Phase 3 Completed N=121 Randomized Quadruple-blind Treatment

A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes

Cystic fibrosis

Source: ClinicalTrials.gov NCT04353817 ↗

Enrolled (actual)

121

Serious AEs

10.7%

Results posted

Jul 2022

Primary outcomePrimary: Absolute Change in Lung Clearance Index 2.5 (LCI2.5) — -0.02; -2.29 index — p=<0.0001

◆ Published Evidence

No publication linked

No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.

Summary

This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes).

Outcome Measures

Outcome	Result	p-value
PRIMARY Absolute Change in Lung Clearance Index 2.5 (LCI2.5)	-0.02; -2.29	<0.0001 sig
SECONDARY Absolute Change in Sweat Chloride (SwCl)	-0.9; -52.1	<0.0001 sig
SECONDARY Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	57; 48; 9; 4	—

Eligibility Criteria

Key Inclusion Criteria

Heterozygous for the F508del mutation (F/MF)
Forced expiratory volume in 1 second (FEV1) value greater than equal to(≥) 70%

Key Exclusion Criteria

Clinically significant cirrhosis with or without portal hypertension
Lung infection with organisms associated with a more rapid decline in pulmonary status
Solid organ or hematological transplantation

Other protocol defined Inclusion/Exclusion criteria may apply

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04353817). 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. Informational only — not medical advice.