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A Study of the effects of Lumacaftor/Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

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

Status
Unknown
Phases
Phase 3
Study type
Interventional
Source
EU CTR
Registry ID
EUCTR2023-000946-41-Outside-EU/EEA
Enrollment
Unknown
Registered
2023-10-17
Start date
Unknown
Completion date
Unknown
Last updated
2023-10-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis

Interventions

Product Name: lumacaftor/ivacaftor Product Code: VX-809/VX-770 Pharmaceutical Form: Granules INN or Proposed INN: Lumacaftor CAS Number: 936727-05-8 Current Sponsor code: VX-809 Concentration unit: mg

Sponsors

Vertex Pharmaceuticals Incorporated
Lead Sponsor

Eligibility

Sex/Gender
All

Inclusion criteria

Inclusion criteria: 1.Subjects From Study VX16-809-122 Part B (Study 122) - Completed the 24-week Treatment Period and the Safety Followup Visit in Study 122B 2.Subjects Not From Study 122 - Subjects will be 1 to less than 2 years of age - Homozygous for the F508del mutation (F/F) Are the trial subjects under 18? yes Number of subjects for this age range: 52 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1.Any clinically significant laboratory abnormalities that would interfere with the study assessments or pose an undue risk for the subject 2. Solid organ or hematological transplantation

Design outcomes

Primary

MeasureTime frame
Main Objective: To evaluate the safety and tolerability of long-term lumacaftor/ivacaftor (LUM/IVA) treatment in subjects with cystic fibrosis (CF), who are homozygous for F508del and 12 to <24 months of age at treatment initiation;Secondary Objective: To evaluate the pharmacodynamics (PD) of long-term LUM/IVA treatment in subjects with CF, who are homozygous for F508del and 12 to <24 months of age at treatment initiation;Primary end point(s): Safety and tolerability as assessed by the number of adverse events (AEs) and serious adverse events (SAEs);Timepoint(s) of evaluation of this end point: Up to 120 weeks—

Secondary

MeasureTime frame
Secondary end point(s): Absolute change in sweat chloride;Timepoint(s) of evaluation of this end point: From Baseline at Week 96—

Countries

Canada, United States

Contacts

Public ContactVertex Pharmaceuticals Incorporate

Vertex Pharmaceuticals Incorporated

medicalinfo@vrtx.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026