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

NCT02638207 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: NGAM-08
• Study Type: interventional
• Target: 142
• Locations: 9 countries
• Sites: 26

Brief Summary

Study to evaluate the Efficacy and Safety of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly(radiculo)neuropathy

Conditions

Chronic Inflammatory Demyelinating Poly(Radiculo)Neuropathy

Keywords

Chronic Inflammatory Demyelinating Poly Neuropathy (CIDP)

Outcome Measures

Primary Outcomes (1)

• 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)

  at Week 24

Secondary Outcomes (11)

• Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score

  at Week 24

• Grip Strength Score

  at Week 24

• Inflammatory Rasch-built Overall Disability Scale (I-RODS Score)

  at Week 24

• Worsening in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score

  Week 24

• Mean Change in Grip Strength

  Up to 24 weeks

Study Arms (3)

0.5 g/kg NewGam

EXPERIMENTAL

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).

Drug: NewGam

1.0 g/kg NewGam

EXPERIMENTAL

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).

Drug: NewGam

2.0 g/kg NewGam

EXPERIMENTAL

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).

Drug: NewGam

Interventions

NewGam DRUG

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

Also known as: Panzyga

0.5 g/kg NewGam; 1.0 g/kg NewGam; 2.0 g/kg NewGam

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 )
• Patients currently depending on treatment with immunoglobulins or corticosteroids
• 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
• Weakness of at least 2 limbs
• \>18 to \<80 years of age
• Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score between 2 and 9 (with a score of 2 coming exclusively from leg disability)
• Voluntarily given, fully informed written consent obtained from patient before any study-related procedures are conducted

You may not qualify if:

• Unifocal forms of Chronic inflammatory demyelinating polyneuropathy (CIDP)
• Pure sensory Chronic inflammatory demyelinating polyneuropathy (CIDP)
• Multifocal motor neuropathy (MMN) with conduction block \[van den Bergh et al., 2010\]
• Patients who previously failed immunoglobulin treatment
• Treatment with immunomodulatory/suppressive agents (cyclosporin, methotrexate, mitoxantrone, mycophenolate mofetil or azathioprine) during the six months prior to baseline visit
• 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
• Respiratory impairment requiring mechanical ventilation
• Myelopathy or evidence of central nervous system demyelination or significant persisting neurological deficits from stroke, or central nervous system (CNS) trauma
• Clinical evidence of peripheral neuropathy from another cause such as
• connective tissue disease or systemic lupus erythematosus (SLE)
• HIV infection, hepatitis, Lyme disease
• cancer (with the exception of basal cell skin cancer)
• IgM paraproteinemia with anti-myelin associated glycoprotein antibodies
• Diabetic neuropathy
• Cardiac insufficiency (New York Heart Association \[NYHA\] III/IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease

Sponsors & Collaborators

• Octapharma: lead

Study Sites (26)

MHAT Puls EOOD

Blagoevgrad, 2700, Bulgaria

Location

St. Naum Hospital

Sofia, 1797, Bulgaria

Location

Toronto General Hospital

Toronto, Ontario, M5G 2C4, Canada

Location

Octapharma Research Site

Montreal, H3A2B4, Canada

Location

Outpatient Clinic of Neurology

Hradec Králové, 500 03, Czechia

Location

Regional Hospital Pardubice

Pardubice, 532 03, Czechia

Location

Thomayer Faculty Hospital

Prague, 140 00, Czechia

Location

University Medical Center Goettigen

Goettigen, 37075, Germany

Location

Jahn Ferenc Del Pesti Korhaz

Budapest, 1204, Hungary

Location

Szegedi Tudományegyetem ÁOK Neurológiai Klinika

Szeged, 6725, Hungary

Location

Samodzielny Publiczny Szpital Kliniczny Nr 4 w Lublinie Klinika Neurologii

Lublin, 20-954, Poland

Location

Wojewodzki Szpital Specjalistyczny W Olsztynie

Olsztyn, 10-561, Poland

Location

Uniwersytecki Szpital Kliniczny

Wroclaw, 50-556, Poland

Location

Theo Health S.R.L.

Brasov, 500091, Romania

Location

Institutul Clinic Fundeni

Bucharest, 022328, Romania

Location

Spitalul Clinic Judetean de Urgenta "Sf.Apostol Andrei" Constanta

Constanța, 900591, Romania

Location

Republican Clinical Neurological Centre

Kazan', 420021, Russia

Location

Neurology Research Centre

Moscow, 125367, Russia

Location

Nizhny Novgorod Regional Clinical Hospital N.A. N.A.Semashko

Nizhny Novgorod, 603126, Russia

Location

National Medical Research Centre of Psychiatry and Neurology n.a. V.M.Bekhterev

Saint Petersburg, 192019, Russia

Location

City Multifield Hospital #2

Saint Petersburg, 194354, Russia

Location

Ivano Frankivsk National Medical University

Ivano-Frankivsk, 76008, Ukraine

Location

National Medical Academy Of Postgraduate Education Named After P.L. Shupyk

Kyiv, 4112, Ukraine

Location

Volyn Regional Clinical Hospital

Lutsk, 4300, Ukraine

Location

Vinnytsia National Medical University

Vinnytsia, 21005, Ukraine

Location

Municipal Institution Zaporizhzhya Regional Clinical Hospital

Zaporizhzhya, 69600, Ukraine

Location

Related Publications (3)

• Bus SR, de Haan RJ, Vermeulen M, van Schaik IN, Eftimov F. Intravenous immunoglobulin for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev. 2024 Feb 14;2(2):CD001797. doi: 10.1002/14651858.CD001797.pub4.

  PMID: 38353301 DERIVED

• Cornblath DR, van Doorn PA, Hartung HP, Merkies ISJ, Katzberg HD, Hinterberger D, Clodi E; ProCID Investigators. Safety and Tolerability of Intravenous Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy: Results of the ProCID Study. Drug Saf. 2023 Sep;46(9):835-845. doi: 10.1007/s40264-023-01326-z. Epub 2023 Jun 28.

  PMID: 37378806 DERIVED

• Cornblath DR, van Doorn PA, Hartung HP, Merkies ISJ, Katzberg HD, Hinterberger D, Clodi E; ProCID Investigators. Randomized trial of three IVIg doses for treating chronic inflammatory demyelinating polyneuropathy. Brain. 2022 Apr 29;145(3):887-896. doi: 10.1093/brain/awab422.

  PMID: 35038723 DERIVED

MeSH Terms

Interventions

Panzyga

Results Point of Contact

• Title: Mikaela Raymond
• Organization: CRMG

ctgov@clinicalresearchmgt.com

866-337-1868

Study Officials

• Wolfgang Frenzel, MD

  Octapharma

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 16, 2015
• First Posted: December 23, 2015
• Study Start: September 27, 2017
• Primary Completion: September 5, 2019
• Study Completion: September 5, 2019
• Last Updated: February 16, 2021
• Results First Posted: February 16, 2021

Record last verified: 2021-01

Data Sharing

• IPD Sharing: Will not share

Locations

CZ (3); DE (1); RO (3); UA (5); PL (3); RU (5); BU (2); CA (2); HU (2)