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Study to evaluate the Efficacy and Safety of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly(radiculo)neuropathy
Prospective, Double-blind, Randomized, Multicenter Phase III Study Evaluating Efficacy and Safety of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly(radiculo)neuropathy ("ProCID trial")
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 0.5 g/kg NewGam | Experimental | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
|
| 1.0 g/kg NewGam | Experimental | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
|
| 2.0 g/kg NewGam | Experimental | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NewGam | Drug | In the Dose-evaluation Phase, all patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5, 1.0 or 2.0 g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). If a patient is randomized to receive the low or medium NewGam dose, the same volume with the same infusion rate as would have been applied in case the patient would have been randomized to 2.0 g/kg NewGam will be used, thus supplemented with an authorized 0.9% w/v isotonic sodium chloride solution as appropriate and detailed in the following infusion bag split to maintain the blinding |
| Measure | Description | Time Frame |
|---|---|---|
| Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score | Efficacy - Proportion of responders in the 1.0 g/kg NewGam arm at Week 24 (Termination Visit) relative to baseline (Week 0). A responder being defined as a patient with a decrease of at least 1 point on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score (a scale from 0 to 10, from healthy to unable to make any purposeful movements with arms and/or legs) | at Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score | Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline compared to the 1.0 g/kg arm, based on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score | at Week 24 |
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Inclusion Criteria:
Exclusion Criteria:
Unifocal forms of Chronic inflammatory demyelinating polyneuropathy (CIDP)
Pure sensory Chronic inflammatory demyelinating polyneuropathy (CIDP)
Multifocal motor neuropathy (MMN) with conduction block [van den Bergh et al., 2010]
Patients who previously failed immunoglobulin treatment
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
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 deep vein thrombosis (DVT)
Body mass index (BMI) ≥40 kg/m2
Patients with uncompensated hypothyroidism (abnormally high Thyroid-Stimulating Hormone [TSH] and abnormally low Thyroxine [T4]) or known vitamin B12 deficiency if patients don't receive adequate substitution therapy
Medical conditions whose symptoms and effects could alter protein catabolism and/or Immunoglobulin G (IgG) utilization (e.g. protein-losing enteropathies, nephrotic syndrome)
Known Immunoglobulin A (IgA) deficiency with antibodies to Immunoglobulin A (IgA)
History of severe hypersensitivity, e.g. anaphylaxis or severe systemic response to immuno-globulin, blood or plasma derived products, or any component of NewGam
Known blood hyperviscosity, or other hypercoagulable states
Use of other blood or plasma-derived products within three months prior to Visit 2
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 investigational medicinal product (IMP) treatment currently or during the three months prior to Visit 2
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
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| Name | Affiliation | Role |
|---|---|---|
| Wolfgang Frenzel, MD | Octapharma | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| MHAT Puls EOOD | Blagoevgrad | 2700 | Bulgaria | |||
| St. Naum Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38353301 | Derived | Bus SR, de Haan RJ, Vermeulen M, van Schaik IN, Eftimov F. Intravenous immunoglobulin for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev. 2024 Feb 14;2(2):CD001797. doi: 10.1002/14651858.CD001797.pub4. | |
| 37378806 | Derived | Cornblath DR, van Doorn PA, Hartung HP, Merkies ISJ, Katzberg HD, Hinterberger D, Clodi E; ProCID Investigators. Safety and Tolerability of Intravenous Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy: Results of the ProCID Study. Drug Saf. 2023 Sep;46(9):835-845. doi: 10.1007/s40264-023-01326-z. Epub 2023 Jun 28. |
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| ID | Title | Description |
|---|---|---|
| FG000 | 0.5 g/kg NewGam | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 8, 2018 | Sep 3, 2020 |
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|
|
| Grip Strength Score |
Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the grip strength (Martin Vigorimeter) using the previously published minimum clinically important difference (MCID) cut-off of 8 kilopascal (kPa) |
| at Week 24 |
| Inflammatory Rasch-built Overall Disability Scale (I-RODS Score) | Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the Inflammatory Rasch-built overall disability scale (I-RODS Score) using the MCID concept related to the varying standard errors (MCID-SE) as recently demonstrated | at Week 24 |
| Worsening in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score | Time to first confirmed worsening on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale by at least 1 point from the value at baseline (Week 0) | Week 24 |
| Mean Change in Grip Strength | Mean change from baseline (Week 0) to Termination Visit in grip strength of both hands (assessed by Martin vigorimeter). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. | Up to 24 weeks |
| Inflammatory Rasch-built Overall Disability Scale (I-RODS) | Mean change from baseline (Week 0) to Termination Visit in Inflammatory Rasch-built overall disability sum score (I-RODS using the concept of MCID-SE as recently reported) and number of improvers. The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. A scale from 0 to 48, from "Not possible to perform" to "Possible without any difficulty". Higher values represent a better outcome. | Up to 24 weeks |
| Motor Nerves | Mean change from baseline (Week 0) to Termination Visit in sum of the distal evoked amplitude of 4 right sided and 4 left sided motor nerves (peroneal, tibial, ulnar and median). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. | Up to 24 weeks |
| Mean Change in Pain Intensity Numerical Rating Scale (PI-NRS Scale) | Mean change from baseline (Week 0) to Termination Visit in Pain Intensity Numeric Rating Scale (PI-NRS). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. The Pain Intensity Numeric Rating Scale (PI-NRS, a numeric scale where 0 = no pain and 10 = worst possible pain) is an 11-point scale for patient self-reporting of pain. A higher value represents a worse outcome. | Up to 24 weeks |
| Worsening on the Inflammatory Rasch-built Overall Disability Scale (I-RODS Scale) | Time to first confirmed worsening on the I-RODS scale. The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. A scale from 0 to 48, from "Not possible to perform" to "Possible without any difficulty". Higher values represent a better outcome. Worsening is determined using the concept of MCID (minimum clinically important difference) using the individually obtained standard errors (MCID-SE). | 24 weeks |
| 1 Point Decrease in the INCAT Disability Score | Time to 1 point decrease (improvement of disability) in adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score | 24 weeks |
| Decrease in Inflammatory Rasch-built Overall Disability Scale (I-RODS Scale) | Time to decrease in Inflammatory Rasch-built overall disability scale (I-RODS) scores | 24 weeks |
| Sofia |
| 1797 |
| Bulgaria |
| Toronto General Hospital | Toronto | Ontario | M5G 2C4 | Canada |
| Octapharma Research Site | Montreal | H3A2B4 | Canada |
| Outpatient Clinic of Neurology | Hradec Králové | 500 03 | Czechia |
| Regional Hospital Pardubice | Pardubice | 532 03 | Czechia |
| Thomayer Faculty Hospital | Prague | 140 00 | Czechia |
| University Medical Center Goettigen | Goettigen | 37075 | Germany |
| Jahn Ferenc Del Pesti Korhaz | Budapest | 1204 | Hungary |
| Szegedi Tudományegyetem ÁOK Neurológiai Klinika | Szeged | 6725 | Hungary |
| Samodzielny Publiczny Szpital Kliniczny Nr 4 w Lublinie Klinika Neurologii | Lublin | 20-954 | Poland |
| Wojewodzki Szpital Specjalistyczny W Olsztynie | Olsztyn | 10-561 | Poland |
| Uniwersytecki Szpital Kliniczny | Wroclaw | 50-556 | Poland |
| Theo Health S.R.L. | Brasov | 500091 | Romania |
| Institutul Clinic Fundeni | Bucharest | 022328 | Romania |
| Spitalul Clinic Judetean de Urgenta "Sf.Apostol Andrei" Constanta | Constanța | 900591 | Romania |
| Republican Clinical Neurological Centre | Kazan' | 420021 | Russia |
| Neurology Research Centre | Moscow | 125367 | Russia |
| Nizhny Novgorod Regional Clinical Hospital N.A. N.A.Semashko | Nizhny Novgorod | 603126 | Russia |
| National Medical Research Centre of Psychiatry and Neurology n.a. V.M.Bekhterev | Saint Petersburg | 192019 | Russia |
| City Multifield Hospital #2 | Saint Petersburg | 194354 | Russia |
| Ivano Frankivsk National Medical University | Ivano-Frankivsk | 76008 | Ukraine |
| National Medical Academy Of Postgraduate Education Named After P.L. Shupyk | Kyiv | 4112 | Ukraine |
| Volyn Regional Clinical Hospital | Lutsk | 4300 | Ukraine |
| Vinnytsia National Medical University | Vinnytsia | 21005 | Ukraine |
| Municipal Institution Zaporizhzhya Regional Clinical Hospital | Zaporizhzhya | 69600 | Ukraine |
| 35038723 | Derived | Cornblath DR, van Doorn PA, Hartung HP, Merkies ISJ, Katzberg HD, Hinterberger D, Clodi E; ProCID Investigators. Randomized trial of three IVIg doses for treating chronic inflammatory demyelinating polyneuropathy. Brain. 2022 Apr 29;145(3):887-896. doi: 10.1093/brain/awab422. |
| 1.0 g/kg NewGam |
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
| FG002 | 2.0 g/kg NewGam | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | 0.5 g/kg NewGam | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
| BG001 | 1.0 g/kg NewGam | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
| BG002 | 2.0 g/kg NewGam | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Weight | Mean | Standard Deviation | kg |
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| Height | Mean | Standard Deviation | cm |
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| BMI | Mean | Standard Deviation | kg/m^2 |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score | Efficacy - Proportion of responders in the 1.0 g/kg NewGam arm at Week 24 (Termination Visit) relative to baseline (Week 0). A responder being defined as a patient with a decrease of at least 1 point on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score (a scale from 0 to 10, from healthy to unable to make any purposeful movements with arms and/or legs) | Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment. | Posted | Number | proportion of subjects | at Week 24 |
|
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| Secondary | Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score | Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline compared to the 1.0 g/kg arm, based on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score | Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment. | Posted | Number | Proportion of responders | at Week 24 |
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| Secondary | Grip Strength Score | Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the grip strength (Martin Vigorimeter) using the previously published minimum clinically important difference (MCID) cut-off of 8 kilopascal (kPa) | Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment. | Posted | Number | Proportion of responders | at Week 24 |
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| Secondary | Inflammatory Rasch-built Overall Disability Scale (I-RODS Score) | Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the Inflammatory Rasch-built overall disability scale (I-RODS Score) using the MCID concept related to the varying standard errors (MCID-SE) as recently demonstrated | Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment. | Posted | Number | Proportion of responders | at Week 24 |
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| Secondary | Worsening in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score | Time to first confirmed worsening on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale by at least 1 point from the value at baseline (Week 0) | There was only 1 patient with worsening (in the 1.0 g/kg group), thus an analysis of the time to first worsening was not possible. | Posted | Median | 95% Confidence Interval | days | Week 24 |
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| Secondary | Mean Change in Grip Strength | Mean change from baseline (Week 0) to Termination Visit in grip strength of both hands (assessed by Martin vigorimeter). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. | Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment. | Posted | Mean | Standard Deviation | kPa | Up to 24 weeks |
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| Secondary | Inflammatory Rasch-built Overall Disability Scale (I-RODS) | Mean change from baseline (Week 0) to Termination Visit in Inflammatory Rasch-built overall disability sum score (I-RODS using the concept of MCID-SE as recently reported) and number of improvers. The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. A scale from 0 to 48, from "Not possible to perform" to "Possible without any difficulty". Higher values represent a better outcome. | Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment. | Posted | Mean | Standard Deviation | score on a scale | Up to 24 weeks |
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| Secondary | Motor Nerves | Mean change from baseline (Week 0) to Termination Visit in sum of the distal evoked amplitude of 4 right sided and 4 left sided motor nerves (peroneal, tibial, ulnar and median). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. | Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment. | Posted | Mean | Standard Deviation | mV | Up to 24 weeks |
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| Secondary | Mean Change in Pain Intensity Numerical Rating Scale (PI-NRS Scale) | Mean change from baseline (Week 0) to Termination Visit in Pain Intensity Numeric Rating Scale (PI-NRS). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. The Pain Intensity Numeric Rating Scale (PI-NRS, a numeric scale where 0 = no pain and 10 = worst possible pain) is an 11-point scale for patient self-reporting of pain. A higher value represents a worse outcome. | Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment. | Posted | Mean | Standard Deviation | score on a scale | Up to 24 weeks |
| |||||||||||||||||||||||||||
| Secondary | Worsening on the Inflammatory Rasch-built Overall Disability Scale (I-RODS Scale) | Time to first confirmed worsening on the I-RODS scale. The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. A scale from 0 to 48, from "Not possible to perform" to "Possible without any difficulty". Higher values represent a better outcome. Worsening is determined using the concept of MCID (minimum clinically important difference) using the individually obtained standard errors (MCID-SE). | There was 1 patient with worsening in the 0.5 g/kg group and 2 in the 1.0 g/kg group; an analysis of the time to first worsening was not possible | Posted | Median | 95% Confidence Interval | days | 24 weeks |
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| Secondary | 1 Point Decrease in the INCAT Disability Score | Time to 1 point decrease (improvement of disability) in adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score | Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment. | Posted | Median | 95% Confidence Interval | days | 24 weeks |
| |||||||||||||||||||||||||||
| Secondary | Decrease in Inflammatory Rasch-built Overall Disability Scale (I-RODS Scale) | Time to decrease in Inflammatory Rasch-built overall disability scale (I-RODS) scores | Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment. | Posted | Median | 95% Confidence Interval | days | 24 weeks |
|
Up to 36 weeks
An AE is any untoward medical occurrence in a patient receiving an IMP and which does not have to have a causal relationship with treatment.
At each visit AEs will be elicited using a standard non-leading question like "How have you been since the last visit / during the previous study period?" Patient diaries will also be checked.
Safety Data Set (SDS): The SDS was based on all randomized patients who received at least part of one infusion of IMP. Therefore n=142 for the SDS.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | NGAM 0.5 g/kg | NGAM 0.5 g/kg | 0 | 35 | 1 | 35 | 20 | 35 |
| EG001 | NGAM 1.0 g/kg | NGAM 1.0 g/kg | 1 | 69 | 4 | 69 | 45 | 69 |
| EG002 | NGAM 2.0 g/kg | NGAM 2.0 g/kg | 1 | 38 | 1 | 38 | 24 | 38 |
| EG003 | Total | Total of all three treatment arms. | 2 | 142 | 6 | 142 | 88 | 142 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Osteomyelitis | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Cardio-respiratory Arrest | Cardiac disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Deafness unilateral | Ear and labyrinth disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Meningioma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (Unspecified) | Systematic Assessment |
| |
| Encephalitis | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Chills | General disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Infusion site coldness | General disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Infusion site discomfort | General disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Body temperature increased | Investigations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Heart rate decreased | Investigations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Blood uric acid increased | Investigations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Fibrin D dimer increased | Investigations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Tension headache | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Carpal tunnel syndrome | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Neuralgia | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Restless legs syndrome | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Seborrhoeic dermatitis | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Decubitis ulcer | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Dermatitis atopic | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Eczema infected | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Infective exacerbation of bronchiectasis | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Viral pharyngitis | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Abdominal pain lower | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Hiatus hernia | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Eosinophilia | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Lymphadenitis | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Thrombocytosis | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Left atrial dilatation | Cardiac disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Left ventricular hypertrophy | Cardiac disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (Unspecified) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (Unspecified) | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA (Unspecified) | Systematic Assessment |
| |
| Ligament injury | Injury, poisoning and procedural complications | MedDRA (Unspecified) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Diabetes mellitus inadequate control | Metabolism and nutrition disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Dyslipidaemia | Metabolism and nutrition disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Electrolyte imbalance | Metabolism and nutrition disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Type 2 diabetes mellitus | Metabolism and nutrition disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Autoimmune hepatitis | Hepatobiliary disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Gallbladder disorders | Hepatobiliary disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Conjunctival oedema | Eye disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Micturition urgency | Renal and urinary disorders | MedDRA (Unspecified) | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Mikaela Raymond | CRMG | 866-337-1868 | ctgov@clinicalresearchmgt.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 6, 2019 | Sep 3, 2020 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| C000617884 | Panzyga |
Not provided
Not provided
Not provided
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| OG002 | 2.0 g/kg NewGam | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
|
|
| OG002 | 2.0 g/kg NewGam | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
|
|
| OG002 | 2.0 g/kg NewGam | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
|
|
|
|
| OG002 | 2.0 g/kg NewGam | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
|
|
| OG002 | 2.0 g/kg NewGam | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
|
|
| OG002 | 2.0 g/kg NewGam | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
|
|
| OG002 | 2.0 g/kg NewGam | All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
|
|
| OG002 |
| 2.0 g/kg NewGam |
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
|
|
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
|
|
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). |
|
|