A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele

2019-10-21 12:45:41 | BioPortfolio


This is a Phase 2 open label 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.

Up to 16 patients will be enrolled in the trial; a minimum of 4 patients will be homozygotes to G542X, and the remaining patients will be compound heterozygotes with G542X and with one of the other allowed mutations.

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 upon the patients observed safety and tolerability, PK at previous doses and the results of laboratory tests

Study Design


Genetic Disease




Schneider Children's Medical Center
Petach Tikvah




Eloxx Pharmaceuticals, Inc.

Results (where available)

View Results


Published on BioPortfolio: 2019-10-21T12:45:41-0400

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