Panzyga in CIDP Administered at Different Infusion Rates (Panzyga-CIDP)
Primary Purpose
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Status
Unknown status
Phase
Phase 3
Locations
Canada
Study Type
Interventional
Intervention
Immune Globulin 10% Intravenous Solution
Sponsored by
About this trial
This is an interventional treatment trial for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Eligibility Criteria
Inclusion Criteria:
- Patients with diagnosis of definite or probable CIDP according to the EFNS/PNS Guideline 2010; including patients with Multifocal Acquired Demyelinating Sensory And Motor Neuropathy (MADSAM) or pure motor CIDP
- Patients with active disease, i.e. not being in remission.
- IVIG naïve patients with clinical indication for IVIG based on progressive or relapsing disease and adjusted INCAT (ONLS) disability score between 2 and 9 (with a score of 2 coming exclusively from leg disability).
- Patients already receiving IVIG must be on 3- or 4-weekly IVIG treatment schedule with a calculated monthly dosage between 0.8 g/kg and 2.0 g/kg BW
- ≥ 18 years of age
- Voluntarily given, fully informed written consent obtained from patient before any study-related procedures are conducted
- For enrolment into the Second Phase: At each of the last three infusions in the First Phase, administration of panzyga® had to be at the maximum infusion rate of 0.08 mL/kg/min and good tolerated- assessment by Investigator according to local site practice
Exclusion Criteria:
- MMN with conduction block
- Patients who previously failed immunoglobulin therapy
- Treatment with immunomodulatory/suppressive agents (cyclosporin, methotrexate, mitoxantrone, mycophenolate mofetil or azathioprine) during the six months prior to baseline visit
- Patients on or treated with rituximab, alemtuzumab, cyclophosphamide, or other intensive chemotherapeutic regimens, previous lymphoid irradiation or stem cell transplantation during the 12 months prior to baseline visit
- Respiratory impairment requiring mechanical ventilation
- Myelopathy or evidence of central nervous system demyelination or significant persisting neurological deficits from stroke, or central nervous system (CNS) trauma
Clinical evidence of peripheral neuropathy from another cause such as
- connective tissue disease or systemic lupus erythematosus (SLE)
- HIV infection, hepatitis, Lyme disease
- cancer (with the exception of basal cell skin cancer)
- IgM paraproteinemia with anti-myelin associated glycoprotein antibodies
- Diabetic neuropathy
- Cardiac insufficiency (New York Heart Association [NYHA] III/IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease
- Severe liver disease (ALAT 3x > normal value)
- Severe kidney disease (creatinine 1.5x > normal value)
- Hepatitis B, hepatitis C or HIV infection
- Thromboembolic events: patients with a history of deep vein thrombosis (DVT) within the last year prior to baseline visit or pulmonary embolism ever; patients with susceptibility to embolism or DVT
- Body mass index (BMI) ≥40 kg/m2
- Selective IgA deficiency with known anti-IgA antibodies
- History of hypersensitivity, anaphylaxis or severe systemic response to immuno-globulin, blood or plasma derived products, or any component of panzyga®
- Known blood hyperviscosity, or other hypercoagulable states
- Use of other blood or plasma-derived products within three months prior to enrolment
- Patients with a past or present history of drug abuse or alcohol abuse within the preceding five years prior to baseline visit
- Patients unable or unwilling to understand or comply with the study protocol
- Participation in another interventional clinical study with IMP treatment currently or during the three months prior to enrolment
- Women who are breast feeding, pregnant, or planning to become pregnant, or are unwilling to use an effective birth control method (such as implants, injectables, combined oral contraceptives, some intrauterine devices (IUDs), sexual abstinence or vasectomized partner) while on study.
Sites / Locations
- UHNToronto
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
Open Label study
Arm Description
open label study using Panzyga immune globulin 10% intravenous solution with no placebo.
Outcomes
Primary Outcome Measures
Occurrence of all adverse events with focus on adverse drug reactions (ADRs)
adverse drug reactions
Secondary Outcome Measures
treatment satisfaction
completion of questionnaire
proportion of patients successfully achieving higher infusion rates
descriptive analysis of number of patients
health utilities
completion of questionnaire
proportion of responders to treatment based on change in clinical scores
completion of scale
grip strength
measurements in kPa
quality of life measures
completion of scale
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03166527
Brief Title
Panzyga in CIDP Administered at Different Infusion Rates
Acronym
Panzyga-CIDP
Official Title
Prospective, Open-Label, Phase IIIb Study Evaluating the Safety, Tolerability and Efficacy of Panzyga® in Patients With Chronic Inflammatory Demyelinating Polyneuropathy Administered at Standard and High Infusion Rates
Study Type
Interventional
2. Study Status
Record Verification Date
May 2017
Overall Recruitment Status
Unknown status
Study Start Date
June 1, 2017 (Anticipated)
Primary Completion Date
December 15, 2018 (Anticipated)
Study Completion Date
December 15, 2018 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Vera Bril
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a treatable form of peripheral neuropathy with suspected autoimmune cause. The current first-line treatment is IVIG (immune globulin), which is infused in a set regimen that requires 4-5 hours in a hospital day unit, taking up resources such as nursing time and hospital space. Chronic treatment is required in most cases.
Detailed Description
The proposed trial will be an exploratory, open-label, single-centre, phase IIIb safety, tolerability and efficacy study, wherein each patient acts as their own control. The primary outcome measure is safety and tolerability of panzyga in patients with active CIDP at standard and high infusion rates as measured by:
Occurrence of all adverse events with focus on adverse drug reactions (ADRs)
The secondary outcomes include: Patients' treatment satisfaction, proportion of patients successfully achieving higher infusion rates, health utilities associated with treatment, proportion of responders to treatment based on change in clinical scores, grip strength, and quality of life measures. The total sample size is 25-30 patients, based on a difference of 30% in adverse events rates between the standard infusion rate and the maximum rate tolerated by each patient.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
Each patient with active CIDP will receive Panzyga (immune globulin) at standard and high infusion rates and will act as their own control.
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Open Label study
Arm Type
Other
Arm Description
open label study using Panzyga immune globulin 10% intravenous solution with no placebo.
Intervention Type
Drug
Intervention Name(s)
Immune Globulin 10% Intravenous Solution
Other Intervention Name(s)
Panzyga IVIG
Intervention Description
standard Immune lobulin 10% intravenous solution infusion at standard and high infusion rates.
Primary Outcome Measure Information:
Title
Occurrence of all adverse events with focus on adverse drug reactions (ADRs)
Description
adverse drug reactions
Time Frame
2 years
Secondary Outcome Measure Information:
Title
treatment satisfaction
Description
completion of questionnaire
Time Frame
2 years
Title
proportion of patients successfully achieving higher infusion rates
Description
descriptive analysis of number of patients
Time Frame
2 years
Title
health utilities
Description
completion of questionnaire
Time Frame
2 years
Title
proportion of responders to treatment based on change in clinical scores
Description
completion of scale
Time Frame
2 years
Title
grip strength
Description
measurements in kPa
Time Frame
2 years
Title
quality of life measures
Description
completion of scale
Time Frame
2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients with diagnosis of definite or probable CIDP according to the EFNS/PNS Guideline 2010; including patients with Multifocal Acquired Demyelinating Sensory And Motor Neuropathy (MADSAM) or pure motor CIDP
Patients with active disease, i.e. not being in remission.
IVIG naïve patients with clinical indication for IVIG based on progressive or relapsing disease and adjusted INCAT (ONLS) disability score between 2 and 9 (with a score of 2 coming exclusively from leg disability).
Patients already receiving IVIG must be on 3- or 4-weekly IVIG treatment schedule with a calculated monthly dosage between 0.8 g/kg and 2.0 g/kg BW
≥ 18 years of age
Voluntarily given, fully informed written consent obtained from patient before any study-related procedures are conducted
For enrolment into the Second Phase: At each of the last three infusions in the First Phase, administration of panzyga® had to be at the maximum infusion rate of 0.08 mL/kg/min and good tolerated- assessment by Investigator according to local site practice
Exclusion Criteria:
MMN with conduction block
Patients who previously failed immunoglobulin therapy
Treatment with immunomodulatory/suppressive agents (cyclosporin, methotrexate, mitoxantrone, mycophenolate mofetil or azathioprine) during the six months prior to baseline visit
Patients on or treated with rituximab, alemtuzumab, cyclophosphamide, or other intensive chemotherapeutic regimens, previous lymphoid irradiation or stem cell transplantation during the 12 months prior to baseline visit
Respiratory impairment requiring mechanical ventilation
Myelopathy or evidence of central nervous system demyelination or significant persisting neurological deficits from stroke, or central nervous system (CNS) trauma
Clinical evidence of peripheral neuropathy from another cause such as
connective tissue disease or systemic lupus erythematosus (SLE)
HIV infection, hepatitis, Lyme disease
cancer (with the exception of basal cell skin cancer)
IgM paraproteinemia with anti-myelin associated glycoprotein antibodies
Diabetic neuropathy
Cardiac insufficiency (New York Heart Association [NYHA] III/IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease
Severe liver disease (ALAT 3x > normal value)
Severe kidney disease (creatinine 1.5x > normal value)
Hepatitis B, hepatitis C or HIV infection
Thromboembolic events: patients with a history of deep vein thrombosis (DVT) within the last year prior to baseline visit or pulmonary embolism ever; patients with susceptibility to embolism or DVT
Body mass index (BMI) ≥40 kg/m2
Selective IgA deficiency with known anti-IgA antibodies
History of hypersensitivity, anaphylaxis or severe systemic response to immuno-globulin, blood or plasma derived products, or any component of panzyga®
Known blood hyperviscosity, or other hypercoagulable states
Use of other blood or plasma-derived products within three months prior to enrolment
Patients with a past or present history of drug abuse or alcohol abuse within the preceding five years prior to baseline visit
Patients unable or unwilling to understand or comply with the study protocol
Participation in another interventional clinical study with IMP treatment currently or during the three months prior to enrolment
Women who are breast feeding, pregnant, or planning to become pregnant, or are unwilling to use an effective birth control method (such as implants, injectables, combined oral contraceptives, some intrauterine devices (IUDs), sexual abstinence or vasectomized partner) while on study.
Facility Information:
Facility Name
UHNToronto
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2C4
Country
Canada
12. IPD Sharing Statement
Plan to Share IPD
No
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Panzyga in CIDP Administered at Different Infusion Rates
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