Trial Number

972-19

Condition

Cystic Fibrosis

Participant Age Range

18 years and older

Participant Gender

Any

Enrolling Participants

Yes

Overview

A Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients with Cystic Fibrosis with at Least One G542X Allele

Cystic fibrosis is the most common severe and lethal genetic disease among Caucasians and is caused by numerous mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CF is an autosomal recessive disease, with over 2000 CFTR gene mutations associated with the disease. Of these mutations, approximately 10-13% are due to nonsense mutation (nmCF), with the most prevalent mutation, G542X, accounting for half. Patients with nmCF experience significant morbidity and mortality despite these aggressive supportive interventions and remain the CF patient class with the highest unmet medical need. Eloxx Pharmaceuticals is developing ELX-02 as a treatment for inherited conditions caused by nonsense mutations whereby the mutation leads to decreased or absent protein expression. ELX-02 is intended to read through the nonsense mutation and induce the translation of full-length functional proteins to have a beneficial clinical effect in these diseases. This is a Phase 2 open label study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple dose levels of ELX-02 in patients with CF with at least one G542X allele.

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