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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02638207




Registration number
NCT02638207
Ethics application status
Date submitted
16/12/2015
Date registered
18/12/2015
Date last updated
4/02/2019

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
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
Secondary ID [1] 0 0
NGAM-08
Universal Trial Number (UTN)
Trial acronym
CIDP
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Chronic Inflammatory Demyelinating Poly(Radiculo)Neuropathy 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
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

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) disability score - 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)
Timepoint [1] 0 0
at Week 24
Secondary outcome [1] 0 0
Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) disability score - 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
Timepoint [1] 0 0
at Week 24
Secondary outcome [2] 0 0
Grip Strength Score - 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)
Timepoint [2] 0 0
at Week 24
Secondary outcome [3] 0 0
Inflammatory Rasch-built overall disability scale (I-RODS Score) - 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
Timepoint [3] 0 0
at Week 24
Secondary outcome [4] 0 0
Worsening in the Inflammatory Neuropathy Cause and Treatment (INCAT) disability score - 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)
Timepoint [4] 0 0
through study completion, an average of 2 years
Secondary outcome [5] 0 0
Mean Change in grip strength - Mean change from baseline (Week 0) to Termination Visit in grip strength of both hands (assessed by Martin vigorimeter)
Timepoint [5] 0 0
through study completion, an average of 2 years
Secondary outcome [6] 0 0
Inflammatory Rasch-built overall disability scale (I-RODS) - 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; Appendix 1) and number of improvers
Timepoint [6] 0 0
through study completion, an average of 2 years
Secondary outcome [7] 0 0
Right sided motor nerves - 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)
Timepoint [7] 0 0
through study completion, an average of 2 years
Secondary outcome [8] 0 0
Mean Change in pain intensity numerical rating scale (PI-NRS Scale) - Mean change from baseline (Week 0) to Termination Visit in Pain Intensity Numeric Rating Scale (PI-NRS)
Timepoint [8] 0 0
through study completion, an average of 2 years
Secondary outcome [9] 0 0
Worsening on the Inflammatory Rasch-built overall disability scale (I-RODS Scale) - Time to first confirmed worsening on the I-RODS scale
Timepoint [9] 0 0
through study completion, an average of 2 years
Secondary outcome [10] 0 0
1 point decrease in the INCAT disability score - Time to 1 point decrease (improvement of disability) in adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score
Timepoint [10] 0 0
through study completion, an average of 2 years
Secondary outcome [11] 0 0
decrease in Inflammatory Rasch-built overall disability scale (I-RODS Scale) - Time to decrease in Inflammatory Rasch-built overall disability scale (I-RODS) scores
Timepoint [11] 0 0
through study completion, an average of 2 years

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
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
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
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Austin Health - Heidelberg
Recruitment hospital [2] 0 0
Royal Melbourne Hosptial - Parkville
Recruitment hospital [3] 0 0
St. Vincent's Hospital Melbourne - Fitzroy
Recruitment postcode(s) [1] 0 0
3084 - Heidelberg
Recruitment postcode(s) [2] 0 0
3050 - Parkville
Recruitment postcode(s) [3] 0 0
3065 - Fitzroy
Recruitment outside Australia
Country [1] 0 0
Bulgaria
State/province [1] 0 0
Blagoevgrad
Country [2] 0 0
Bulgaria
State/province [2] 0 0
Plovdiv
Country [3] 0 0
Bulgaria
State/province [3] 0 0
Sofia
Country [4] 0 0
Canada
State/province [4] 0 0
Ontario
Country [5] 0 0
Canada
State/province [5] 0 0
Quebec
Country [6] 0 0
Canada
State/province [6] 0 0
Montréal
Country [7] 0 0
Czechia
State/province [7] 0 0
Brno
Country [8] 0 0
Czechia
State/province [8] 0 0
Hradec Králové
Country [9] 0 0
Czechia
State/province [9] 0 0
Pardubice
Country [10] 0 0
Czechia
State/province [10] 0 0
Prague
Country [11] 0 0
Denmark
State/province [11] 0 0
Aarhus
Country [12] 0 0
Germany
State/province [12] 0 0
Essen
Country [13] 0 0
Germany
State/province [13] 0 0
Goettigen
Country [14] 0 0
Germany
State/province [14] 0 0
Leipzig
Country [15] 0 0
Hungary
State/province [15] 0 0
Budapest
Country [16] 0 0
Hungary
State/province [16] 0 0
Gyor
Country [17] 0 0
Hungary
State/province [17] 0 0
Pécs
Country [18] 0 0
Hungary
State/province [18] 0 0
Szeged
Country [19] 0 0
Poland
State/province [19] 0 0
Lublin
Country [20] 0 0
Poland
State/province [20] 0 0
Olsztyn
Country [21] 0 0
Poland
State/province [21] 0 0
Wroclaw
Country [22] 0 0
Romania
State/province [22] 0 0
Brasov
Country [23] 0 0
Romania
State/province [23] 0 0
Bucharest
Country [24] 0 0
Romania
State/province [24] 0 0
Cluj-Napoca
Country [25] 0 0
Romania
State/province [25] 0 0
Constanta
Country [26] 0 0
Russian Federation
State/province [26] 0 0
Kazan'
Country [27] 0 0
Russian Federation
State/province [27] 0 0
Moscow
Country [28] 0 0
Russian Federation
State/province [28] 0 0
Nizhny Novgorod
Country [29] 0 0
Russian Federation
State/province [29] 0 0
Saint Petersburg
Country [30] 0 0
Sweden
State/province [30] 0 0
Lund
Country [31] 0 0
Ukraine
State/province [31] 0 0
Ivano-Frankivs'k
Country [32] 0 0
Ukraine
State/province [32] 0 0
Kyiv
Country [33] 0 0
Ukraine
State/province [33] 0 0
Luts'k
Country [34] 0 0
Ukraine
State/province [34] 0 0
Vinnytsia
Country [35] 0 0
Ukraine
State/province [35] 0 0
Zaporizhzhya

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Octapharma
Address
Country

Ethics approval
Ethics application status

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
Trial website
https://clinicaltrials.gov/show/NCT02638207
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Wolfgang Frenzel, MD
Address 0 0
Octapharma
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Mikaela Grupp
Address 0 0
Country 0 0
Phone 0 0
866-337-1868
Fax 0 0
Email 0 0
ctgov@clinicalresearchmgt.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02638207