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

Phase 3

Completed

• Conditions: Cystic Fibrosis
• Interventions: Drug: LUM/IVA

• Registration Number: NCT04235140
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-01-15
• Study First Submitted QC: 2020-01-15
• Study First Posted: 2020-01-21
• Study Start: 2020-02-24
• Primary Completion: 2023-08-22
• Study Completion: 2023-08-22
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-20
• Last Update Posted: 2023-09-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 52

Inclusion Criteria

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

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

• Subjects 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
LUM/IVA	LUM/IVA	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.

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Day 1 up to Week 120

Secondary Outcome Measures

Name	Time	Method
Absolute Change in Sweat Chloride (SwCl)	From Baseline at Week 96	Sweat samples were collected using an approved collection device.

Trial Locations

Locations (23)

University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States

Arkansas Children's Hospital; 🇺🇸 Little Rock, Arkansas, United States

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

Yale New Haven Hospital; 🇺🇸 New Haven, Connecticut, United States

Children's Healthcare of Atlanta; 🇺🇸 Atlanta, Georgia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

Riley Hospital for Children at Indiana University Health; 🇺🇸 Indianapolis, Indiana, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States

The Children's Mercy Hospital; 🇺🇸 Kansas City, Missouri, United States

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