Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del

Vertex Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: LUM; Drug: IVA

Study type: Interventional

Funder types: Industry

Identifiers

NCT03601637

VX16-809-122

2017-004794-13 (EudraCT Number)

Details and patient eligibility

About

This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in participants 1 to less than 2 years of age with cystic fibrosis (CF), homozygous for F508del (F/F).

Enrollment: 61 patients

Sex: All

Ages: 12 to 23 months old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Participants will be 1 to less than 2 years of age on day 1 of the relevant part of the study
• Homozygous for F508del (F/F)

Key Exclusion Criteria:

• Any clinically significant laboratory abnormalities at the screening visit that would interfere with the study assessments or pose an undue risk for the participants
• Solid organ or hematological transplantation

Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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61 participants in 2 patient groups

Part A: LUM/IVA

Experimental group

Description:

Participants weighing 7 to less than (\<)10 kilograms (kg) at screening received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to \<14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants weighing greater than or equal to (\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.

Treatment:

Drug: IVA

Drug: LUM

Part B: LUM/IVA

Experimental group

Description:

Participants weighing 7 to \<9 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.

Treatment:

Drug: IVA

Drug: LUM