Alport Syndrome Clinical Trial
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
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.
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 |
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 |
Lead Sponsor | Collaborator |
---|---|
Eloxx Pharmaceuticals, Inc. |
Australia, United Kingdom,
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 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT01696253 -
Multi-center Controlled Clinical Trials in Alport Syndrome-A Feasibility Study
|
||
Terminated |
NCT01602835 -
Human Urine Sample Collection for Alport Nephropathy Biomarker Studies
|
N/A | |
Recruiting |
NCT00481130 -
Alport Syndrome Treatments and Outcomes Registry
|
||
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
Recruiting |
NCT06065852 -
National Registry of Rare Kidney Diseases
|
||
Recruiting |
NCT06274489 -
A Study to Evaluate Setanaxib in Patients With Alport Syndrome
|
Phase 1/Phase 2 | |
Terminated |
NCT02855268 -
Study of Lademirsen (SAR339375) in Patients With Alport Syndrome
|
Phase 2 | |
Recruiting |
NCT06226896 -
Effects of Dapagliflozin on Progression of Alport Syndrome
|
||
Recruiting |
NCT05927467 -
Eurbio-Alport (RaDiCo Cohort) (RaDiCo Eurbio-Alport)
|
||
Terminated |
NCT03749447 -
An Extended Access Program for Bardoxolone Methyl in Patients With CKD (EAGLE)
|
Phase 3 | |
Completed |
NCT03019185 -
A Phase 2/3 Trial of the Efficacy and Safety of Bardoxolone Methyl in Patients With Alport Syndrome - CARDINAL
|
Phase 2/Phase 3 | |
Recruiting |
NCT06425055 -
Vonafexor ALPort Syndrome Efficacy & Safety TRIAl-1 (ALPESTRIA-1)
|
Phase 2 | |
Completed |
NCT01705132 -
Urinary Biomarkers of the Progression of Alport Kidney Disease
|
N/A | |
Recruiting |
NCT05267262 -
Study to Evaluate R3R01 in Patients With Alport Syndrome and Patients With Focal Segmental Glomerulosclerosis
|
Phase 2 | |
Completed |
NCT00622544 -
A Prospective Study of Microalbuminuria in Untreated Boys With Alport Syndrome
|
||
Recruiting |
NCT02378805 -
European Alport Therapy Registry - European Initiative Towards Delaying Renal Failure in Alport Syndrome
|
||
Not yet recruiting |
NCT05655728 -
Treatment With Metformin in Chinese Children With Alport Syndrome
|
Phase 4 | |
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 | |
Recruiting |
NCT04947813 -
Genotype-Phenotype Correlations in Patients With Alport Syndrome
|