A Study of ELX-02 in Patients With Alport Syndrome
- Registration Number
- NCT05448755
- Lead Sponsor
- Eloxx Pharmaceuticals, Inc.
- Brief Summary
This is a Phase 2 open label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with X-linked or autosomal recessive Alport Syndrome with Col4A5 and Col4A3/4 nonsense mutation.
In total, up to 8 participants, with a minimum of 3 adults, will be enrolled in the trial.
The study will be comprised of the following periods for each participant:
* a Screening period of up to 6 weeks (42 days)
* a total Treatment Period of 8 weeks (60 days)
* a safety/efficacy Follow-up Period of 12 weeks (90 days) after the last treatment The Treatment Period will be a treatment of ELX-02 0.75 mg/kg SC QD for 8 weeks.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 8
- 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 for ages ≥18 and Schwartz formula for participants <18)
- Urinary protein based on two spot urine collections [urine protein/creatinine ratio (UPCR) ≥ 500 mg/g]
- Stable regimen of ACEi/ARB for at least 4 weeks before screening (unless there is a contraindication)
- History of any organ transplantation
- Mutation consistent with autosomal dominant Alport Syndrome
- Liver disease characterized by cirrhosis or portal hypertension. Participants with alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or a total bilirubin 3.0 times the upper limit of normal (ULN) will be excluded
- History of congestive heart failure diagnosed clinically or with documented left ventricular ejection fraction (LVEF) ≤ 40%
- History of dialysis
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Open label study drug treatment ELX-02 -
- Primary Outcome Measures
Name Time Method The incidence and characteristics of adverse events From the time of first dosing through the end of the follow-up period, a total of 5 months
- Secondary Outcome Measures
Name Time Method Change in proteinuria From screening assessment to end of study treatment and end of follow up period, two and five months respectively Change in Col IV expression in renal biopsy From biopsy collected at screening to the biopsy collected at the end of study treatment, a two months interval Change in hematuria From screening assessment to end of study treatment and end of follow up period, two and five months respectively
Trial Locations
- Locations (4)
Great Ormond Street Hospital
🇬🇧London, United Kingdom
Monash Medical Center
🇦🇺Clayton, Victoria, Australia
Royal Free Hospital
🇬🇧London, United Kingdom
Royal Children's Hospital
🇦🇺Parkville, Victoria, Australia