Long-term Safety of Lumacaftor/Ivacaftor in Participants With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

Vertex Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: LUM/IVA

Study type: Interventional

Funder types: Industry

Identifiers

NCT04235140

VX19-809-124

Details and patient eligibility

About

This is a Phase 3, multicenter, open-label and roll-over study in participants who are 12 to <24 months of age at initiation of Lumacaftor/Ivacaftor (LUM/IVA) treatment.

Enrollment: 52 patients

Sex: All

Ages: 12+ months old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Participants From Study VX16-809-122 Part B (Study 122)

  • Completed the 24-week Treatment Period and the Safety Follow-up Visit in Study 122B

• Participants Not From Study 122

  • Subjects will be 1 to less than 2 years of age
  • Homozygous for the F508del mutation (F/F)

Key Exclusion Criteria:

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

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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52 participants in 1 patient group

LUM/IVA

Experimental group

Description:

Participants weighing 7 to less than (\<) 9 kilograms (kg) received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg q12h in the treatment period of 96 weeks. Participants weighing greater than or equal to (\>=)14 kg received LUM 150 mg/IVA 188 mg FDC q12h in the treatment period of 96 weeks.

Treatment:

Drug: LUM/IVA