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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 18 May 2020
Main ID:  EUCTR2017-002511-34-GB
Date of registration: 08/11/2019
Prospective Registration: No
Primary sponsor: Academic Medical Centre, Amsterdam, NL
Public title: Initial treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) - combined immunoglobulin and steroid treatment versus immunoglobulin treatment alone.
Scientific title: Intravenous immunoglobulin and intravenous methylprednisolone as optimal induction treatment in CIDP - OPTIC Trial
Date of first enrolment: 01/04/2019
Target sample size: 96
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-002511-34
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: yes Placebo: yes Other: no Number of treatment arms in the trial: 2  
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Netherlands United Kingdom
Contacts
Name: Laura Zambreanu   
Address:  8-11 Queen Square WC1N 3BG London United Kingdom
Telephone: 07799626653
Email: l.zambreanu@nhs.net
Affiliation:  MRC Centre for Neuromuscular Diseases, National Hospital for Neurology and Neurosurgery, UCLH
Name: Laura Zambreanu   
Address:  8-11 Queen Square WC1N 3BG London United Kingdom
Telephone: 07799626653
Email: l.zambreanu@nhs.net
Affiliation:  MRC Centre for Neuromuscular Diseases, National Hospital for Neurology and Neurosurgery, UCLH
Key inclusion & exclusion criteria
Inclusion criteria:
1) Probable or definite CIDP according to the EFNS/PNS criteria 2010 (all CIDP phenotypes).
2) Age = 18 years.
3a) Treatment naïve patients (no previous immune treatment for CIDP); or
3b) Previously immune-treated patients who have a relapse after a remission of at least 1 year; or
3c) Patients treated with subjective or objective improvement after a single loading course of IVIg in the last 3 months, and subsequent deterioration as judged by their treating physician.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 45
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion criteria:
1) Presence of IgM paraproteinemia, anti-MAG antibodies or CIDP-specific antibodies associated with poor treatment response to IVIg.
2) Use of drugs associated with a demyelinating neuropathy.
3) Use of any immunosuppressive or immunomodulatory drugs in previous 6 months (except for a single loading dose of IVIg within 3 months or low dose prednisolone (20 mg or less) during a short period (maximum duration of two weeks).
4) Known serious adverse events with previous IVIg or corticosteroid treatment.
5) One of more of the risk factors associated with increased risk of adverse events 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).
6) History of osteoporosis or osteoporotic fractures.
7) Known active malignancy, currently treated with chemotherapy or immunomodulatory drugs, or with a life expectancy of less than 1 year. .
8) Bodyweight more than 120 kg.
9) Pregnancy or nursing mother; intention to become pregnant during the course of the study; female patients of childbearing potential (from menarche until 1 year after the last period, surgical menopause or sterilization procedure) either not using or not willing to use a medically reliable method of contraception for the entire duration of the study.
10) Known cataract or cataract obvious on fundoscopy.
11) Current psychosis or past history of psychosis.
12) Poor dental status.
13) Known pulmonary embolism or other deep venous thrombosis in patient’s medical history, without current anticoagulant therapy.
14) Patient lacking capacity to consent to enter the trial.
15) Lack of written informed consent


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
MedDRA version: 21.1 Level: PT Classification code 10057645 Term: Chronic inflammatory demyelinating polyradiculoneuropathy System Organ Class: 10029205 - Nervous system disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Intervention(s)

Trade Name: methylprednisolone
Product Name: methylprednisolone
Pharmaceutical Form: Powder and solvent for solution for injection/infusion
Pharmaceutical form of the placebo: Solution for infusion
Route of administration of the placebo: Intravenous use

Trade Name: sodium chloride 0.9%
Product Name: sodium chloride 0.9%
Pharmaceutical Form: Solution for infusion

Primary Outcome(s)
Main Objective: Does initial treatment with both intravenous immunoglobulin and intravenous methylprednisolone lead to more frequent long-term remission (defined as sustained improvement without need for further treatment) in patients with chronic inflammatory demyelinating polyneuropathy (CIDP) compared to initial treatment with intravenous immunoglobulin alone?
Primary end point(s): The primary outcome measure is the number of patients in remission at 1 year after start of an 18 weeks treatment period.

Remission is defined as sustained improvement without receiving any further immune treatment (as defined below under treatment failure).

Improvement is defined as improvement by at least the minimal clinical important difference (MCID) on the inflammatory Rasch-Overall Disability Scale (I-RODS), and/or improvement of one or more points on the Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale, at 18 weeks compared to baseline.

Sustained is defined as no deterioration between 18 weeks and 52 weeks, where deterioration is defined as worsening on the I-RODS by at least the MCID and/or on the INCAT disability scale by one or more points.

A patient will be considered as a treatment failure and therefore not in remission if they 1) receive additional immune treatment for CIDP (other than that in the protocol) during the 18-week intervention period, 2) have not improved at 18 weeks, 3) receive any immune treatment for CIDP for any reason between 18 and 52 weeks, or 4) do not show a sustained improvement at 52 weeks as defined above.
Secondary Objective: Compared to intravenous immunoglobulin alone, does initial combination treatment with intravenous immunoglobulin and intravenous methylprednisolone in chronic inflammatory demyelinating polyneuropathy (CIDP) patients lead to:
1. higher rates of improvement?
2. faster speed of improvement?
3. less healthcare costs?
Timepoint(s) of evaluation of this end point: 1 year after the start of an 18 week treatment period.
Secondary Outcome(s)
Secondary end point(s): 1) The number of patients with improvement on disability equal or more than the minimal clinically important difference (MCID)
2) Time to improvement (= MCID) on disability;
3) Mean change in disability;
4) Mean change in grip strength;
5) Mean change in muscle strength by MRC sum score;
6) Mean change in sensory impairment;
7) Mean change in fatigue;
8) Mean change in pain;
9) Mean change in health related quality of life (HRQL);
10) Number of (serious) adverse events (including corticosteroid associated adverse events);
11) Care use and overall healthcare-related costs.
Timepoint(s) of evaluation of this end point: Secondary endpoints will be assessed at 18 and 52 weeks, or earlier if a preliminary endpoint (treatment failure) is reached (i.e. additional treatment is started during the 18 week treatment period or between 18 and 52 weeks).
Secondary ID(s)
2017-002511-34-NL
ISRCTN15893334
v3.0.15JAN2019
Source(s) of Monetary Support
GBS/CIDP Foundation International
Princes Beatrix Spierfonds
Sanquin
ZonMW
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 01/04/2019
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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