An observational study in Focal Segmental Glomerulosclerosis, Proteinuria and Nephrotic Syndrome, sponsored by Christoph Aufricht. Recruiting at 1 site in Austria. Open to participants aged Up to 75 Years. Per ClinicalTrials.gov, last updated 2023-12-08.
Sponsored by Christoph Aufricht · Observational
Nephrotic syndrome is the clinical phenotype of a heterogeneous group of glomerular diseases that may present with varying degrees of urinary protein loss (proteinuria), dysproteinemia in the blood, fluid retention and impaired renal function.
The AustRian RESistanT NEPHROtic Syndrome Treatment Response RegIStry and Biobank (ARREST-NEPHROSIS) sets out to achieve the following goals, as typical categories of rare disease registries
A significant proportion of patients with nephrotic syndrome remains unresponsive to currently used therapies ("resistant nephrotic syndrome"), the majority of them with focal segmental glomerulosclerosis (FSGS). FSGS is reported as an underlying condition in 5% of adult and 12% of pediatric patients with end-stage kidney disease (ESKD), ultimately requiring renal replacement therapy. The estimated incidence for FSGS is 7 patients per 1 million people.
Despite its clinical and financial burden, the pharmaceutical market for such rare (orphan) diseases is narrow, and industry is less likely to invest in research and to develop treatments for these than for more prevalent medical conditions. However, there remains an urgent need to find new treatments and test their efficacy in suitable trials in resistant nephrotic syndrome.
Patient registries are critical tools to tackle that unmet medical need in orphan diseases. The AustRian RESistanT NEPHROtic Syndrome Treatment Response RegIStry and Biobank (ARREST-NEPHROSIS) therefore sets out to achieve the following goals, in line with typical categories of rare disease registries: (1) networking between affected patients, families, and clinicians; (2) obtaining real world data on practice patterns and outcomes (3); establish a patient base for facilitated recruitment in studies of drugs, medical devices, and products, and (4) development of a biobank to enable research of potential biomarkers predicting disease courses under therapy.
To achieve these goals, the ARREST-NEPHROSIS registry aims for long-term data collection in patients from childhood to adulthood. Patients who fulfill the inclusion criteria (resistance to standard immunosuppressive agents (if clinically indicated, e.g. for primary/non-genetic forms), persistent urinary protein-to-creatinine (UP/C) ratio >1.0 g/g, eGFR > 30 ml/min per 1.73 m2 and a biopsy or a disease-causing genetic mutation associated with nephrotic syndrome) will be invited to the registry, and, after consent, will undergo clinical assessments and receive care as determined by the patient's treating physician. Clinical routine data will be transcribed into the registry to describe patient characteristics (e.g. age, sex, etc.), disease typology (e.g. biopsy and genetics findings, treatment history, etc.), disease activity (e.g. level of proteinuria) and renal function (e.g. eGFR decline).
The continuous prospective assessment of internationally harmonized endpoints suggested by the SONG-GD group (such as proteinuria, including patient reported outcomes (PROs), and the low barrier for access to novel therapeutic studies, will represent an important component of rare disease research in Austria and might ultimately improve health outcomes in this vulnerable population.
105 studies on the registry are indexed under Glomerulosclerosis, Focal Segmental; 33 are open to participants now.
This study's planned enrollment of 100 is below the median of 175 across 28 observational studies indexed under Glomerulosclerosis, Focal Segmental.
Browse Glomerulosclerosis, Focal Segmental studies →Christoph Aufricht is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Patients with nephrotic syndrome are informed about the project as part of a routine check-up and invited to participate by their treating physician. Patients are also informed verbally that there is no obligation to participate and that they can withdraw their willingness to participate at any time.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Other: Registry
Registry Participation, providing data and clinical specimen
Obtaining real world data on practice patterns and outcomes in resistant nephrotic syndrome
Time frame: up to 20 years
Enable networking between patients, families, and clinicians affected by resistant nephrotic syndrome
Time frame: up to 20 years
Establish a patient base for facilitated recruitment in studies of drugs, medical devices, and products on resistant nephrotic syndrome
Time frame: up to 20 years
Development of a Biobank to enable research of potential biomarkers and therapy or disease courses
Time frame: up to 20 years
Plan to share: No
No publications or documents are linked to this record.
Started Jan 2023 (actual). Primary completion is estimated for Dec 2033, 131 months after the start.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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Glomerulosclerosis, Focal Segmental →
Christoph Aufricht