Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ELX-02 · 6 trials · 5 indications
Treatment Emerging Adverse Events were defined as any adverse events with onset after the first administration of study medication through the end of the study, or any event that was present at baseline but worsened in intensity or was subsequently considered drug-related by the Investigator through the end of the study. Severe Treatment Emerging Adverse Event is a Treatment Emerging Adverse Events with CTCAE Grade 3 or above. Related Treatment Emerging Adverse Events is defined as a Treatment Emerging Adverse Events with a certain, probable/likely, or possible relationship to the study drug. Serious Adverse Events were Adverse Events resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening.
Full PK profile 8 blood samples over 24 hours
Full PK profile 8 blood samples over 24 hours
Full PK profile 8 blood samples up to 24 hours
Area under the curve (AUC0-24) of ELX-02 plasma concentration following a single subcutaneous (SC) dose
Peak Plasma Concentration (Cmax) of ELX-02 following a single subcutaneous (SC) dose in subjects with normal renal function, mild, moderate, or severe renal impairment
Area under the curve (AUC0-inf) of ELX-02 plasma concentration following a single subcutaneous (SC) dose
Time to maximum concentration (Tmax) of ELX-02 plasma concentration following a single subcutaneous (SC) dose
Cumulative amount of unchanged drug excreted into urine (Ae0-t) of ELX-02 following a single subcutaneous (SC) dose
Maximum rate of urinary extraction (Rmax) of ELX-02 following a single subcutaneous (SC) dose
TEAEs are undesirable events not present prior to medical treatment, or an already present event that worsens either in intensity or frequency following the study treatment
Day 1 Area under the curve (AUC0-24) of ELX-02 plasma concentration following the subcutaneous (SC) dose on Day 1 to 24 hours post-ose
Day 1 Peak Plasma Concentration (Cmax) of ELX-02 following the subcutaneous (SC) dose on Day 1 to 72 hours post-dose
Day 1 Area under the curve (AUC0-inf) of ELX-02 plasma concentration following the subcutaneous (SC) dose on Day 1 extrapolated to infinity
Day 1 Time to maximum concentration (Tmax) of ELX-02 plasma concentrations following the subcutaneous (SC) dose on Day 1
Day 29 Time to maximum concentration (Tmax) of ELX-02 plasma concentrations following the subcutaneous (SC) dose on Day 29
Accumulation ratio, calculated as AUC24h Day29/AUC24h Day 1
Accumulation ratio, calculated as Cmax Day29/Cmax Day 1
Day 1 Cumulative amount of unchanged drug excreted into urine (Ae72h) of ELX-02 following the subcutaneous (SC) dose on Day 1
Percent of dose excreted (Fe) in urine on Day 1
Percent of dose excreted (Fe) in urine on Day 29
Renal clearance on Day 1 (CLR=Ae24h/plasmaAUC24h)
Renal clearance on Day 29 (CLR=Ae24h/plasmaAUC24h)
Incidence and characteristics of adverse events occurring following single doses of ELX-02
Cmax will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
tmax will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
AUC24h will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
AUC48h will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
AUCinf will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
MRT will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
t1/2 will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Vd/F will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
CL/F will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
| Arm | Type | Description |
|---|---|---|
| Open label study drug treatment | EXPERIMENTAL | - |
| ELX-02 | EXPERIMENTAL | Eukaryotic ribosomal selective glycoside (ERSG) |
| Placebo Comparator Arm | PLACEBO_COMPARATOR | Placebo |
| Active treatment | ACTIVE_COMPARATOR | ELX-02 |
| Name | Type | Description |
|---|---|---|
| ELX-02 | DRUG | ELX-02 is a small molecule, new chemical entity being developed for the treatment of genetic diseases caused by nonsense mutations. ELX-02 is a eukaryotic ribosomal selective glycoside (ERSG). |
| Ivacaftor | DRUG | CFTR potentiator |
| Placebo | DRUG | Placebo |
Inclusion Criteria: * A confirmed diagnosis of X-linked or autosomal recessive Alport Syndrome with a documented nonsense mutation of Col4A5 in a male or nonsense mutation of Col4A3 or Col4A4 (male or female) * The nonsense mutation should be UAG or UGA * eGFR\>60 ml/min/1.73 m2 (based on CKD-EPI f...