Safety and Efficacy Study of Three Different Dosages of NewGam in Patients With CIDP

NCT01225276 | Terminated Phase 2 DMC

• 1 other identifier: NGAM-03
• Study Type: interventional
• Target: 2
• Locations: 0 countries
• Sites: N/A

Brief Summary

NewGam (current working title for a new IGIV formulation) is a newly developed human normal immunoglobulin solution ready for intravenous administration (IGIV). This study will evaluate the safety and efficacy of three different dosages of NewGam 10% in patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy.

Conditions

Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Keywords

CIDP; IVIG

Outcome Measures

Primary Outcomes (1)

• Adjusted INCAT disability score

  Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)

Secondary Outcomes (4)

• Vital Signs

  During each infusion - Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)

• Grip Strength

  Visit 9 & 13

• Nerve Conduction Studies

  Visti 9 & 13

• Motor Impairment Assessment utlizing the Expanded MRC Sum Score

  Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)

Study Arms (4)

Dosage Arm 1

EXPERIMENTAL

NewGam 10% 0.4 g/kg

Drug: NewGam 10%

Dosage Arm 2

EXPERIMENTAL

NewGam 10% 1.0 g/kg

Drug: NewGam 10%

Dosage Arm 3

EXPERIMENTAL

NewGam 10% 2.0 g/kg

Drug: NewGam 10%

Dosage Arm 4

PLACEBO COMPARATOR

Placebo 0.9% Saline

Drug: Placebo

Interventions

NewGam 10% DRUG

Loading dose at baseline (Week 0) will be 2.0 g/kg NewGam . The maintenance doses to be infused 7 times will be 2.0 g/kg every 21 (+/-4) days.

Dosage Arm 1

Placebo DRUG

Loading dose at baseline (Week 0) in Placebo arm will be corresponding volume of an authorised 0.9% saline solution . The maintenance doses of the 0.9% saline solution to be infused 7 times will be given every 21 (+/-4) days.

Also known as: 0.9% saline solution

Dosage Arm 4

Eligibility Criteria

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

You may qualify if:

• Patients diagnosed as having CIDP based on fulfilment of clinical criteria of the INCAT Group and the definite electrophysiological criteria for CIDP ; patients with MADSAM or pure motor CIDP will be included provided they fulfil these criteria
• Worsening of disability and objective increase in weakness or sensory deficit during the 6 months prior to screening
• \>=18 years of age

You may not qualify if:

• Unifocal forms of CIDP
• Pure sensory CIDP
• MMN with conduction block
• 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 plasma exchange (PE) during the last 3 months prior to baseline visit
• Treatment with cyclosporin, methotrexate, mitoxantrone, mycophenolate mofetil, interferon or other immunosuppressive or immunomodulatory drugs during the three months prior to baseline visit
• Clinical evidence of peripheral neuropathy from another
• Known diabetes mellitus
• Other serious medical condition complicating assessment or treatment
• Thromboembolic events: patients with a history of deep vein thrombosis (DVT) within the last year prior to baseline visit or pulmonary embolism ever
• 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

Sponsors & Collaborators

• Octapharma: lead

Related Publications (1)

• 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

MeSH Terms

Conditions

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Interventions

Saline Solution

Condition Hierarchy (Ancestors)

Polyradiculoneuropathy; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Study Officials

• Wolfgang Frenzel, MD

  Octapharma

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 6, 2010
• First Posted: October 20, 2010
• Study Start: October 1, 2011
• Primary Completion: October 1, 2012
• Study Completion: October 1, 2012
• Last Updated: February 23, 2017

Record last verified: 2017-02

Data Sharing

• IPD Sharing: Will not share