Primary objective is to define and validate a patient perceived minimal clinical important difference (pMCID) on a linear disability scale. Secondary objectives are to determine whether pMCID during improvement is larger compared to the pMCID during…
ID
Source
Brief title
Condition
- Peripheral neuropathies
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
To define treatment response in CIDP using patient pMCID versus traditional
MCID cut-offs on a linear disability scale.
Secondary outcome
1) To determine whether pMCID during improvement is larger than the pMCID
during deterioration.
2) To determine the margins of score fluctuation on the I-RODS during follow-up
in patients with stable disease.
Background summary
Currently, improvement and deterioration in chronic inflammatory demyelinating
polyradiculoneuropathy (CIDP) is defined by clinical outcome measures because
biomarkers of disease activity are lacking. Despite various outcome measures,
it remains unclear when patients should be considered a *responder to
treatment*. Better definitions of *responder to treatment* are needed to design
better trials and identify new treatment strategies, but also to provide
guidance to tailor treatment in daily practice. Ideally, patients* voice should
be centralized in the definition of treatment response, rather than relying on
fixed and predefined cut-offs currently (statistical MCID or sMCID) used in
clinical trial designs.
Study objective
Primary objective is to define and validate a patient perceived minimal
clinical important difference (pMCID) on a linear disability scale. Secondary
objectives are to determine whether pMCID during improvement is larger compared
to the pMCID during deterioration and to determine the margins of natural
fluctuation on disability scales during follow-up of patients with stable
disease.
Study design
Observational longitudinal study with a follow-up of 24 weeks
Study burden and risks
The burden associated with this observational study is limited to four to six
self -assessed measurements at home consisting of completing questionnaires and
measuring grip strength plus three to four visits to the hospital, which will
be embedded as part of the routine evaluation of patients otherwise performed
outside the study. Part of the outcome measures used in this study are also
used in routine clinical practice. There are no risks related to participation
in this study.
Meibergdreef 9
Amsterdam 1105 AZ
NL
Meibergdreef 9
Amsterdam 1105 AZ
NL
Listed location countries
Age
Inclusion criteria
Possible, probable or definite clinical and electrophysiological EFNS/PNS
criteria for CIDP (Group 1 and 2).5 In addition:
A) Patients in Group 1 need to have active disease (history of progression
justifying start or change of treatment as determined by treating physician).
B) Patients in Group 2 with stable disease for three months on treatment with
IVIg and/or corticosteroids.
OR
have a polyneuropathy associated with monoclonal IgM paraproteinemia (with or
without anti-MAG antibodies) (Group 3, controls).
- Adult males or females (18 year or more).
Exclusion criteria
- Any concomitant disease (e.g. joint problems) or condition (e.g. misuse of
alcohol) that might interfere with functionality of the patient, which could
potentially interfere with capturing MCID changes related predominantly to CIDP.
- Legally incompetent adults
- No informed consent
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 | NL69897.018.19 |