The primary objective is to determine whether subjects with CIDP are overtreated with maintenance IVIg treatment and to reduce overtreatment-associated subjects* burden and health care costs.
ID
Source
Brief title
Condition
- Peripheral neuropathies
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
For the primary outcome we will use the Rasch-Overall Disability Scale (R-ODS),
a patient self-report linearly weighted scale that measures activity and social
participation limitations.
Secondary outcome
1) The proportion of subjects remaining stable on their individual R-ODS score
and completing the follow-period. A subject will be considered stable if he/she
does not reach the individual predefined limits of a clinically important
deterioration on the R-ODS during follow-up.
2) Muscle strength using the Medical Research Council (MRC) sum score of 12
predefined muscle groups (range 0 to 60, including shoulder abduction, elbow
flexion, wrist extension, hip flexion, knee extension and foot dorsiflexion).
3) Grip strength, measured in kPa by a Martin-Vigorimeter.
4) Sensory impairment using the modified INCAT Sensory Sum Score (INCAT*SS,
range 0-20).
5) Subject*s perception of clinical deterioration on a 5-point Likert scale.
6) Disase-non-specific disability using the AMC Linear Disability Scale (ALDS,
range 0 [dead] to 100 [fully able]).
7) Quality of life using Short Form-36 (SF-36).
8) Pain using the Pain-Intensity Numerical Rating Scale (PI-NRS, a 11-point
scale).
9) Fatigue using a 7-item linear modified Rasch-built fatigue scale.
10) Costs of health care use, costs of production loss, and out-of-pocket
expenses.
11) Difference between serum IgG levels before and after last IVIg infusion
prior to first study treatment.
Background summary
IVIg is an efficacious treatment for CIDP but there is no evidence on how long
maintenance treatment should be administered. CIDP runs different disease
courses including monophasic, relapsing-remitting and chronic courses. In some
patients treated with IVIg, there is no disease activity making ongoing
maintenance treatment unnecessary. The only way to assess whether treatment is
needed is to try and stop treatment. There is however reluctance to perform
IVIg withdrawal attempts because there is a chance of temporary deterioration
in IVIg-dependent patients. Several recent studies have showed that there is
overtreatment with IVIg in patients with CIDP. This can lead to unnecessary
adverse events, discomfort of frequent infusions and very high health care
cost.
Study objective
The primary objective is to determine whether subjects with CIDP are
overtreated with maintenance IVIg treatment and to reduce
overtreatment-associated subjects* burden and health care costs.
Study design
A multicentre, randomized, double-blind, standard treatment-controlled
non-inferiority trial.
Intervention
1) IVIg withdrawal (tapering consists of 3 infusions (75%, 50% and respectively
25% of the subjects* prestudy IVIg dose combined with placebo) which will be
followed by 100% placebo infusions . Placebo will consist of sodium chloride
0.9% in comparable amounts and intervals as the previous IVIg treatment.
2) Comparative treatment will be the standard treatment in which subjects will
be randomised to IVIg will receive the same IVIg infusions (dose and interval)
as prior to the study.
Study burden and risks
The risk of this study is considered *negligible*. Upon IVIg withdrawal a
certain number of patients will deteriorate. The exact number is unknown and
this is one of the main research questions. Participation in this trial is not
associated with an additional risk for increases of disability as withdrawal
attempts are part of daily care. It is generally considered that deterioration
during withdrawal is temporary and can be counteracted by re-instituting IVIg
treatment. Most patients, if not all, are expected to reach their baseline
level in less than 6 weeks.
The risks for participants will be minimized by close clinical monitoring,
using patient-relevant outcomes to measure deterioration and immediate
restarting of IVIg treatment when a confirmed worsening has occurred. We have
developed a pragmatic restabilisation protocol allowing higher IVIg dosages if
these are deemed necessary by the treating physician.
Participation in this trial has some other minor risks such as changing of
individual home care nurse in some patients possibly leading to more difficult
intravenous access. Measurements during the trial are not considered invasive,
except for vena punctures.
Unknown risks to participants related to the study and aside of the risk of
deterioration are not expected as patients would have normally continued
maintenance treatment.
Meibergdreef 9
Amsterdam 1105 AZ
NL
Meibergdreef 9
Amsterdam 1105 AZ
NL
Listed location countries
Age
Inclusion criteria
1) Probable or definite CIDP according to the EFNS/PNS criteria 2010
2) Stable disease for 6 months (e.g. no progression of disease in last 6 months)
3) IVIg treatment for at least 6 months
4) IVIg infusion interval of 2 to 6 weeks.
5) Age of at least 18 years
Exclusion criteria
1) Deterioration after IVIg withdrawal in the last 12 months
2) Changes in IVIg treatment dose/interval in last 6 months
3) Change of additional CIDP treatment, if any, in the last 3 months (e.g. corticosteroids or immunosuppressive treatment)
4) A prolonged period (> 6 weeks) of disability increase following an earlier IVIg withdrawal attempt
5) History of respiratory failure related to CIDP
6) Legally incompetent
7) Lack of written informed consent
Design
Recruitment
Medical products/devices used
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 |
|---|---|
| EudraCT | EUCTR2013-005363-52-NL |
| CCMO | NL47383.018.14 |