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Study identifier: NCT04135495 Synced from ClinicalTrials.gov · July 28, 2026
● Completed

A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X Allele

Condition: Cystic Fibrosis  ·  Sponsor: Eloxx Pharmaceuticals, Inc.

PhasePhase 2
Planned participants17
Who can joinAll sexes, 18 Years to no upper limit
Healthy volunteersNo

About this study

This is a Phase 2 open-label, dose-escalation study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 with and without ivacaftor in patients with CF with at least one G542X allele. In total, up to 16 patients will be enrolled in the trial; up to 4 patients will be homozygotes for G542X, and the remaining patients will be compound heterozygotes with one G542X or phenotypically similar nonsense allele and any Class 1 or Class 2 mutation. Each patient will receive up to 5 escalating doses as follows: * ELX-02 0.3 mg/kg per day SC * ELX-02 0.75 mg/kg per day SC * ELX-02 1.5 mg/kg per day SC * An individualized dose of ELX-02, as high as 3.0 mg/kg per day SC, based on the patients observed safety and tolerability, PK at previous doses and the results of laboratory tests. * ELX-02 1.5 mg/kg per day SC plus 150 mg ivacaftor every 12 bid

This description comes directly from the study's public registry record.

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Contact details are listed per location below or on the registry record.

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Locations (10)

Long Beach MemorialLong Beach, California, United States
Stanford School of MedicinePalo Alto, California, United States
National Jewish HealthDenver, Colorado, United States
Johns HopkinsBaltimore, Maryland, United States
Boston Children's HospitalBoston, Massachusetts, United States
Nationwide Children's HospitalColumbus, Ohio, United States
Baylor College of MedicineHouston, Texas, United States
Foothills Hospital Calgary (University of Calgary)Calgary, Alberta, Canada
St. Michael's HospitalToronto, Ontario, Canada
The University of Montreal Health CentreMontreal, Quebec, Canada

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Source record: clinicaltrials.gov/study/NCT04135495