Alport Syndrome Clinical Trial
Official title:
European Alport Therapy Registry - European Initiative Towards Delaying Renal Failure in Alport Syndrome: Current and Novel Therapies
NCT number | NCT02378805 |
Other study ID # | Alport-UMG2010 |
Secondary ID | |
Status | Recruiting |
Phase | |
First received | |
Last updated | |
Start date | July 1995 |
Est. completion date | January 2035 |
The hereditary type IV collagen disease Alport syndrome inevitably leads to end-stage renal disease. Currently there are no therapies known to improve outcome. Our non-interventional, observational study investigates, if medications such as ACE-inhibitors can (1) delay time to dialysis and (2) improve life-expectancy within three generations of Alport-families in Europe.
Status | Recruiting |
Enrollment | 500 |
Est. completion date | January 2035 |
Est. primary completion date | July 2010 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | N/A and older |
Eligibility | Inclusion Criteria/ Exclusion Criteria: The diagnosis of Alport syndrome (AS) was proven by kidney biopsy or mutation analysis (or both). Patients were included if they were affected males with X-linked AS or patients with genetically proven homozygous autosomal AS. Patients were excluded if they did not give informed consent or the diagnosis was suspected but not confirmed. The diagnosis of the heterozygous status was proven by (1) mutation analysis or (2) kidney biopsy plus genetic consultation for decision in between XLAS or ARAS inheritance (including a conclusive genealogic tree and/or linkage analysis). Patients were excluded if they were affected males with XLAS or patients with genetically proven homozygous ARAS. Patients were excluded if they did not give informed consent or the diagnosis was suspected but not confirmed or if they donated a kidney (living donor to affected family member). |
Country | Name | City | State |
---|---|---|---|
Germany | University Hospital Goettingen | Goettingen |
Lead Sponsor | Collaborator |
---|---|
University Hospital Goettingen | Alport Selbsthilfe e.V., Association pour l'Information et la Recherche sur les Maladies Rénales Génétiques (AIRG), Deutsche Gesellschaft für Nephrologie, KfH Foundation Preventive Medicine, Society for Pediatric Nephrology (Germany) |
Germany,
Boeckhaus J, Hoefele J, Riedhammer KM, Nagel M, Beck B, Choi M, Gollasch M, Bergmann C, Sonntag JE, Troesch V, Stock J, Gross O. Lifelong Effect of Therapy in Young Patients with the COL4A5 Alport Missense Variant p.(Gly624Asp): a Prospective Cohort Study — View Citation
Frese J, Kettwig M, Zappel H, Hofer J, Gröne HJ, Nagel M, Sunder-Plassmann G, Kain R, Neuweiler J, Gross O. Kidney Injury by Variants in the COL4A5 Gene Aggravated by Polymorphisms in Slit Diaphragm Genes Causes Focal Segmental Glomerulosclerosis. Int J M — View Citation
Gross O, Licht C, Anders HJ, Hoppe B, Beck B, Tönshoff B, Höcker B, Wygoda S, Ehrich JH, Pape L, Konrad M, Rascher W, Dötsch J, Müller-Wiefel DE, Hoyer P; Study Group Members of the Gesellschaft für Pädiatrische Nephrologie, Knebelmann B, Pirson Y, Grunfe — View Citation
Kashtan CE, Gross O. Clinical practice recommendations for the diagnosis and management of Alport syndrome in children, adolescents, and young adults-an update for 2020. Pediatr Nephrol. 2021 Mar;36(3):711-719. doi: 10.1007/s00467-020-04819-6. Epub 2020 Nov 6. Review. Erratum in: Pediatr Nephrol. 2021 Jan 12;:. — View Citation
Stock J, Kuenanz J, Glonke N, Sonntag J, Frese J, Tönshoff B, Höcker B, Hoppe B, Feldkötter M, Pape L, Lerch C, Wygoda S, Weber M, Müller GA, Gross O. Prospective study on the potential of RAAS blockade to halt renal disease in Alport syndrome patients wi — View Citation
Temme J, Kramer A, Jager KJ, Lange K, Peters F, Müller GA, Kramar R, Heaf JG, Finne P, Palsson R, Reisæter AV, Hoitsma AJ, Metcalfe W, Postorino M, Zurriaga O, Santos JP, Ravani P, Jarraya F, Verrina E, Dekker FW, Gross O. Outcomes of male patients with A — View Citation
Temme J, Peters F, Lange K, Pirson Y, Heidet L, Torra R, Grunfeld JP, Weber M, Licht C, Müller GA, Gross O. Incidence of renal failure and nephroprotection by RAAS inhibition in heterozygous carriers of X-chromosomal and autosomal recessive Alport mutatio — View Citation
Weinstock BA, Feldman DL, Fornoni A, Gross O, Kashtan CE, Lagas S, Lennon R, Miner JH, Rheault MN, Simon JF; Workshop Participants. Clinical trial recommendations for potential Alport syndrome therapies. Kidney Int. 2020 Jun;97(6):1109-1116. doi: 10.1016/j.kint.2020.02.029. Epub 2020 Apr 6. — View Citation
Zhang Y, Böckhaus J, Wang F, Wang S, Rubel D, Gross O, Ding J. Genotype-phenotype correlations and nephroprotective effects of RAAS inhibition in patients with autosomal recessive Alport syndrome. Pediatr Nephrol. 2021 Sep;36(9):2719-2730. doi: 10.1007/s0 — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | end stage renal disease | Age at onset of end stage renal failure | unlimited | |
Primary | life-expectancy | life-expectancy of patients and carriers | unlimited | |
Secondary | proteinuria after initiation of ACE-inhibitor-therapy | unlimited | ||
Secondary | proportion of patients with a clinical diagnosis of hypertension | unlimited | ||
Secondary | proportion of patients experiencing side effects from ACE-inhibitors | defined as acute renal failure (doubling of serum-creatinine), angioedema, hyperkalemia >5.0 mmol/l, dry cough, symptomatic hypotension (orthostatic collapse) and others, and death from all causes. | unlimited |
Status | Clinical Trial | Phase | |
---|---|---|---|
Terminated |
NCT01602835 -
Human Urine Sample Collection for Alport Nephropathy Biomarker Studies
|
N/A | |
Completed |
NCT01696253 -
Multi-center Controlled Clinical Trials in Alport Syndrome-A Feasibility Study
|
||
Recruiting |
NCT00481130 -
Alport Syndrome Treatments and Outcomes Registry
|
||
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
Recruiting |
NCT06065852 -
National Registry of Rare Kidney Diseases
|
||
Recruiting |
NCT05448755 -
A Study of ELX-02 in Patients With Alport Syndrome
|
Phase 2 | |
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
|
||
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
|