A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis

Vertex Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: IVA; Drug: Placebo; Drug: TEZ/IVA

Study type: Interventional

Funder types: Industry

Identifiers

NCT03559062

2016-004479-35 (EudraCT Number)

VX16-661-115

Details and patient eligibility

About

This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).

Enrollment: 67 patients

Sex: All

Ages: 6 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
• Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
• Participants with a screening LCI2.5 result ≥7.5.
• Participants who are able to swallow tablets.

Key Exclusion Criteria:

• Clinically significant cirrhosis with or without portal hypertension.
• Colonization with organisms associated with a more rapid decline in pulmonary status.
• Solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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67 participants in 3 patient groups

Placebo

Other group

Description:

Participants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks.

Treatment:

Drug: Placebo

Drug: Placebo

TEZ/IVA

Experimental group

Description:

Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.

Treatment:

Drug: Placebo

Drug: Placebo

Drug: TEZ/IVA

Drug: IVA

Ivacaftor

Experimental group

Description:

Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks.

Treatment:

Drug: Placebo

Drug: Placebo

Drug: IVA