Safety and Efficacy Study of Three Different Dosages of NewGam in Patients With CIDP - Full Text View

Purpose

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%	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

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
Estimated Study Completion Date:	October 2014
Estimated Primary Completion Date:	April 2014 (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 in all three NewGam treatment arms (or a corresponding volume of an authorised 0.9% saline solution in the placebo arm). The maintenance doses to be infused 7 times will be 0.4 g/kg, 1.0 g/kg or 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 (or a corresponding volume of an authorised 0.9% saline solution in the placebo arm). The maintenance doses to be infused 7 times will be 0.4 g/kg, 1.0 g/kg or 2.0 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 (or a corresponding volume of an authorised 0.9% saline solution in the placebo arm). The maintenance doses to be infused 7 times will be 0.4 g/kg, 1.0 g/kg or 2.0 g/kg every 21 (+/-4) days.
Placebo Comparator: Placebo 0.9% Saline	Drug: NewGam 10% Loading dose at baseline (Week 0) will be 2.0 g/kg NewGam in all three NewGam treatment arms (or a corresponding volume of an authorised 0.9% saline solution in the placebo arm). The maintenance doses to be infused 7 times will be 0.4 g/kg, 1.0 g/kg or 2.0 g/kg every 21 (+/-4) days.

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.

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Please refer to this study by its ClinicalTrials.gov identifier: NCT01225276

Sponsors and Collaborators

Octapharma

Investigators

Study Director:

Wolfgang Frenzel, MD

Octapharma

More Information

No publications provided

Responsible Party:	Octapharma
ClinicalTrials.gov Identifier:	NCT01225276 History of Changes
Other Study ID Numbers:	NGAM-03
Study First Received:	October 6, 2010
Last Updated:	October 10, 2012
Health Authority:	United States: Food and Drug Administration

Keywords provided by Octapharma:

CIDP
IVIG

Additional relevant MeSH terms:

Polyradiculoneuropathy
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases

Polyneuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases

ClinicalTrials.gov processed this record on May 23, 2013

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