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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: 26 February 2018
Main ID:  EUCTR2009-017805-13-RO
Date of registration: 15/07/2010
Prospective Registration: Yes
Primary sponsor: Octapharma AG
Public title: "PROSPECTIVE, PARALLEL GROUP, DOUBLE-BLIND, PLACEBO-CONTROLLED, RANDOMIZED, MULTICENTER, ADAPTIVE, TWO-STAGE PHASE II/III STUDY EVALUATING SAFETY AND EFFICACY OF THREE DIFFERENT DOSAGES OF NEWGAM IN PATIENTS WITH CHRONIC INFLAMMATORY DEMYELINATING POLYRADICULONEUROPATHY (POINT TRIAL)” - NGAM-03
Scientific title: "PROSPECTIVE, PARALLEL GROUP, DOUBLE-BLIND, PLACEBO-CONTROLLED, RANDOMIZED, MULTICENTER, ADAPTIVE, TWO-STAGE PHASE II/III STUDY EVALUATING SAFETY AND EFFICACY OF THREE DIFFERENT DOSAGES OF NEWGAM IN PATIENTS WITH CHRONIC INFLAMMATORY DEMYELINATING POLYRADICULONEUROPATHY (POINT TRIAL)” - NGAM-03
Date of first enrolment: 08/09/2010
Target sample size: 172
Recruitment status: Not Recruiting
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-017805-13
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: yes
Other trial design description: prospective
If controlled, specify comparator, Other Medicinial Product: no
Placebo: yes
Other: yes
Other specify the comparator: three dosage forms
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Bulgaria Czech Republic Romania
Contacts
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Key inclusion & exclusion criteria
Inclusion criteria:
•Patients diagnosed as having CIDP by a neurologist experienced in neuromuscular diseases based on fulfillment of clinical criteria of the INCAT Group and the definite electrophysiological criteria for CIDP of the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS); patients with Multifocal Acquired Demyelinating Sensory And Motor neuropathy (MADSAM) or pure motor CIDP will be included
•Worsening of disability and objective increase in weakness or sensory deficit during the 6 months prior to screening
• =18 years of age
•Overall adjusted INCAT disability score between 2-9 (see appendix 1); a score of 2 has to result exclusively from leg disability
•Expanded Medical Research Council (MRC) sumscore of = 78 (see appendix 2)
•Freely given written informed consent from patient
•Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study
•Women of reproductive age: negative result of pregnancy test (human chorionic gonadotropin [HCG]-based assay in urine) and either surgically sterile, sexually inactive, or willing to use adequate contraception for the duration of the trial.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
•Unifocal forms of CIDP
•Pure sensory CIDP
•Multifocal motor neuropathy (MMN) with conduction block, defined as a lower motor neuron disorder with motor weakness in an upper limb, without sensory deficit and with proximal conduction block (50% decrease in amplitude/area with proximal compared with distal stimulation) in motor nerves and normal sensory nerve conduction studies (NCS)
•Treatment of CIDP with immunoglobulins (intravenous or subcutaneous) at any time prior to study entry
•Steroids of any type equivalent to prednisolone or prednisone > 10 mg/day or equivalent, e.g., > 20 mg every 2 days or plasma exchange (PE) during the last 3 months prior to baseline visit
•Treatment with azathioprine during 12 months prior to baseline visit unless on a stable dose throughout at least 12 months prior to baseline. Patients who are on a stable dose for at least 12 months prior to baseline should be expected to continue taking the same dose during the trial.
•Treatment with cyclosporin, methotrexate, mitoxantrone, mycophenolate mofetil, interferon or other immunosuppressive or immunomodulatory drugs during the three months prior to baseline visit
•Patients ever treated with rituximab, alemtuzumab, cyclophosphamide, or other intensive chemotherapeutic regimens, previous lymphoid irradiation or stem cell transplantation
•Respiratory impairment requiring mechanical ventilation
•Myelopathy or clinical evidence of central nervous system (CNS) demyelination, trauma or stroke
•Clinical evidence of peripheral neuropathy from another cause such as
-connective tissue disease or systemic lupus erythematosus,
-HIV infection, hepatitis, Lyme disease,
-cancer (with the exception of benign skin cancer), lymphoma, malignant plasma cell dyscrasia, Castleman’s disease
-IgM paraproteinaemia with anti-myelin associated glycoprotein antibodies
•Known diabetes mellitus
•Other serious medical condition complicating assessment or treatment including but not limited to
ocardiac insufficiency (New York Heart Association [NYHA] III/IV), known cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease,
osevere liver disease (ALAT 3x > upper limit of normal for the testing laboratory),
osevere kidney disease (creatinine > 120 µM),
ohepatitis B or C or HIV infection
•Thromboembolic events: patients with a history of deep vein thrombosis (DVT) within the last year prior to baseline visit or pulmonary embolism ever; patients with susceptibility to embolism or DVT
•Patients with known uncompensated hypothyroidism (abnormally high Thyroid-Stimulating Hormone (TSH), and abnormally low thyroxine (T4)) or known vitamin B12 deficiency
•Medical conditions whose symptoms and effects could alter protein catabolism and/or IgG utilization (e.g. protein-losing enteropathies, nephrotic syndrome)
•Known IgA deficiency with antibodies to IgA
•History of hypersensitivity, anaphylaxis or severe systemic response to immunoglobulin, blood or plasma derived products, or any component of NewGam
•Known blood hyperviscosity, or other hypercoagulable states
•Operation within 3 months prior to baseline visit in which blood products have been used
•Patients with a past or present history of drug abuse or alcohol abuse
•Patients unable or unwilling to understand or comply with the study protocol
•Patients with any condition that would make the patient, in the opinion of the Investigator, unsuitable for the study
•Live viral vaccination within


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
MedDRA version: 12.1 Level: LLT Classification code 10057645 Term: Chronic inflammatory demyelinating polyradiculoneuropathy
Intervention(s)

Product Name: NewGam
Product Code: NewGam
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Immunoglobuline G
Other descriptive name: NewGam
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: range
Concentration number: 86-110
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Intravenous use

Primary Outcome(s)
Main Objective: Stage 1 (phase II dose-finding part):
•Determination and selection of one dosage from the three NewGam maintenance dosage arms in comparison with a placebo arm, based on the percentage of responders (defined as a decrease in the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score by at least 1 point) at week 24. The selected NewGam dosage and placebo will be employed and compared in Stage 2.

Stage 2 (phase III confirmatory part):
•To demonstrate superiority of the dosage regimen selected at study Stage 1 (out of 0.4 g/kg, 1.0 g/kg or 2.0 g/kg NewGam) over placebo in patients with CIDP as assessed by response rate at week 24 (responder is defined as a decrease in the adjusted INCAT disability score by at least 1 point).
Primary end point(s): Efficacy:
Response relative to baseline at 24 weeks or at time of rescue NewGam infusion using the adjusted INCAT disability score. Response will be defined as a decrease of at least 1 point on the adjusted INCAT disability score, a scale from 0 to 10 (from healthy to unable to make any purposeful movements with arms or legs in which the change from 0 to 1 in the upper limb component is discounted, see appendix 1).
The results from the Stage 1 (dose-finding phase II part) of this seamless phase II/III trial will be used to adopt the sample size in the Stage 2 (efficacy phase III part), and also to only continue - in addition to the placebo arm - with the NewGam arm that is the most efficacious as shown by IA.
Secondary Objective: To evaluate the safety and efficacy of NewGam administration in patients with CIDP through other parameters
Secondary Outcome(s)
Secondary ID(s)
2009-017805-13-CZ
NGAM-03
Source(s) of Monetary Support
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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