Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older

Vertex Pharmaceuticals

Status and phase: Invitation-only; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: VNZ/TEZ/D-IVA

Study type: Interventional

Funder types: Industry

Identifiers

NCT05844449

2022-503081-74-00 (Other Identifier)

VX22-121-106

Details and patient eligibility

About

The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).

Enrollment: 174 estimated patients

Sex: All

Ages: 1+ year old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

Parts A and B:

• Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222)

Part B:

-Meets at least 1 of the following criteria:

• Completed study drug treatment in Part A
• Had study drug interruption(s) in Part A, but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the treatment period of Part A

Key Exclusion Criteria:

• Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that might pose an additional risk in administering study drug
• History of solid organ, hematological transplantation, or cancer
• History of drug intolerance in the parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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174 participants in 1 patient group

VNZ/TEZ/D-IVA

Experimental group

Description:

Part A: Participants (Cohort 1: 6 through 11 years of age (inclusive); Cohort 2: 2 through 5 years of age (inclusive) and Cohort 3: 1 to less than (\<) 2 years of age) will receive VNZ/TEZ/D-IVA once daily at doses determined based on their age and weight. The age range is based on date of informed consent in parent study. Part B: Participants in Cohort 1 who meet the eligibility criteria will receive VNZ/TEZ/D-IVA for an additional 96 weeks.

Treatment:

Drug: VNZ/TEZ/D-IVA