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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/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
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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
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Secondary ID [1]
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EL-014
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Alport Syndrome
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Renal and Urogenital
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Kidney disease
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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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).
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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The incidence and characteristics of adverse events
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Assessment method [1]
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Timepoint [1]
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From the time of first dosing through the end of the follow-up period, a total of 5 months
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Secondary outcome [1]
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Change in proteinuria
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Assessment method [1]
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Timepoint [1]
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From screening assessment to end of study treatment and end of follow up period, two and five months respectively
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Secondary outcome [2]
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Change in Col IV expression in renal biopsy
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Assessment method [2]
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Timepoint [2]
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From biopsy collected at screening to the biopsy collected at the end of study treatment, a two months interval
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Secondary outcome [3]
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Change in hematuria
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Assessment method [3]
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Timepoint [3]
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From screening assessment to end of study treatment and end of follow up period, two and five months respectively
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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)
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Minimum age
6
Years
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Maximum age
30
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Unknown status
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
28/11/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
30/05/2023
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Actual
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Sample size
Target
8
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Monash Medical Center - Clayton
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Recruitment hospital [2]
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Royal Children's Hospital - Parkville
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Recruitment postcode(s) [1]
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3168 - Clayton
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Recruitment postcode(s) [2]
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3051 - Parkville
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Recruitment outside Australia
Country [1]
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United Kingdom
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State/province [1]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Eloxx Pharmaceuticals, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05448755
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Eloxx Pharmaceuticals
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Address
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Country
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Phone
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+1(781) 577-5300
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05448755
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