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

• Status: Completed
• Trial end date: 2020-10-01
• Target enrollment: 0
• Participant gender: All

Summary

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).

• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated
• Treatments: Ivacaftor

Criteria

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