Phase 3 N=52 Treatment

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

Cystic Fibrosis

Bottom Line

View on ClinicalTrials.gov: NCT04235140 ↗

Enrolled (actual)

Serious AEs

23.1%

Results posted

Sep 2024

Primary outcome: Primary: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 52; 12 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: LUM/IVA (Drug)
Age: Pediatric, Adult, Older Adult · 0+ yrs
Sex: All
Sponsor: Vertex Pharmaceuticals Incorporated
Primary completion: Aug 2023

Outcome Measures

Outcome	Result	p-value
PRIMARY Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	52; 12	—
SECONDARY Absolute Change in Sweat Chloride (SwCl)	-21.0	—

Summary

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.

Eligibility 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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04235140). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.