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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05448755
Other study ID # EL-014
Secondary ID
Status Recruiting
Phase Phase 2
First received
Last updated
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
Health authority
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

Study Design


Related Conditions & MeSH terms


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
See also
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Completed NCT00309257 - Effects of an Intensified Treatment With ACE-I,ATA II and Statins in Alport Syndrome Phase 2
Not yet recruiting NCT05133050 - Safety and Efficacy of ACEI in Alport Syndrome Patients With COL4A3/COL4A4/COL4A5 Variants N/A
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