Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)

Vertex Pharmaceuticals

Status: Completed

Conditions: Cystic Fibrosis

Treatments: Drug: ivacaftor

Study type: Observational

Funder types: Industry

Identifiers

NCT02445053

VX14-770-116

2014-002704-24 (EudraCT Number)

Details and patient eligibility

About

To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D).

Enrollment: 75 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female with confirmed diagnosis of CF16
• At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D
• Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent
• Signed ICFs and, where appropriate, signed Assent Form
• Able to understand the study requirements and comply with study data collection procedures

Exclusion criteria

• Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment
• Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation
• History of organ transplantation