Last updated on November 2019

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


Brief description of 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 in patients with CF with at least one G542X allele.

In total, up to eight patients will be enrolled in the trial; up to 4 patients will be homozygotes to G542X, and the remaining patients will be compound heterozygotes with G542X and with any Class 1 or Class 2 mutation, excluding F508del.

Each patient will receive 4 escalating doses as follows:

  • 0.3 mg/kg per day SC
  • 0.75 mg/kg per day SC
  • 1.5 mg/kg per day SC
  • An individualized dose, 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.

Clinical Study Identifier: NCT04135495

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Boston Children's Hospital

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