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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
28/11/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/05/2023
Actual
Sample size
Target
8
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
[email protected]
Contact person for scientific queries



Summary Results

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