Ignite Creation Date:
2024-05-06 @ 7:35 PM
Last Modification Date:
2024-10-26 @ 3:10 PM
Study NCT ID:
NCT06065852
Status:
RECRUITING
Last Update Posted:
2023-10-04
First Post:
2023-09-19
Brief Title:
National Registry of Rare Kidney Diseases
Sponsor:
UK Kidney Association
Organization:
UK Kidney Association
Study Overview
Official Title:
National Registry of Rare Kidney Diseases RaDaR
Status:
RECRUITING
Status Verified Date:
2023-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
RaDaR
Brief Summary:
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
The purpose of this research is to
Develop Clinical Guidelines for specific rare kidney diseases These are written recommendations on how to diagnose and treat a medical condition
Audit treatments and outcomes An audit makes checks to see if what should be done is being done and asks if it could be done better
Further the development of future treatments
Participants will be invited to participate on clinical trials and other studies The registry has the capacity to feedback relevant information to patients and in conjunction with Patient Knows Best Home - Patients Know Best allows patients to provide information themselves including their own reported quality of life and outcome measures
The purpose of this research is to
Develop Clinical Guidelines for specific rare kidney diseases These are written recommendations on how to diagnose and treat a medical condition
Audit treatments and outcomes An audit makes checks to see if what should be done is being done and asks if it could be done better
Further the development of future treatments
Participants will be invited to participate on clinical trials and other studies The registry has the capacity to feedback relevant information to patients and in conjunction with Patient Knows Best Home - Patients Know Best allows patients to provide information themselves including their own reported quality of life and outcome measures
Detailed Description:
Background
Rare diseases are arbitrarily defined as having an incidence such that they cannot be studied effectively on patient groups drawn from one or a few medical centres
A high proportion of such disorders have a genetic background and often these diseases are first expressed in childhood The success of chronic and end-stage renal failure programmes in childhood permit increased numbers of these patients to survive into adulthood There are 13 centres for paediatric nephrology in the UK For a rare disorder that a paediatric nephrologist might diagnosis only once a year and assuming 100 survival to adulthood a renal physician might be asked to take over such a case only once in seven or eight years of practice Research is hampered by this dilution of clinical experience Similarly in adult practice there are rare complications of diseases or their treatment so that a nephrologist might encounter such an event less often than once in every 5 years National aggregation of clinical experience is essential to further study
Research groups investigating a rare disease Rare Disease Groups RDGs have difficulty accessing patients who are widely distributed While rare disease groups are often successful in identifying novel genotypes in a few individuals it is more difficult to define phenotype and undertake phenotype-genotype correlations Moreover the scarcity of patients makes it difficult to develop biomarkers or identify well-defined cohorts in which to test novel treatments As a result the progression and outcome for many rare diseases are unknown and treatment remains underdeveloped
Purpose
The purpose of the National Registry of Rare Kidney Diseases RaDaR rare disease registry is to facilitate translational and epidemiological research into rare kidney diseases by setting up and maintaining a comprehensive clinical database in partnership with Rare Disease Groups
RaDaR facilitates the identification of well-characterized cohorts of patients who may be invited to participate in clinical trials the development of biomarkers phenotype-genotype correlations or outcome studies This will inform the development of clinical guidelines for specific rare diseases audit treatment and outcome and further the development of future therapies
RaDaR provides an infrastructure to capture both generic and disease-specific clinical information and to collate longitudinal information Patients and clinicians can view information about the conditions covered by RaDaR on RareRenalorg which links closely with RaDaR
RaDaR is predominately aimed at UK patients however international recruits who are consented in the UK by an NHS hospital are also eligible subject to local approval
Rare diseases are arbitrarily defined as having an incidence such that they cannot be studied effectively on patient groups drawn from one or a few medical centres
A high proportion of such disorders have a genetic background and often these diseases are first expressed in childhood The success of chronic and end-stage renal failure programmes in childhood permit increased numbers of these patients to survive into adulthood There are 13 centres for paediatric nephrology in the UK For a rare disorder that a paediatric nephrologist might diagnosis only once a year and assuming 100 survival to adulthood a renal physician might be asked to take over such a case only once in seven or eight years of practice Research is hampered by this dilution of clinical experience Similarly in adult practice there are rare complications of diseases or their treatment so that a nephrologist might encounter such an event less often than once in every 5 years National aggregation of clinical experience is essential to further study
Research groups investigating a rare disease Rare Disease Groups RDGs have difficulty accessing patients who are widely distributed While rare disease groups are often successful in identifying novel genotypes in a few individuals it is more difficult to define phenotype and undertake phenotype-genotype correlations Moreover the scarcity of patients makes it difficult to develop biomarkers or identify well-defined cohorts in which to test novel treatments As a result the progression and outcome for many rare diseases are unknown and treatment remains underdeveloped
Purpose
The purpose of the National Registry of Rare Kidney Diseases RaDaR rare disease registry is to facilitate translational and epidemiological research into rare kidney diseases by setting up and maintaining a comprehensive clinical database in partnership with Rare Disease Groups
RaDaR facilitates the identification of well-characterized cohorts of patients who may be invited to participate in clinical trials the development of biomarkers phenotype-genotype correlations or outcome studies This will inform the development of clinical guidelines for specific rare diseases audit treatment and outcome and further the development of future therapies
RaDaR provides an infrastructure to capture both generic and disease-specific clinical information and to collate longitudinal information Patients and clinicians can view information about the conditions covered by RaDaR on RareRenalorg which links closely with RaDaR
RaDaR is predominately aimed at UK patients however international recruits who are consented in the UK by an NHS hospital are also eligible subject to local approval
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None