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

Sponsor

Octapharma (Industry)

Overall Status

Terminated

CT.gov ID

NCT01225276

Collaborator

(none)

Enrollment

Arms

Duration (Months)

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Chronic Inflammatory Demyelinating Polyradiculoneuropathy	Drug: NewGam 10% Drug: Placebo Drug: NewGam 10% Drug: NewGam 10%	Phase 2/Phase 3

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.

Study Design

Study Type:

Interventional

Actual Enrollment :

2 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Double (Participant, 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

Study Start Date :

Oct 1, 2011

Actual Primary Completion Date :

Oct 1, 2012

Actual Study Completion Date :

Oct 1, 2012

Arms and Interventions

Arm	Intervention/Treatment
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 Names: 0.9% saline solution

Outcome Measures

Primary Outcome Measures

Adjusted INCAT disability score [Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)]

Secondary Outcome Measures

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)]
Expanded 'Medical Research Council sum score' will be measured as improvement in MRC units .

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Locations

No locations specified.

Sponsors and Collaborators

Octapharma

Investigators

Study Director: Wolfgang Frenzel, MD, Octapharma

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Octapharma

ClinicalTrials.gov Identifier:

NCT01225276

Other Study ID Numbers:

NGAM-03

First Posted:

Oct 20, 2010

Last Update Posted:

Feb 23, 2017

Last Verified:

Feb 1, 2017

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Keywords provided by Octapharma

CIDP

IVIG

Additional relevant MeSH terms:

Polyradiculoneuropathy

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Study Results

No Results Posted as of Feb 23, 2017