A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele
Summary
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
Conditions
Genetic Disease
Intervention
ELX-02
Location
Schneider Children's Medical Center
Petach Tikvah
Israel
Status
Recruiting
Source
Eloxx Pharmaceuticals, Inc.
Results (where available)
Links
- Source: http://clinicaltrials.gov/show/NCT04126473
- ClinicalTrials.gov processed this data on October 18, 2019
Published on BioPortfolio: 2019-10-21T12:45:41-0400
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