Focal Segmental GlomerulosclerosisNCT06162546
ARREST-NEPHROSIS - Austrian Resistant Nephrotic Syndrome Treatment Response Registry and Biobank
An observational study of Focal Segmental Glomerulosclerosis and Proteinuria, run by Christoph Aufricht at 1 site in Austria.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 75 years
- Conditions the study is for
- Focal Segmental Glomerulosclerosis, Proteinuria, Nephrotic Syndrome
- What is being tested?
- Registry
- How many people
- 100 participants
- Where
- 1 siteAustria
- Study dates
- 1 Jan 2023to 31 Dec 2033
- How long will I take part?
- 10 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: 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.
Who may be able to join
- Resistant to standard Immunosuppressive agents (if clinically indicated, e.g. primary/non-genetic forms)
- Persistent urinary protein-to-creatinine (UP/C) ratio >1.0 g/g
- eGFR > 30 ml/min per 1.73 m2
- biopsy or a disease-causing genetic mutation associated with nephrotic syndrome
Who cannot join
- Inability or unwillingness to comply with repeated assessments
- Objections against participation at discretion of the investigator
- Secondary
- Patients with steroid-dependence/frequently relapsing disease (but achievement of complete remission)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Austria1 site, 1 recruiting
- Recruiting
Division of Pediatric Nephrology and Gastroenterology, Department of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics, Medical University of Vienna, 1090 Vienna, Austria.
Vienna, 1090
Christoph Aufricht, Prof MD, +4314040021115, christoph.aufricht@meduniwien.ac.at
Lisa Daniel-Fischer, MD, +4314040021115, lisa.daniel-fischer@meduniwien.ac.at
About this study
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
- Obtaining real world data on practice patterns and outcomes
- Networking between affected patients, families, and clinicians.
- Establish a patient base for facilitated recruitment in studies of drugs, medical devices, and products
- Development of a Biobank to enable research of potential biomarkers and therapy or disease courses
Read the detailed description
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.
- Conditions
- Focal Segmental Glomerulosclerosis, Proteinuria, Nephrotic Syndrome, Nephrotic Syndrome Steroid-Resistant
- Being tested
- Registry
- Medical subject headings
- Glomerulosclerosis, Focal Segmental, Proteinuria, Nephrotic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RegistryOther
Registry Participation, providing data and clinical specimen
| Study group | Role | What they receive |
|---|---|---|
| ARREST NEPHROSIS Participants | Not described Receives: Other: Registry |
What is being measured
The primary outcome is the main question the study is designed to answer.
Obtaining real world data on practice patterns and outcomes in resistant nephrotic syndrome
Measured: up to 20 years
Enable networking between patients, families, and clinicians affected by resistant nephrotic syndrome
Measured: up to 20 years
Establish a patient base for facilitated recruitment in studies of drugs, medical devices, and products on resistant nephrotic syndrome
Measured: up to 20 years
Development of a Biobank to enable research of potential biomarkers and therapy or disease courses
Measured: up to 20 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2023
- Study start1 Jan 2023
- Primary completion31 Dec 2033 (estimated)
- Study completion31 Dec 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Focal Segmental GlomerulosclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- Christoph Aufricht
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Christoph Aufricht, Medical University of Vienna
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06162546
- Sponsor's protocol ID
- ARREST-NEPHROSIS
- Status last verified
- Nov 2023
- First submitted
- 30 Nov 2023
- Last updated
- 8 Dec 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ARREST-NEPHROSIS - Austrian Resistant Nephrotic Syndrome Treatment Response Registry and Biobank
- ClinicalTrials.gov ID
- NCT06162546
- Acronym / sponsor ID
- ARREST-NEPHROSIS
- Conditions
- Focal Segmental Glomerulosclerosis, Proteinuria, Nephrotic Syndrome, Nephrotic Syndrome Steroid-Resistant
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Christoph AufrichtUniversity, hospital or other
- Study start
- 1 Jan 2023 (actual)
- Primary completion
- 31 Dec 2033 (estimated)
- Study completion
- 31 Dec 2033 (estimated)
- First posted
- 8 Dec 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
- Obtaining real world data on practice patterns and outcomes
- Networking between affected patients, families, and clinicians.
- Establish a patient base for facilitated recruitment in studies of drugs, medical devices, and products
- Development of a Biobank to enable research of potential biomarkers and therapy or disease courses
Detailed description
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 10 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ARREST NEPHROSIS Participants | – | Other: Registry |
Who may qualify
- Ages
- Up to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- 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.
Key inclusion criteria (4 listed)
- Resistant to standard Immunosuppressive agents (if clinically indicated, e.g. primary/non-genetic forms)
- Persistent urinary protein-to-creatinine (UP/C) ratio >1.0 g/g
- eGFR > 30 ml/min per 1.73 m2
- biopsy or a disease-causing genetic mutation associated with nephrotic syndrome
Key exclusion criteria (4 listed)
- Inability or unwillingness to comply with repeated assessments
- Objections against participation at discretion of the investigator
- Secondary
- Patients with steroid-dependence/frequently relapsing disease (but achievement of complete remission)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RegistryRegistry Participation, providing data and clinical specimen | Other | ARREST NEPHROSIS Participants |
Primary outcome
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Focal Segmental Glomerulosclerosis and Proteinuria, run by Christoph Aufricht at 1 site in Austria.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Austria. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Christoph Aufricht, Prof. MD (+4314040021115, christoph.aufricht@meduniwien.ac.at), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Obtaining real world data on practice patterns and outcomes in resistant nephrotic syndrome (time frame: up to 20 years). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Dec 2023.