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A placebo-controlled trial of insulin therapy with or without adjuvant metformin in patients with Cystic Fibrosis-Related Diabetes

Investigate the effect of adjuvant metformin therapy on insulin need and on glycaemic control in CFRD patients.

Long Term Safety and Tolerability Study of Open-Label Liposomal Amikacin for Inhalation (Arikace*) in Cystic Fibrosis Patients with Chronic Infection due to Pseudomonas aeruginosa

To evaluate the long-term safety, tolerability and efficacy of ArikaceTM 590 mg for a maximum 12 cycles administered once daily, where each cycle consists of 28 days on treatment followed by 28 days off treatment.

A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis

The primary objective of this Phase 3 extension study will be to obtain essential long term safety data to augment the overall safety database. The secondary objective will be to augment the efficacy information collected in the double-blind study (…

Long Term Administration of Inhaled Mannitol in Cystic Fibrosis - A Safety and Efficacy Study

The purpose of this study is to examine the efficacy and safety of 26 weeks treatment with inhaled mannitol in subjects with cystic fibrosis. Previous studies with inhaled mannitol have demonstrated improvements in lung function, mucociliary…

A Phase 3b Open-label Study to Assess the Effect of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance in Cystic Fibrosis Subjects with Abnormal Glucose Metabolism

To evaluate the effect of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) on glucose tolerance in CF subjects with impaired glucose tolerance (IGT) or CF related diabetes (CFRD)

A Phase 3 Double-blind, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of ELX/TEZ/IVA in Cystic Fibrosis Subjects 6 Years of Age and Older With a Non-F508del ELX/TEZ/IVA-responsive CFTR Mutation

To evaluate the efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA

A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes

This study has been transitioned to CTIS with ID 2024-515637-14-00 check the CTIS register for the current data. To evaluate the long-term safety and tolerability of elexacaftor(ELX)/tezacaftor (TEZ)/ivacaftor (IVA)

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

This study has been transitioned to CTIS with ID 2024-514173-22-00 check the CTIS register for the current data. Primary ObjectiveTo evaluate the long-term safety and tolerability of VNZ/TEZ/D-IVA in subjects with CFSecondary ObjectiveTo evaluate…

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121
Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for F508del,
Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive CFTR Mutation and No F508del Mutation

To evaluate the efficacy of VX-121/TEZ/D-IVA in CF subjects who are homozygous forF508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, orhave at least 1 other TCR CFTR mutation and no F508del mutation

A Phase 3b, Randomized, Placebo-Controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

Primary Objective:To evaluate the efficacy of ELX/TEZ/IVA in subjects 6 through 11 years of age with CF, heterozygous for F508del and a MF mutation (F/MF)Secondary Objectives:• To evaluate the PD of ELX/TEZ/IVA• To evaluate the safety of ELX/TEZ/IVA

Randomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization

To evaluate the safety and efficacy of a 14-day course vs a 28 day course of AZLI 75 mg three times a day (TID) in subjects with new onset PA respiratory tract colonization/infection as determined by PA eradication over a 28-day post treatment…