The primary objective is to gain information regarding the natural course of SFN and the change over time of the diagnostics using clinimetrically sound outcome measures representing various levels of outcome assessment. The secondary objective is…
ID
Source
Brief title
Condition
- Peripheral neuropathies
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary endpoint is an electronic web-based registry, which reflects the
natural course of SFN and change over time in the diagnostics of SFN.
Changes in neurological examination, nerve conduction studies, IENFD,
quantitative sensory testing and their relation to SFN-related symptoms, daily
functioning and QoL will be demonstrated.
Secondary outcome
The secondary endpoint is if and what diagnostics could ultimately be used in
future trials.
Background summary
Small fiber neuropathy (SFN) is characterized by neuropathic pain and
dysautonomia and may lead to limitations in activity and participation with a
decrement in quality of life (QoL). Increasing knowledge in the underlying
pathophysiological mechanisms leads to the development of more targeted
therapies. However, before treatments can be investigated in clinical trials, a
better understanding regarding the natural clinical course is needed. In
addition, it is crucial to determine whether applied outcome measures show
sensitivity to capture what is considered clinically meaningful responsiveness.
A national registry for SFN patients including diagnostics will provide
long-term follow-up data to achieve clinical trial readiness.
Study objective
The primary objective is to gain information regarding the natural course of
SFN and the change over time of the diagnostics using clinimetrically sound
outcome measures representing various levels of outcome assessment. The
secondary objective is to determine if and which diagnostics will be
ultimately used in future trials.
Study design
Prospective longitudinal study.
Study burden and risks
The online questionnaires will take about 30 minutes per year. The 100 patients
will yearly visit the SFN expertise center during 3 years. The diagnostics will
take 90 minutes per visit including a skin biopsy, carrying very low risk of
infection or persistent bleeding. SFN patients will get insight into the
natural course of their own disease. This can be helpful in daily living, in
participation and for coping. In addition, the assessment of various outcome
measures, including patient reported outcomes, gives the patient the
possibility to express their experience regarding impact of their illness on
their daily living and quality of life. The registry will facilitate in trial
design and recruitment, since information will be gathered regarding the most
sensitive metrics to be used.
P. Debyelaan 25 25
Maastricht 6229 HX
NL
P. Debyelaan 25 25
Maastricht 6229 HX
NL
Listed location countries
Age
Inclusion criteria
Definite small fiber neuropathy (SFN):
a) Subjects of 18 years and older
b) Clinical symptoms and signs of SFN
c) Abnormal intraepidermal nerve fiber density (IENFD) in skin biopsy of the
distal leg
d) Subjects must give informed consent by signing and dating an informed
consent form
Probable SFN:
a) Subjects of 18 years and older
b) Clinical symptoms and signs of SFN
c) Normal IENFD in skin biopsy of the distal leg
d) Abnormal quantitative sensory testing (QST)
e) Subjects must give informed consent by signing and dating an informed
consent form
Exclusion criteria
a) Signs of large nerve fiber neuropathy in neurological examination (weakness,
loss of vibration sense, hypo-/areflexia)
b) Abnormal nerve conduction studies
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 | NL83155.068.22 |