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

Chronic Inflammatory Demyelinating Polyradiculoneuropathy Clinical Trial

— POINT  

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01225276
• Other study ID #: NGAM-03
• Status: Terminated
• Phase: Phase 2/Phase 3
• First received: October 6, 2010
• Last updated: February 20, 2017
• Start date: October 2011
• Est. completion date: October 2012

Study information

• Verified date: February 2017
• Source: Octapharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 2
• Est. completion date: October 2012
• Est. primary completion date: October 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Chronic Inflammatory Demyelinating Polyradiculoneuropathy
• Polyradiculoneuropathy
• Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Intervention

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.
• 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.
• 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.
• 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.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Octapharma

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adjusted INCAT disability score		Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)
Secondary	Vital Signs		During each infusion - Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)
Secondary	Grip Strength		Visit 9 & 13
Secondary	Nerve Conduction Studies		Visti 9 & 13
Secondary	Motor Impairment Assessment utlizing the Expanded MRC Sum Score	Expanded 'Medical Research Council sum score' will be measured as improvement in MRC units .	Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)