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

This study has been terminated.
(Study Terminated.priority changes in product development.)
Information provided by (Responsible Party):
Octapharma Identifier:
First received: October 6, 2010
Last updated: October 17, 2016
Last verified: June 2015
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.

Condition Intervention Phase
Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Drug: NewGam 10%
Drug: Placebo
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: Double-blind, Placebo-controlled, Randomised, Multicentre, Adaptive, Two-stage Phase 2/3 Study Evaluating Safety and Efficacy of Three Dosages of NewGam in CIDP Patients

Resource links provided by NLM:

Further study details as provided by Octapharma:

Primary Outcome Measures:
  • Adjusted INCAT disability score [ Time Frame: Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2) ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Vital Signs [ Time Frame: During each infusion - Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2) ] [ Designated as safety issue: Yes ]
  • Grip Strength [ Time Frame: Visit 9 & 13 ] [ Designated as safety issue: No ]
  • Nerve Conduction Studies [ Time Frame: Visti 9 & 13 ] [ Designated as safety issue: No ]
  • Motor Impairment Assessment utlizing the Expanded MRC Sum Score [ Time Frame: Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2) ] [ Designated as safety issue: No ]
    Expanded 'Medical Research Council sum score' will be measured as improvement in MRC units .

Enrollment: 2
Study Start Date: October 2011
Study Completion Date: October 2012
Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Dosage Arm 1
NewGam 10% 0.4 g/kg
Drug: NewGam 10%
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.
Experimental: Dosage Arm 2
NewGam 10% 1.0 g/kg
Drug: NewGam 10%
Loading dose at baseline (Week 0) will be 2.0 g/kg NewGam in all three NewGam treatment arms. The maintenance doses to be infused 7 times will be 1.0 g/kg every 21 (+/-4) days.
Experimental: Dosage Arm 3
NewGam 10% 2.0 g/kg
Drug: NewGam 10%
Loading dose at baseline (Week 0) will be 2.0 g/kg NewGam in all three NewGam treatment arms. The maintenance doses to be infused 7 times will be 0.4 g/kg every 21 (+/-4) days.
Placebo Comparator: Dosage Arm 4
Placebo 0.9% Saline
Drug: Placebo
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.
Other Name: 0.9% saline solution

Detailed Description:

This is a Phase 2/3 study that will take place in 2 stages. The primary objective of Stage 1 (Phase 2 dose-finding part)is to determine and select one dosage from three NewGam maintenance dosage arms in comparison with a placebo arm, based on the percentage of responders (response defined as a decrease, meaning improvement, in the adjusted INCAT disability score by at least 1 point). The selected NewGam dosage and placebo will be employed and compared in Stage 2.

The primary objective of Stage 2 (Phase 3 confirmatory part) is to demonstrate superiority of the maintenance dosage regimen selected at study Stage 1 over placebo in patients with CIDP as assessed by the percentage of responders.

The secondary objective is to evaluate the safety (measured by number of adverse events)and efficacy of NewGam administration in patients with CIDP compared to baseline.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01225276

Sponsors and Collaborators
Study Director: Wolfgang Frenzel, MD Octapharma
  More Information

Responsible Party: Octapharma Identifier: NCT01225276     History of Changes
Other Study ID Numbers: NGAM-03 
Study First Received: October 6, 2010
Last Updated: October 17, 2016
Health Authority: United States: Food and Drug Administration
Individual Participant Data  
Plan to Share IPD: No
Plan Description: no (IPD) data exist for this study

Keywords provided by Octapharma:

Additional relevant MeSH terms:
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases processed this record on October 27, 2016