Overall aim: Given the aforementioned rationale it is therefore important to know the natural course of the disease and stage specific morbidity and mortality factors in a longitudinal observational study, to evaluate the levels of and associations…
ID
Source
Brief title
Condition
- Renal and urinary tract disorders congenital
- Nephropathies
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
To investigate disease progression factors and association of disease
biomarkers with the onset and severity of ADPKD-related outcomes like
hypertension, renal function, renal pain renal, albuminuria, renal urine
concentrating ability, hematuria, cyst infection, nephrolithiasis and others.
Secondary outcome
To summarize the levels of self-estimated health status, pain, QoL,
ADPKD-related health burden, health care resource use.
Background summary
Autosomal dominant polycystic kidney disease (ADPKD) is characterized by
progressive cyst formation in both kidneys, leading to end stage renal disease.
It is the most common hereditary disease, with a prevalence rate of 1 in 400 to
1 in 1,000 persons. ADPKD is an important cause of renal failure.
Unfortunately, there are not many marketed proven effective therapies to halt
disease progression yet. At the moment, one drug has been proven to be
effective and a number of other drugs are being evaluated. Now efficacy of one
drug has been established, and in the future probably others, pivotal questions
are whom to treat and when to initiate such treatment. Given that ADPKD is a
progressive condition, it seems most appropriate to initiate intervention as
early in life as possible to delay or prevent long-term consequences, including
renal failure and cardiovascular and liver complications. On the other hand,
ESRD occurs in only approximately 70% of affected subjects and not all affected
subjects will experience disease related adverse health outcomes. It will
therefore not be appropriate to expose all subjects to life long medical
treatment that may cause adverse events.Therefore, the present studie is
designed large scale observational cohort with sufficient duration of follow-up
to investigate new biomarkers and determinants to predict and monitor renal
function decline.
Study objective
Overall aim: Given the aforementioned rationale it is therefore important to
know the natural course of the disease and stage specific morbidity and
mortality factors in a longitudinal observational study, to evaluate the levels
of and associations between the impacts of patients reported disease outcome
(quality of life (QoL), pain, self-estimated health status, health burden) and
to develop markers that alone, or in combination, predict prognosis in ADPKD.
Primary Objective: To investigate renal disease progression and association of
disease biomarkers with renal disease progression, and with the onset and
severity of ADPKD-related outcomes.
Secondary objectives: 1. To investigate liver disease progression and
association of disease biomarkers with liver disease progression, and with the
onset and severity of ADPKD-related outcomes. 2. To evaluate and establish the
level of the renal and liver disease impact on patient reported outcomes. 3. To
explore and identify additional parameters of disease progression.
Study design
Multicenter, longitudinal, observational investigator driven cohort study to
investigate renal disease progression and association of disease biomarkers
with renal disease progression in subjects with based on the Ravine criteria
(with number of cysts known from a previous ultrasound or magnetic resonance
imaging [MRI]) over a period of at least 6 years.
Study burden and risks
When compared to routine clinical care the burden and risk associated with
participation are:
• In general ADPKD patients, depending on their renal function will visit an
out-patient department once every 3 to 12 months routinely. Therefore this
study imposes 0 extra visits to an outpatient department when compared to
routine care, since the observationele visits are every 12 months.
• In general ADPKD patients when visiting an out-patient department collect
24hr urine and blood is drawn for routine clinical chemistry. During the every
visit extra blood will be drawn for biobanking.
• 4 times a MRI of liver and kidneys (without contrast)
• 4 times a questionnaire
The subjects have no potential benefit.
Hanzeplein 1
Groningen 9713 GZ
NL
Hanzeplein 1
Groningen 9713 GZ
NL
Listed location countries
Age
Inclusion criteria
1. Diagnosis of ADPKD, based upon the modified Ravine criteria or documented by
treating
nephrologist or internist.
2. Age 18 years and older.
3. Providing informed consent.
Exclusion criteria
• Patients, who are unlikely to adequately comply with the trial*s procedures
(due for instance to medical condition likely to require an extended
interruption or discontinuation)
• Patients taking medications or having concomitant illnesses that are likely
to influence the natural course of ADPKD (e.g. nephrotoxic medications such as
chronic NSAID, cyclosporine, lithium and immunosuppressant use, and e.g.
diabetes mellitus and patients with proteinuria > 1 g/24hr).
• Patients, who receive renal function replacement therapy
• Pregnancy at moment of inclusion
• Patients with a life expectancy less than 1 year at moment of inclusion
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL43496.042.13 |