Study to Evaluate Safety and Efficacy of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly (Radiculo) Neuropathy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02638207
• Date of registration: 16/12/2015
• Prospective Registration: Yes
• Primary sponsor: Octapharma
• Public title: Study to Evaluate Safety and Efficacy of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly (Radiculo) Neuropathy CIDP
• 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
• Date of first enrolment: September 27, 2017
• Target sample size: 142
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02638207
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Australia, Bulgaria, Canada, Czechia, Denmark, Germany, Hungary, Poland, Romania, Russian Federation, Sweden, Ukraine

Contacts

• Name: Wolfgang Frenzel, MD
• Affiliation: Octapharma

Key inclusion & exclusion criteria

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

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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Chronic Inflammatory Demyelinating Poly(Radiculo)Neuropathy

Intervention(s)

Drug: NewGam

Primary Outcome(s)

Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) disability score [Time Frame: at Week 24]

Secondary Outcome(s)

Inflammatory Rasch-built overall disability scale (I-RODS) [Time Frame: through study completion, an average of 2 years]

Worsening in the Inflammatory Neuropathy Cause and Treatment (INCAT) disability score [Time Frame: through study completion, an average of 2 years]

Mean Change in pain intensity numerical rating scale (PI-NRS Scale) [Time Frame: through study completion, an average of 2 years]

Worsening on the Inflammatory Rasch-built overall disability scale (I-RODS Scale) [Time Frame: through study completion, an average of 2 years]

Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) disability score [Time Frame: at Week 24]

Right sided motor nerves [Time Frame: through study completion, an average of 2 years]

Grip Strength Score [Time Frame: at Week 24]

decrease in Inflammatory Rasch-built overall disability scale (I-RODS Scale) [Time Frame: through study completion, an average of 2 years]

Mean Change in grip strength [Time Frame: through study completion, an average of 2 years]

1 point decrease in the INCAT disability score [Time Frame: through study completion, an average of 2 years]

Inflammatory Rasch-built overall disability scale (I-RODS Score) [Time Frame: at Week 24]

Secondary ID(s)

NGAM-08

Source(s) of Monetary Support

Please refer to primary and secondary sponsors