Skip to main content
Clinical Trials/NCT04235140
NCT04235140CompletedPhase 3

A Phase 3, Open-label, and Rollover Study to Evaluate the Long-term Safety and Tolerability of Lumacaftor/Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

Vertex Pharmaceuticals Incorporated23 sites in 2 countries52 target enrollmentStarted: February 24, 2020Last updated:
Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 3
Status
Completed
Enrollment
52
Locations
23
Primary Endpoint
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Study Overview

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.

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
12 Months to — (Child, Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

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)

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.

Arms & Interventions

LUM/IVA

Experimental

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.

Intervention: LUM/IVA (Drug)

Outcomes

Primary Outcomes

Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Time Frame: Day 1 up to Week 120

Secondary Outcomes

  • Absolute Change in Sweat Chloride (SwCl)(From Baseline at Week 96)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (23)

Loading locations...

Similar Trials