A Study of ELX-02 in Patients With Alport Syndrome

Alport Syndrome Clinical Trial

Official title:

A Phase 2 Open Label Pilot Study to Evaluate the Safety and Efficacy of Subcutaneously Administered ELX-02 in Patients With Alport Syndrome With Col4A5 and Col4A3/4 Nonsense Mutation

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05448755
• Other study ID #: EL-014
• Status: Recruiting
• Phase: Phase 2
• Start date: November 28, 2022
• Est. completion date: May 30, 2023

Study information

• Verified date: June 2022
• Source: Eloxx Pharmaceuticals, Inc.
• Contact: Eloxx Pharmaceuticals
• Phone: +1(781) 577-5300
• Email: info[@]eloxxpharma.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 8
• Est. completion date: May 30, 2023
• Est. primary completion date: May 30, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 30 Years

Eligibility

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 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:

• 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

Related Conditions & MeSH terms

• Alport Syndrome
• Nephritis, Hereditary
• Syndrome

Intervention

Drug:
• 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).

Locations

Country	Name	City	State
Australia	Monash Medical Center	Clayton	Victoria
Australia	Royal Children's Hospital	Parkville	Victoria
United Kingdom	Great Ormond Street Hospital	London
United Kingdom	Royal Free Hospital	London

Sponsors (1)

Lead Sponsor	Collaborator
Eloxx Pharmaceuticals, Inc.

Countries where clinical trial is conducted

Australia,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Change in proteinuria		From screening assessment to end of study treatment and end of follow up period, two and five months respectively
Secondary	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
Secondary	Change in hematuria		From screening assessment to end of study treatment and end of follow up period, two and five months respectively

External Links

•   Eloxx Pharmaceuticals Website