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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05448755




Registration number
NCT05448755
Ethics application status
Date submitted
29/06/2022
Date registered
7/07/2022
Date last updated
20/12/2022

Titles & IDs
Public title
A Study of ELX-02 in Patients With Alport Syndrome
Scientific 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
Secondary ID [1] 0 0
EL-014
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Alport Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Renal and Urogenital 0 0 0 0
Kidney disease
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ELX-02

Experimental: Open label study drug treatment -


Treatment: Drugs: 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).

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The incidence and characteristics of adverse events
Timepoint [1] 0 0
From the time of first dosing through the end of the follow-up period, a total of 5 months
Secondary outcome [1] 0 0
Change in proteinuria
Timepoint [1] 0 0
From screening assessment to end of study treatment and end of follow up period, two and five months respectively
Secondary outcome [2] 0 0
Change in Col IV expression in renal biopsy
Timepoint [2] 0 0
From biopsy collected at screening to the biopsy collected at the end of study treatment, a two months interval
Secondary outcome [3] 0 0
Change in hematuria
Timepoint [3] 0 0
From screening assessment to end of study treatment and end of follow up period, two and five months respectively

Eligibility
Key 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)
Minimum age
6 Years
Maximum age
30 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
UNKNOWN
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Monash Medical Center - Clayton
Recruitment hospital [2] 0 0
Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment postcode(s) [2] 0 0
3051 - Parkville
Recruitment outside Australia
Country [1] 0 0
United Kingdom
State/province [1] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Eloxx Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT05448755
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Eloxx Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
+1(781) 577-5300
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05448755