Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Nervous System Diseases Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Conditions
Interventions
All participants are treated with intravenous IVIg, which is considered standard care. In addition, participants are randomized to receive either 7 infusions methylprednisolone (IVMP) 1000 mg in sodiu
Sponsors
Academic Medical Center (AMC)
Eligibility
Sex/Gender
All
Age
18 Years to 100 Years
Inclusion criteria
Inclusion criteria: 1. Probable or definite CIDP according to the EFNS/PNS criteria 2010 (all CIDP phenotypes) 2. Age = 18 years 3.1. Treatment naïve patients; or 3.2. Previously treated patients who have a relapse after a remission of at least 1 year; or 3.3. Patients treated with subjective or objective improvement after a single loading dose of IVIg in the last 3 months, and subsequent deterioration as judged by his or her treating physician.
Exclusion criteria
Exclusion criteria: Current exclusion criteria as of 22/11/2022: 1. Presence of IgM paraproteinemia and/or anti-MAG antibodies or known CIDP-specific antibodies associated with poor treatment response to IVIg 2. Use of drugs known to cause a demyelinating neuropathy 3. Use of any immunosuppressive or immunomodulatory drugs in the previous 6 months (except for a single loading dose of IVIg within 3 months or low dose prednisolone (20 mg or less) for a short period not exceeding two weeks) 4. Known serious adverse events (SAEs) with previous IVIg or corticosteroid treatment. Hypersensitivity to methylprednisolone or any component of the formulation. Hypersensitivity to the human immunoglobulins or to any of the excipients. Selective IgA deficiency patients who developed antibodies to IgA 5. Systemic fungal infections, unless specific anti-infective therapy is employed 6. Known hyperprolinaemia type I or II or known fructose intolerance 7. One or more of the risk factors associated with increased risk of AEs of IVIg or IVMP or conditions that could lead to unblinding of treatment (i.e. diabetes; IgA deficiency; gastric ulcers; psychosis; severe hypertension (180/110 mmHg or more on repeated measurements); hypocalcaemia (lower than 2.20 mmol/L, corrected for albumin); moderate or severe heart failure; severe cardiovascular disease (i.e. more than one myocardial infarction and or ischemic stroke); renal failure (glomerular filtration rate < 30 ml/min) 8. History of osteoporosis or osteoporotic fractures 9. Known active malignancy, currently treated with chemotherapy or immunomodulatory drugs, or with a life expectancy of less than one year 10. Bodyweight more than 120 kg 11. Pregnancy or nursing mother; intention to become pregnant during the course of the study; female patients of childbearing potential either not using or not willing to use a medically reliable method of contraception for the entire duration of the study. A woman is considered of childbearing potential from menarche and until becoming post-menopausal, unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. Acceptable methods of contraception are: combined (oestrogen and progestogen-containing) hormonal contraception associated with inhibition of ovulation (whether oral, intravaginal or transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (whether oral, injectable or implantable), progestogen-only oral hormonal contraception, where inhibition of ovulation is not the primary mode of action, male or female condom with or without spermicide, cap or diaphragm, intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner (provided that partner is the sole sexual partner of the trial participant and that the vasectomised partner has received medical assessment of the surgical success) 12. Known cataract requiring surgery (previously: Known cataract or cataract obvious on fundoscopy) 13. Current psychosis or past history of psychosis 14. Poor dental status 15. Known pulmonary embolism or other deep venous thrombosis in patient’s medical history, without current anticoagulant therapy 16. Adults lacking capacity to give informed consent (IC) 17. Lack of written IC _____ Previous exclusion criteria as of 30/05/2018, to reflect c
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The number of patients in remission is a value determined 1 year after start of the intervention period by accounting for the patients who fit the criteria for remission. Remission is defined as: sustained improvement without the need for further treatment. Improvement is defined as improvement by at least the minimal clinical important difference (MCID) on the inflammatory Rasch Disability Scale (I-RODS) and/or improvement (ie, decrease) of one point or more on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale at 18 weeks compared to baseline. Sustained is defined as no deterioration between week 18 and week 52, i.e. difference on the I-RODS of less than the individual MCID difference and/or no increase on the adjusted INCAT disability scale. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary parameters are assessed at 18 and 52 weeks, or earlier if a preliminary endpoint is reached: 1. The number of patients with improvement on disability equal or more than the MCID: disability is measured using the iRODS and the INCAT disability scale, the number of patients are calculated by counting the patients who fit the definition of improvement 2. Time to improvement (= MCID) on disability: time to improvement is calculated and given in days and/or weeks 3. Mean change in disability: disability is measured using the iRODS and INCAT disability scale 4. Mean change in grip strength: grip strength (in kPa) is measured using a handheld (Martin) Vigorimeter 5. Mean change in muscle strength: The Medical Research Council (MRC) sum score of 12 predefined muscle groups (including shoulder abduction, elbow flexion, wrist extension, hip flexion, knee extension and foot dorsiflexion) is used to measure muscle strength 6. Mean change in sensory impairment: measured using the Inflammatory Neuropathy Cause and Treatment sensory sumscore (INCAT-SS) 7. Mean change in fatigue: measured using the Fatigue Severity Scale (FSS) 8. Mean change in pain: measured using the pain intensity numeric rating scale (PI-NRS) 9. Mean change in health related quality of life (HRQL): measured using the EuroQol 10. Number of (serious) adverse events (including corticosteroid associated adverse events): number of (serious) adverse events (including corticosteroid associated adverse events) are measured using a structured questionnaire with short term corticosteroid and IVIg related AE’s at the 18 week visit. AEs are scored as mild, moderate or severe by the investigator. Long-term corticosteroid related AEs will be filled in by the investigator at 52 weeks (secondary outcome) and 104 weeks (safety follow-up). Adverse events questionnaires will be filled in only after completion of all other outcome assessments. 11. Care use and overall healthcare-related costs: measured using the workin | — |
Countries
England, Netherlands, Scotland, United Kingdom
Contacts
Public ContactIris van Doorn
Outcome results
None listed