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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02638207
Registration number
NCT02638207
Ethics application status
Date submitted
16/12/2015
Date registered
23/12/2015
Date last updated
16/02/2021
Titles & IDs
Public title
Study to Evaluate Safety and Efficacy of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly (Radiculo) Neuropathy
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Scientific title
Prospective, Double-blind, Randomized, Multicenter Phase III Study Evaluating Efficacy and Safety of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly(Radiculo)Neuropathy
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Secondary ID [1]
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NGAM-08
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Universal Trial Number (UTN)
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Trial acronym
CIDP
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Chronic Inflammatory Demyelinating Poly(Radiculo)Neuropathy
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Condition category
Condition code
Neurological
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Other neurological disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - NewGam
Experimental: 0.5 g/kg NewGam - All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Experimental: 1.0 g/kg NewGam - All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Experimental: 2.0 g/kg NewGam - All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Treatment: Drugs: NewGam
In the Dose-evaluation Phase, all patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5, 1.0 or 2.0 g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). If a patient is randomized to receive the low or medium NewGam dose, the same volume with the same infusion rate as would have been applied in case the patient would have been randomized to 2.0 g/kg NewGam will be used, thus supplemented with an authorized 0.9% w/v isotonic sodium chloride solution as appropriate and detailed in the following infusion bag split to maintain the blinding
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score
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Assessment method [1]
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Efficacy - Proportion of responders in the 1.0 g/kg NewGam arm at Week 24 (Termination Visit) relative to baseline (Week 0). A responder being defined as a patient with a decrease of at least 1 point on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score (a scale from 0 to 10, from healthy to unable to make any purposeful movements with arms and/or legs)
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Timepoint [1]
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at Week 24
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Secondary outcome [1]
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Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score
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Assessment method [1]
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Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline compared to the 1.0 g/kg arm, based on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score
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Timepoint [1]
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at Week 24
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Secondary outcome [2]
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Grip Strength Score
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Assessment method [2]
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Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the grip strength (Martin Vigorimeter) using the previously published minimum clinically important difference (MCID) cut-off of 8 kilopascal (kPa)
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Timepoint [2]
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at Week 24
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Secondary outcome [3]
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Inflammatory Rasch-built Overall Disability Scale (I-RODS Score)
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Assessment method [3]
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Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the Inflammatory Rasch-built overall disability scale (I-RODS Score) using the MCID concept related to the varying standard errors (MCID-SE) as recently demonstrated
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Timepoint [3]
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at Week 24
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Secondary outcome [4]
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Worsening in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score
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Assessment method [4]
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Time to first confirmed worsening on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale by at least 1 point from the value at baseline (Week 0)
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Timepoint [4]
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Week 24
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Secondary outcome [5]
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Mean Change in Grip Strength
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Assessment method [5]
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Mean change from baseline (Week 0) to Termination Visit in grip strength of both hands (assessed by Martin vigorimeter). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point.
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Timepoint [5]
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Up to 24 weeks
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Secondary outcome [6]
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Inflammatory Rasch-built Overall Disability Scale (I-RODS)
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Assessment method [6]
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Mean change from baseline (Week 0) to Termination Visit in Inflammatory Rasch-built overall disability sum score (I-RODS using the concept of MCID-SE as recently reported) and number of improvers. The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point.
A scale from 0 to 48, from "Not possible to perform" to "Possible without any difficulty". Higher values represent a better outcome.
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Timepoint [6]
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Up to 24 weeks
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Secondary outcome [7]
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Motor Nerves
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Assessment method [7]
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Mean change from baseline (Week 0) to Termination Visit in sum of the distal evoked amplitude of 4 right sided and 4 left sided motor nerves (peroneal, tibial, ulnar and median). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point.
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Timepoint [7]
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Up to 24 weeks
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Secondary outcome [8]
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Mean Change in Pain Intensity Numerical Rating Scale (PI-NRS Scale)
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Assessment method [8]
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Mean change from baseline (Week 0) to Termination Visit in Pain Intensity Numeric Rating Scale (PI-NRS). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point.
The Pain Intensity Numeric Rating Scale (PI-NRS, a numeric scale where 0 = no pain and 10 = worst possible pain) is an 11-point scale for patient self-reporting of pain. A higher value represents a worse outcome.
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Timepoint [8]
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Up to 24 weeks
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Secondary outcome [9]
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Worsening on the Inflammatory Rasch-built Overall Disability Scale (I-RODS Scale)
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Assessment method [9]
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Time to first confirmed worsening on the I-RODS scale. The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. A scale from 0 to 48, from "Not possible to perform" to "Possible without any difficulty". Higher values represent a better outcome.
Worsening is determined using the concept of MCID (minimum clinically important difference) using the individually obtained standard errors (MCID-SE).
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Timepoint [9]
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24 weeks
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Secondary outcome [10]
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1 Point Decrease in the INCAT Disability Score
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Assessment method [10]
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Time to 1 point decrease (improvement of disability) in adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score
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Timepoint [10]
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24 weeks
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Secondary outcome [11]
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Decrease in Inflammatory Rasch-built Overall Disability Scale (I-RODS Scale)
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Assessment method [11]
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Time to decrease in Inflammatory Rasch-built overall disability scale (I-RODS) scores
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Timepoint [11]
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24 weeks
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Eligibility
Key inclusion criteria
1. Patients with diagnosis of definite or probable Chronic inflammatory demyelinating
polyneuropathy (CIDP) according to the European Federation of Neurological
Societies/Peripheral Nerve Society (EFNS/PNS) Guideline 2010 [van den Bergh et al.,
2010]; including patients with Multifocal Acquired Demyelinating Sensory And Motor
Neuropathy (MADSAM) or pure motor Chronic inflammatory demyelinating polyneuropathy
(CIDP )
2. Patients currently depending on treatment with immunoglobulins or corticosteroids
3. Patients with active disease, i.e. not being in remission, who are progressive or
relapsing prior to trial start or during the Wash-out Phase
4. Weakness of at least 2 limbs
5. >18 to <80 years of age
6. Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score between
2 and 9 (with a score of 2 coming exclusively from leg disability)
7. Voluntarily given, fully informed written consent obtained from patient before any
study-related procedures are conducted
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Minimum age
18
Years
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Maximum age
80
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Unifocal forms of Chronic inflammatory demyelinating polyneuropathy (CIDP)
2. Pure sensory Chronic inflammatory demyelinating polyneuropathy (CIDP)
3. Multifocal motor neuropathy (MMN) with conduction block [van den Bergh et al., 2010]
4. Patients who previously failed immunoglobulin treatment
5. Treatment with immunomodulatory/suppressive agents (cyclosporin, methotrexate,
mitoxantrone, mycophenolate mofetil or azathioprine) during the six months prior to
baseline visit
6. Patients on or treated with rituximab, alemtuzumab, cyclophosphamide, or other
intensive chemotherapeutic regimens, previous lymphoid irradiation or stem cell
transplantation during the 12 months prior to baseline visit
7. Respiratory impairment requiring mechanical ventilation
8. Myelopathy or evidence of central nervous system demyelination or significant
persisting neurological deficits from stroke, or central nervous system (CNS) trauma
9. Clinical evidence of peripheral neuropathy from another cause such as
1. connective tissue disease or systemic lupus erythematosus (SLE)
2. HIV infection, hepatitis, Lyme disease
3. cancer (with the exception of basal cell skin cancer)
4. IgM paraproteinemia with anti-myelin associated glycoprotein antibodies
10. Diabetic neuropathy
11. Cardiac insufficiency (New York Heart Association [NYHA] III/IV), cardiomyopathy,
significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic
heart disease
12. Severe liver disease (ALAT 3x > normal value)
13. Severe kidney disease (creatinine 1.5x > normal value)
14. Hepatitis B, hepatitis C or HIV infection
15. 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 deep vein thrombosis (DVT)
16. Body mass index (BMI) =40 kg/m2
17. Patients with uncompensated hypothyroidism (abnormally high Thyroid-Stimulating
Hormone [TSH] and abnormally low Thyroxine [T4]) or known vitamin B12 deficiency if
patients don't receive adequate substitution therapy
18. Medical conditions whose symptoms and effects could alter protein catabolism and/or
Immunoglobulin G (IgG) utilization (e.g. protein-losing enteropathies, nephrotic
syndrome)
19. Known Immunoglobulin A (IgA) deficiency with antibodies to Immunoglobulin A (IgA)
20. History of severe hypersensitivity, e.g. anaphylaxis or severe systemic response to
immuno-globulin, blood or plasma derived products, or any component of NewGam
21. Known blood hyperviscosity, or other hypercoagulable states
22. Use of other blood or plasma-derived products within three months prior to Visit 2
23. Patients with a past or present history of drug abuse or alcohol abuse within the
preceding five years prior to baseline visit
24. Patients unable or unwilling to understand or comply with the study protocol
25. Participation in another interventional clinical study with investigational medicinal
product (IMP) treatment currently or during the three months prior to Visit 2
26. Women who are breast feeding, pregnant, or planning to become pregnant, or are
unwilling to use an effective birth control method (such as implants, injectables,
combined oral contraceptives, some intrauterine devices (IUDs), sexual abstinence or
vasectomized partner) while on study
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
27/09/2017
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
5/09/2019
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Sample size
Target
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Accrual to date
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Final
142
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
Country [1]
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Bulgaria
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State/province [1]
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Blagoevgrad
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Bulgaria
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State/province [2]
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Sofia
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Canada
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State/province [3]
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Ontario
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Country [4]
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Canada
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State/province [4]
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Montréal
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Country [5]
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Czechia
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State/province [5]
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Hradec Králové
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Czechia
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State/province [6]
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Pardubice
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Czechia
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State/province [7]
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Prague
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Germany
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State/province [8]
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Goettigen
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Hungary
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State/province [9]
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Budapest
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Hungary
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State/province [10]
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Szeged
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Poland
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State/province [11]
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Lublin
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Poland
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State/province [12]
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Olsztyn
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Poland
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State/province [13]
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Wroclaw
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Romania
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State/province [14]
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Brasov
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Romania
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State/province [15]
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Bucharest
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Country [16]
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Romania
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State/province [16]
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Constanta
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Country [17]
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Russian Federation
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State/province [17]
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Kazan'
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Country [18]
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Russian Federation
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State/province [18]
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Moscow
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Country [19]
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Russian Federation
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State/province [19]
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Nizhny Novgorod
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Country [20]
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Russian Federation
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State/province [20]
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Saint Petersburg
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Country [21]
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Ukraine
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State/province [21]
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Ivano-Frankivs'k
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Country [22]
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Ukraine
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State/province [22]
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Kyiv
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Country [23]
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Ukraine
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State/province [23]
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Luts'k
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Country [24]
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Ukraine
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Vinnytsia
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Country [25]
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Ukraine
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State/province [25]
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Zaporizhzhya
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Octapharma
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Study to evaluate the Efficacy and Safety of Three Different Dosages of NewGam in Patients
With Chronic Inflammatory Demyelinating Poly(radiculo)neuropathy
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02638207
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Trial related presentations / publications
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Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed
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Contacts
Principal investigator
Name
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Wolfgang Frenzel, MD
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Address
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Octapharma
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Phone
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Fax
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02638207
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