Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE2
8 participants
INTERVENTIONAL
2022-11-28
2023-05-30
Brief Summary
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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
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Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Open label study drug treatment
ELX-02
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).
Interventions
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ELX-02
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).
Eligibility Criteria
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Inclusion Criteria
* 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)
Exclusion Criteria
* 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
6 Years
30 Years
ALL
No
Sponsors
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Eloxx Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Monash Medical Center
Clayton, Victoria, Australia
Royal Children's Hospital
Parkville, Victoria, Australia
Royal Free Hospital
London, , United Kingdom
Great Ormond Street Hospital
London, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Jayasinghe Kushani, MD
Role: primary
Jo Nandkumar
Role: backup
Catherine Quinlan, MD
Role: primary
Lauren Shaw
Role: backup
Daniel Gale, MD
Role: primary
Rachel Davies
Role: backup
Detlef Bockenhauer, MD
Role: primary
Laura Chiverton
Role: backup
References
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Rheault MN. Treatment Approaches for Alport Syndrome. J Am Soc Nephrol. 2025 Sep 12. doi: 10.1681/ASN.0000000897. Online ahead of print.
Related Links
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Eloxx Pharmaceuticals Website
Other Identifiers
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EL-014
Identifier Type: -
Identifier Source: org_study_id