A Phase 2 interventional study of ELX-02 and Ivacaftor in Cystic Fibrosis, sponsored by Eloxx Pharmaceuticals, Inc.. Completed at 9 sites in 3 countries. Open to participants aged 16 Years and older. Per ClinicalTrials.gov, last updated 2023-08-21.
Sponsored by Eloxx Pharmaceuticals, Inc. · Phase 2, Interventional, and Treatment
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 with and without ivacaftor in patients with CF with at least one G542X allele or phenotypically similar nonsense allele.
Up to 16 patients will be enrolled in the trial; up 4 patients will be homozygotes to G542X, and the remaining patients will be compound heterozygotes with G542X or phenotypically similar nonsense mutation and any Class 1 or Class 2 mutation.
Each patient will receive 5 escalating doses as follows:
1,619 studies on the registry are indexed under Cystic Fibrosis; 199 are open to participants now.
This study's enrollment of 17 is below the median of 36 across 1,051 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Eloxx Pharmaceuticals, Inc. is the lead sponsor of 9 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Patients must meet the following criteria to participate in this study:
Patients with any of the following characteristics/conditions will not be included in the study:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
FEV1
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Eukaryotic ribosomal selective glycoside (ERSG)
Drug: ELX-02 · Drug: Ivacaftor
AEs associated with different dose levels of ELX-02
Time frame: From the time of first dosing through the follow-up visit, an average of approximately 9 weeks
Area under the plasma concentration curve from time zero to 24 hours (AUC0-24)
Full PK profile 8 blood samples up to 24 hours
Time frame: Day 1 of treatment periods 1, 2, 3, and 4
Maximum observed plasma concentration (Cmax) on Day 1
Full PK profile 8 blood samples over 24 hours
Time frame: Day 1 of treatment periods 1, 2, 3, and 4
Peak observed plasma concentration (Cpeak) over time
Time frame: Days 1, 2 and 7 of treatment periods 1-3, Days 1, 2, 7, and 14 of treatment period 4, sparse sampling, blood sampling at 30 min and 1 hour post-dose
Trough observed plasma concentrations (Cpredose) over time
Time frame: Days 1, 2 and 7 of treatment periods 1-3, Days 1, 2, 7 and 14 of treatment period 4, sparse blood sampling at pre-dose
Changes from baseline in sweat chloride concentration
Time frame: From baseline to Day 7 of treatment periods 1-3, and Days 7 and 14 of treatment period 4
Changes from baseline in percent predicted forced expiratory volume (ppFEV1)
Time frame: From baseline to Day 7 of treatment periods 1-3, and Days 7 and 14 of treatment period 4
Changes from baseline in percent predicted forced vital capacity (ppFVC)
Time frame: From baseline to Day 7 of treatment periods 1-3, and Days 7 and 14 of treatment period 4
Changes from baseline in percent predicted forced expiratory flow at 25-75% (ppFEF25-75)
Time frame: From baseline to Day 7 of treatment periods 1-3, and Days 7 and 14 of treatment period 4
This study is completed, as verified in Jul 2021. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: ELX-02
Eloxx Pharmaceuticals, Inc.