A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele
- Registration Number
- NCT04126473
- Lead Sponsor
- Eloxx Pharmaceuticals, Inc.
- Brief 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 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:
* 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
* ELX-02 1.5 mg/kg per day SC plus 150 mg ivacaftor every 12 bid
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 17
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ELX-02 ELX-02 Eukaryotic ribosomal selective glycoside (ERSG) ELX-02 Ivacaftor Eukaryotic ribosomal selective glycoside (ERSG)
- Primary Outcome Measures
Name Time Method Peak observed plasma concentration (Cpeak) over time 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 AEs associated with different dose levels of ELX-02 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) Day 1 of treatment periods 1, 2, 3, and 4 Full PK profile 8 blood samples up to 24 hours
Maximum observed plasma concentration (Cmax) on Day 1 Day 1 of treatment periods 1, 2, 3, and 4 Full PK profile 8 blood samples over 24 hours
Trough observed plasma concentrations (Cpredose) over time 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
- Secondary Outcome Measures
Name Time Method Changes from baseline in sweat chloride concentration 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) 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) 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) From baseline to Day 7 of treatment periods 1-3, and Days 7 and 14 of treatment period 4
Trial Locations
- Locations (9)
Hadassah Medical Center
🇮🇱Jerusalem, Israel
Universitätsklinikum Frankfurt
🇩🇪Frankfurt, Germany
The Royal Prince Alfred Hospital
🇦🇺Camperdown, New South Whales, Australia
Schneider Children's Medical Center
🇮🇱Petach Tikvah, Israel
Carmel Medical Center
🇮🇱Haifa, Israel
The Alfred Hospital
🇦🇺Melbourne, Victoria, Australia
Safra Children's Hospital - Chaim Sheba Medical Center
🇮🇱Ramat Gan, Israel
Universitätsmedizin Essen Ruhrlandklinik
🇩🇪Essen, North Rhine-Westphalia, Germany
The Royal Adelaide Hospital
🇦🇺Adelaide, South Australia, Australia