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A Study of ELX-02 in Patients With Alport Syndrome

Primary Purpose

Alport Syndrome

Status

Recruiting

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

ELX-02

About this trial

This is an interventional treatment trial for Alport Syndrome focused on measuring Aminoglycoside, Nonsense Mutation, Translational read through

Eligibility Criteria

6 Years - 30 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Monash Medical CenterRecruiting
Royal Children's Hospital
Royal Free HospitalRecruiting
Great Ormond Street HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Open label study drug treatment

Arm Description

Outcomes

Primary Outcome Measures

The incidence and characteristics of adverse events

Secondary Outcome Measures

Change in proteinuria

Change in Col IV expression in renal biopsy

Change in hematuria

Full Information

NCT ID

NCT05448755

First Posted

June 29, 2022

Last Updated

December 19, 2022

Sponsor

Eloxx Pharmaceuticals, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT05448755

Brief Title

A Study of ELX-02 in Patients With Alport Syndrome

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

Study Type

Interventional

2. Study Status

Record Verification Date

June 2022

Overall Recruitment Status

Recruiting

Study Start Date

November 28, 2022 (Actual)

Primary Completion Date

May 30, 2023 (Anticipated)

Study Completion Date

May 30, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Eloxx Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Alport Syndrome

Keywords

Aminoglycoside, Nonsense Mutation, Translational read through

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

8 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Open label study drug treatment

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

ELX-02

Intervention Description

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).

Primary Outcome Measure Information:

Title

The incidence and characteristics of adverse events

Time Frame

From the time of first dosing through the end of the follow-up period, a total of 5 months

Secondary Outcome Measure Information:

Title

Change in proteinuria

Time Frame

From screening assessment to end of study treatment and end of follow up period, two and five months respectively

Title

Change in Col IV expression in renal biopsy

Time Frame

From biopsy collected at screening to the biopsy collected at the end of study treatment, a two months interval

Title

Change in hematuria

Time Frame

From screening assessment to end of study treatment and end of follow up period, two and five months respectively

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Years

Maximum Age & Unit of Time

30 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Eloxx Pharmaceuticals

Phone

+1(781) 577-5300

info@eloxxpharma.com

Facility Information:

Facility Name

Monash Medical Center

City

Clayton

State/Province

Victoria

ZIP/Postal Code

3168

Country

Australia

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Jayasinghe Kushani, MD

kushani.jayasinghe@monashhealth.org

First Name & Middle Initial & Last Name & Degree

Jo Nandkumar

Phone

+61 3 9594 3521

jyotsna.nandkumar@monashhealth.org

First Name & Middle Initial & Last Name & Degree

Jayasinghe Kushani, MD

Facility Name

Royal Children's Hospital

City

Parkville

State/Province

Victoria

ZIP/Postal Code

3051

Country

Australia

Individual Site Status

Not yet recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Catherine Quinlan, MD

Catherine.Quinlan@rch.org.au

First Name & Middle Initial & Last Name & Degree

Lauren Shaw

Phone

+61 3 9345 5054

Loren.Shaw@mcri.edu.au

First Name & Middle Initial & Last Name & Degree

Catherine Quinlan, MD

Facility Name

Royal Free Hospital

City

London

ZIP/Postal Code

NW3 2QG

Country

United Kingdom

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Daniel Gale, MD

d.gale@ucl.ac.uk

First Name & Middle Initial & Last Name & Degree

Rachel Davies

Phone

020 7472 6491

rachel.davies21@nhs.net

First Name & Middle Initial & Last Name & Degree

Daniel Gale, MD

Facility Name

Great Ormond Street Hospital

City

London

ZIP/Postal Code

WC1N 3JH

Country

United Kingdom

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Detlef Bockenhauer, MD

d.bockenhauer@ucl.ac.uk

First Name & Middle Initial & Last Name & Degree

Laura Chiverton

Phone

020 7405 9200

Ext

6892

laura.chiverton@gosh.nhs.uk

First Name & Middle Initial & Last Name & Degree

Detlef Bockenhauer, MD

12. IPD Sharing Statement

Links:

URL

http://www.eloxxpharma.com

Description

Eloxx Pharmaceuticals Website

Learn more about this trial

A Study of ELX-02 in Patients With Alport Syndrome

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