**Acknowledgements**

**Molecule Title of Study ClinicalTrials.gov Identifier**

NCT02039986

NCT01784419

NCT02194881

NCT02392234

NCT02412111

NCT02390219

NCT01937325

NCT02347657

NCT02310789

NCT02139306

NCT02275936

NCT02323100

NCT02359357

Ivacaftor (Kalydeco) and Insulin in Cystic

Ivacaftor in French Patients with Cystic Fibrosis and a G551D Mutation

Safety of Ivacaftor and VX-661 in

Clinically Responsive to Ivacaftor

Short Term Effects of Ivacaftor in Non-G551D

A Phase 3 Study to Evaluate the Efficacy and

Combination with Ivacaftor in Subjects Aged 12 Years and Older with Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation

A Phase 3 Study of VX-661 in Combination with Ivacaftor in Subjects Aged 12 Years and Older with Cystic Fibrosis, Who Have One F508del-CFTR Mutation and a Second Mutation that Has Been Demonstrated to Be

Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older with Advanced Lung Disease

Airway Infection, Inflammatory Markers and Exercise Capacity in Patients with Cystic Fibrosis and at Least One G551D Mutation Taking VX-770 (Ivacaftor) (Phase IV)

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination with Ivacaftor (Phase III)

(Study: Vertex IIS) Does Ivacaftor Alter Wild Type CFTR-Open Probability in the Sweat

Gland Secretory Coil?

Mutation (SNO4) (Phase I)

Cystic Fibrosis (ACT CF), Phase III

Study of N91115 in Patients with Cystic Fibrosis Homozygous F508del-CFTR

FDL169 in Healthy Volunteers (Phase I)

Ataluren(PTC124) Study of Ataluren in Nonsense Mutation

Glycerol Phenylbutyrate Glycerol Phenylbutyrate Corrector Therapy For CF (GPBA)

**Table 2.** Current open clinical trials on CFTR modulators (source ClinicalTrials.gov).

FDL169 FTIH – Single and Repeat Oral Doses of

Fibrosis (CF)

(IVACAFTOR1)

(Phase III)

Cystic Fibrosis Patients

Ivacaftor(VX770; Kalydeco)

318 Cystic Fibrosis in the Light of New Research

N91115

This work was supported by contributions from Fondazione Cariparo (Cassa di Risparmio di Padova e Rovigo) to RG, Italian Cystic Fibrosis Research Foundation (FFC#2/2010 and FFC#1/2012) to M.B and (FFC#17/2010 and FFC#8/2014) to RG.

Our thanks to Dr. Amanda J Neville MSB for her comments and corrections of the English text.
