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

Primary Purpose

Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Status

Terminated

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

NewGam 10%

Placebo

NewGam 10%

About this trial

This is an interventional treatment trial for Chronic Inflammatory Demyelinating Polyradiculoneuropathy focused on measuring CIDP, IVIG

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Placebo Comparator

Arm Label

Dosage Arm 1

Dosage Arm 2

Dosage Arm 3

Dosage Arm 4

Arm Description

NewGam 10% 0.4 g/kg

NewGam 10% 1.0 g/kg

NewGam 10% 2.0 g/kg

Placebo 0.9% Saline

Outcomes

Primary Outcome Measures

Adjusted INCAT disability score

Secondary Outcome Measures

Vital Signs

Grip Strength

Nerve Conduction Studies

Motor Impairment Assessment utlizing the Expanded MRC Sum Score

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

Full Information

NCT ID

NCT01225276

First Posted

October 6, 2010

Last Updated

February 20, 2017

Sponsor

Octapharma

1. Study Identification

Unique Protocol Identification Number

NCT01225276

Brief Title

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

Acronym

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

Study Type

Interventional

2. Study Status

Record Verification Date

February 2017

Overall Recruitment Status

Terminated

Why Stopped

Study Terminated.priority changes in product development.

Study Start Date

October 2011 (undefined)

Primary Completion Date

October 2012 (Actual)

Study Completion Date

October 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Octapharma

4. Oversight

Data Monitoring Committee

Yes

5. 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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Keywords

CIDP, IVIG

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

2 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Dosage Arm 1

Arm Type

Experimental

Arm Description

NewGam 10% 0.4 g/kg

Arm Title

Dosage Arm 2

Arm Type

Experimental

Arm Description

NewGam 10% 1.0 g/kg

Arm Title

Dosage Arm 3

Arm Type

Experimental

Arm Description

NewGam 10% 2.0 g/kg

Arm Title

Dosage Arm 4

Arm Type

Placebo Comparator

Arm Description

Placebo 0.9% Saline

Intervention Type

Drug

Intervention Name(s)

NewGam 10%

Intervention Description

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.

Intervention Type

Drug

Intervention Name(s)

Placebo

Other Intervention Name(s)

0.9% saline solution

Intervention Description

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.

Intervention Type

Drug

Intervention Name(s)

NewGam 10%

Intervention Description

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.

Intervention Type

Drug

Intervention Name(s)

NewGam 10%

Intervention Description

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.

Primary Outcome Measure Information:

Title

Adjusted INCAT disability score

Time Frame

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

Secondary Outcome Measure Information:

Title

Vital Signs

Time Frame

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

Title

Grip Strength

Time Frame

Visit 9 & 13

Title

Nerve Conduction Studies

Time Frame

Visti 9 & 13

Title

Motor Impairment Assessment utlizing the Expanded MRC Sum Score

Description

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

Time Frame

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Wolfgang Frenzel, MD

Organizational Affiliation

Octapharma

Official's Role

Study Director

12. IPD Sharing Statement

Plan to Share IPD

IPD Sharing Plan Description

no (IPD) data exist for this study

Learn more about this trial

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

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