase® MT20. This study is sponsored by Aptalis (formerly Axcan).
- Trial name
- NCT00513682
- Testing
- Ultrase® MT20
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Forest Laboratories
Cystic Fibrosis
A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)
This is a Phase 1/2 multicenter, open-label, single dose trial of SP-101 investigational gene therapy in adults with CF who are ineligible for or intolerant to CFTR modulator therapy.
- Trial name
- SAAVe (NCT06526923)
- Testing
- SP-101 and doxorubicin Cohort 1, SP-101 and doxorubicin Cohort 2
- Where
- 4 sites in United States
- Volunteers
- 15 people
- Sponsor
- Spirovant Sciences, Inc.
Cystic Fibrosis
Safety Study of Ivacaftor in Subjects With Cystic Fibrosis
The purpose of this study was to evaluate the safety and tolerability of ivacaftor in patients with cystic fibrosis (CF) who were aged 18 years or older and have a G551D mutation in the cystic fibrosis transmembrane conductance…
- Trial name
- NCT00457821
- Testing
- Ivacaftor 25 mg/75 mg, Ivacaftor 75 mg/150 mg, Ivacaftor 150 mg or 250 mg
- Where
- 15 sites in United States, Canada and 1 more
- Volunteers
- 39 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis, Pseudomonas Aeruginosa
Safety of AZLI in Children With Cystic Fibrosis (CF) and Chronic Pseudomonas Aeruginosa in the Lower Airways
This was an open-label, multicenter study in children ≤ 12 years of age with cystic fibrosis (CF) and chronic Pseudomonas aeruginosa (PA) infection in the lower airways using three 28-day courses of Aztreonam for Inhalation…
- Trial name
- PALS (NCT01404234)
- Testing
- AZLI
- Where
- 29 sites in United States, France and 4 more
- Volunteers
- 61 people
- Sponsor
- Gilead Sciences
Cystic Fibrosis
Phase 1b Safety, Tolerability, and PK Study to Assess GS-5737 in Subjects With CF
This study will determine the safety, tolerability, and pharmacokinetics of a single dose of GS-5737 administered with a 2.8% saline solution vehicle in adult subjects with CF.
- Trial name
- NCT01840735
- Testing
- GS-5737
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Gilead Sciences
Cystic Fibrosis
Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis
This Phase 1/2, first-in-human study evaluated the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization to the respiratory tract of adult subjects with cystic fibrosis (CF).
- Trial name
- RESTORE-CF (NCT03375047)
- Testing
- MRT5005, Normal saline
- Where
- 16 sites in United States
- Volunteers
- 42 people
- Sponsor
- Sanofi
Cystic Fibrosis
A Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease (TOPIC)
This was a multicenter, randomized, double-blind, placebo-controlled study of patients with severe, though stable, cystic fibrosis (CF) whose routine treatment included Pulmozyme.
- Trial name
- NCT00434278
- Testing
- Dornase alfa
- Where
- 40 sites in United States
- Volunteers
- 27 people
- Sponsor
- Genentech, Inc.
Cystic Fibrosis
PTC124 for the Treatment of Cystic Fibrosis
In some patients with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein.
- Trial name
- NCT00351078
- Testing
- PTC124
- Where
- Israel
- Volunteers
- 19 people
- Sponsor
- PTC Therapeutics
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, Non-Cystic Fibrosis Bronchiectasis
A Clinical Study to Investigate Safety, Tolerability and Distribution of CHF 6333 After One or After Repeated Inhalation in Patients With Cystic Fibrosis (CF) and in Patients With Non Cystic Fibrosis (NCFB) Bronchiectasis
CHF 6333 is a medicinal product on development for the treatment of cystic fibrosis and non-CF bronchiectasis and undergoing clinical testing. It has not yet been approved by the authorities for the treatment of these diseases.
- Trial name
- NCT04010799
- Testing
- CHF 6333
- Where
- Germany
- Volunteers
- 68 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Cystic Fibrosis
Deposition of Inhaled Prolastin in Cystic Fibrosis Patients
The objective of this trial is to determine the optimal region of the lung for depositing Prolastin (alpha-1 antitrypsin; AAT) by inhalation in order to treat cystic fibrosis (CF).
- Trial name
- CF2 (NCT00486837)
- Testing
- Alpha1-Proteinase Inhibitor (Human)
- Volunteers
- 72 people
- Sponsor
- Grifols Therapeutics LLC
Cystic Fibrosis (CF)
Study to Evaluate Adverse Events and Change in Disease Activity With Oral Capsules of Galicaftor/Navocaftor/ABBV-119 or Galicaftor/Navocaftor/ABBV-576 Combination Therapies in Adult Participants With Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-threatening, genetic disease that affects the lungs and digestive system, significantly impairing the quality of life, with those affected having a median age of death at 40.
- Trial name
- NCT04853368
- Testing
- ABBV-576, Galicaftor, Navocaftor
- Where
- 41 sites in United States, Australia and 5 more
- Volunteers
- 48 people
- Sponsor
- AbbVie