Clinical trials: Alport Syndrome
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
The primary objective of this multicenter observational registry is to characterize the clinical, epidemiological, and biological profiles of individuals of Middle Eastern, North African, and Levantine ancestry, including…
- Conditions
- Antiphospholipid (aPL)-Positive, Rheumatic Arthritis, Celiac Disease, Focal Segmental Glomerulosclerosis (FSGS)
- Trial name
- MERIT (NCT07892651)
- Where
- Jordan
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Axiom Bioregistry
Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive
EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
This is a randomized, double-Blind, placebo-controlled study to evaluate the efficacy and safety of exaluren in Alport Syndrome patients with nonsense mutations in COL4A3/4/5 genes.
- Trial name
- NCT07523581
- Testing
- Exaluren
- Where
- 11 sites in United States, United Kingdom
- Volunteers
- 24 people
- Sponsor
- Eloxx Pharmaceuticals, Inc.
Alport Syndrome
Eurbio-Alport (RaDiCo Cohort) (RaDiCo Eurbio-Alport)
Alport syndrome is a rare, inherited condition characterized by a combination of glomerular nephropathy progressing to kidney failure, deafness, and eye involvement.
- Trial name
- Eurbio-Alport (NCT05927467)
- Where
- France
- Volunteers
- 700 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Nephrotic Syndrome in Children, Focal Segmental Glomerulosclerosis
NEPTUNE Match Study
NEPTUNE Match is an additional opportunity offered to NEPTUNE study participants to prospectively recruit and communicate patient-specific clinical trial matching with kidney patients and their physician investigators.
- Conditions
- Nephrotic Syndrome in Children, Focal Segmental Glomerulosclerosis, Minimal Change Disease, Minimal Change Nephrotic Syndrome
- Trial name
- NEPTUNE Match (NCT04571658)
- Testing
- Communication
- Where
- 16 sites in United States
- Volunteers
- 375 people
- Sponsor
- University of Michigan
Focal Segmental Glomerulosclerosis, Minimal Change Disease
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases
To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and tablets, and assess changes in proteinuria after once-daily dosing over 108 weeks.
- Conditions
- Focal Segmental Glomerulosclerosis, Minimal Change Disease, Immunoglobulin A Nephropathy, IgA Vasculitis
- Trial name
- EPPIK (NCT05003986)
- Testing
- Sparsentan
- Where
- 47 sites in United States, Germany and 6 more
- Volunteers
- 67 people
- Sponsor
- Travere Therapeutics, Inc.
Alport Syndrome, Thin Basement Membrane Disease
ASF Alport Patient Registry
Alport Syndrome Foundation's (ASF's) Alport Patient Registry (the Registry) is open to individuals living with Alport syndrome in the United States (US) and US territories and outlying islands.
- Conditions
- Alport Syndrome, Thin Basement Membrane Disease, Hereditary Nephritis
- Trial name
- NCT06526741
- Testing
- Longitudinal data collection
- Where
- United States
- Volunteers
- 2,500 people
- Sponsor
- Alport Syndrome Foundation
Alport Syndrome
A Study to Evaluate Setanaxib in Patients With Alport Syndrome
The purpose of the study is to learn about the safety and tolerability of setanaxib in subjects with Alport syndrome, when added to their standard of care treatment.
- Trial name
- NCT06274489
- Testing
- Setanaxib
- Where
- 17 sites in Austria, Czechia and 3 more
- Volunteers
- 20 people
- Sponsor
- Calliditas Therapeutics AB
Alport Syndrome, X-Linked
Study of Hydroxychloroquine in Patients With X-linked Alport Syndrome in China (CHXLAS)
This Phase 2 randomized controlled trial will study the safety, tolerability, and efficacy of Hydroxychloroquine in qualified patients with Alport syndrome.
- Trial name
- CHXLAS (NCT04937907)
- Testing
- Hydroxychloroquine Sulfate 100 milligram (mg) Tab, Benazepril hydrochloride 10 milligram (mg) Tab
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Shanghai Children's Hospital
Alport Syndrome
A Study of ELX-02 in Patients With Alport Syndrome
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.
- Trial name
- NCT05448755
- Testing
- ELX-02
- Where
- 4 sites in Australia, United Kingdom
- Volunteers
- 3 people
- Sponsor
- Eloxx Pharmaceuticals, Inc.
Alport Syndrome
Vonafexor ALPort Syndrome Efficacy & Safety TRIAl-1 (ALPESTRIA-1)
This study is a proof-of-concept trial of vonafexor safety, its effects on kidney function in subjects with at risk of progression Alport syndrome.
- Trial name
- ALPESTRIA-1 (NCT06425055)
- Testing
- Vonafexor
- Where
- 13 sites in United States, France and 1 more
- Volunteers
- 26 people
- Sponsor
- Enyo Pharma
Alport Syndrome
Alport Syndrome Treatments and Outcomes Registry
ASTOR's primary purpose is to enroll families and patients with a history of Alport syndrome in a central registry. The information we gather will be used as a basis for studies designed to test potential treatments for Alport…
- Trial name
- ASTOR (NCT00481130)
- Where
- United States
- Volunteers
- 655 people
- Sponsor
- University of Minnesota
Alport Syndrome, Focal Segmental Glomerulosclerosis
Study to Evaluate R3R01 in Patients With Alport Syndrome and Patients With Focal Segmental Glomerulosclerosis
This is a Phase 2, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary…
- Trial name
- NCT05267262
- Testing
- R3R01
- Where
- 22 sites in United States, Belgium and 4 more
- Volunteers
- 43 people
- Sponsor
- River 3 Renal Corp.
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Alport Syndrome
A Phase 2/3 Trial of the Efficacy and Safety of Bardoxolone Methyl in Patients With Alport Syndrome - CARDINAL
This international, multi-center, Phase 2/3 trial will study the safety, tolerability, and efficacy of bardoxolone methyl in qualified patients with Alport syndrome.
- Trial name
- CARDINAL (NCT03019185)
- Testing
- Bardoxolone Methyl
- Where
- 63 sites in United States, Australia and 6 more
- Volunteers
- 187 people
- Sponsor
- Biogen
Chronic Kidney Diseases, Alport Syndrome
An Extended Access Program for Bardoxolone Methyl in Patients With CKD (EAGLE)
This extended access study will assess the long-term safety and tolerability of bardoxolone methyl in qualified patients with chronic kidney disease (CKD) who previously participated in one of the qualifying clinical studies with…
- Conditions
- Chronic Kidney Diseases, Alport Syndrome, Autosomal Dominant Polycystic Kidney
- Trial name
- EAGLE (NCT03749447)
- Testing
- Bardoxolone methyl
- Where
- 87 sites in United States, Australia and 4 more
- Volunteers
- 270 people
- Sponsor
- Biogen
Alport Syndrome, Hereditary Kidney Disease
Alport Therapy Registry - European Initiative Towards Delaying Renal Failure in Alport Syndrome
The hereditary type IV collagen disease Alport syndrome leads to kidney failure early in life. Currently there are no specific medications approved for treatment, however, several therapies have been evaluated preclinically and…
- Conditions
- Alport Syndrome, Hereditary Kidney Disease, Pediatric Kidney Disease, Thin Basement Membrane Disease
- Trial name
- Alport-XXL (NCT02378805)
- Testing
- ACE-inhibitor, Angiotensin-receptor blocker (ARB), HMG-Coenzyme inhibitor (statin)
- Where
- Germany
- Volunteers
- 800 people, healthy volunteers welcome
- Sponsor
- University Hospital Goettingen
Alport Syndrome
Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome
The goal of this clinical trial is to evaluate the safety and efficacy of human umbilical cord mesenchymal stem cells (hUC-MSC) in the treatment of Alport syndrome (AS) in a randomized, single-blind, placebo-controlled trial, to…
- Trial name
- HUCMSC (NCT06731192)
- Testing
- hUC-MSC
- Volunteers
- 40 people
- Sponsor
- Guangzhou Women and Children's Medical Center
Alport Syndrome
Effects of Dapagliflozin on Progression of Alport Syndrome
Recently, a series of large clinical trials have confirmed the cardio-renal protective effects of sodium-glucose cotransporter 2 (SGLT2) inhibitors. but few patients with hereditary nephritis were included in these studies.
- Trial name
- NCT06226896
- Testing
- Dapagliflozin 10mg Tab
- Where
- China
- Volunteers
- 222 people
- Sponsor
- Nanjing University School of Medicine
Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis
National Registry of Rare Kidney Diseases
The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research.
- Conditions
- Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis, AHL Amyloidosis, AL Amyloidosis
- Trial name
- RaDaR (NCT06065852)
- Where
- United Kingdom
- Volunteers
- 35,000 people
- Sponsor
- UK Kidney Association