Safety and Efficacy of Recombinant Adeno-Associated Virus Containing the CFTR Gene in the Treatment of Cystic Fibrosis

PTC124 for Cystic Fibrosis

NCT00234663 ·Status: COMPLETED ·Phase: PHASE2

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation

NCT00909532 ·Status: COMPLETED ·Phase: PHASE3

A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation

NCT02742519 ·Status: TERMINATED ·Phase: PHASE3

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)

NCT05668741 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion

NCT02070744 ·Status: COMPLETED ·Phase: PHASE2

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation

NCT00953706 ·Status: TERMINATED ·Phase: PHASE2

A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis

NCT03224351 ·Status: COMPLETED ·Phase: PHASE2

A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation

NCT03068312 ·Status: COMPLETED ·Phase: PHASE3

A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation

NCT02875366 ·Status: COMPLETED ·Phase: PHASE4

Surrogate Markers of Response to New Therapies in Cystic Fibrosis Patients

NCT02965326 ·Status: RECRUITING ·Phase: NA

Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations

NCT02971839 ·Status: TERMINATED ·Phase: PHASE2

A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation

NCT01807923 ·Status: COMPLETED ·Phase: PHASE3

A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF

NCT06237335 ·Status: RECRUITING ·Phase: PHASE2

4D-710 in Adult Patients With Cystic Fibrosis

NCT05248230 ·Status: RECRUITING ·Phase: PHASE2

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

NCT03625466 ·Status: COMPLETED ·Phase: PHASE2

A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation

NCT02516410 ·Status: COMPLETED ·Phase: PHASE3

Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis

NCT03451045 ·Status: COMPLETED ·Phase: PHASE2

Phase I Study of the Third Generation Adenovirus H5.001CBCFTR in Patients With Cystic Fibrosis

NCT00004287 ·Status: COMPLETED ·Phase: PHASE1

Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation

NCT00865904 ·Status: COMPLETED ·Phase: PHASE2

Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function

NCT01685801 ·Status: COMPLETED ·Phase: PHASE2

A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation

NCT01807949 ·Status: COMPLETED ·Phase: PHASE3

A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation

NCT02514473 ·Status: COMPLETED ·Phase: PHASE3

Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)

NCT02323100 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation

NCT01225211 ·Status: COMPLETED ·Phase: PHASE2

A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

NCT02508207 ·Status: COMPLETED ·Phase: PHASE2