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Primary objective:
To assess the efficacy of I10E in improving the disability of patients with CIDP.
Secondary objective:
To assess the safety of I10E in patients with CIDP.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| I10E Arm | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| I10E | Drug | Patients who meet all eligibility criteria will receive one dose of IMP at 2g/kg over 2-5 days followed by 7 doses of IMP at 1g/kg over 1-2 day(s), every 3 weeks. Duration of treatment period: 21 weeks +/- 7 days. |
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy Endpoint: Responder Rate at End of Study | Responders were defined as patients with a decrease ≥1 point in the adjusted INCAT disability score compared to baseline. Adjusted INCAT disability score can vary from 0 (normal) to 9 (maximal disability). If a patient was treated with a not-allowed treatment during the study period, then all adjusted INCAT disability score measured after the intake of these not-allowed treatments were censored. If the score at EoS visit was missing, then the Last Observation Carried Forward (LOCF) approach was applied to replace this missing value. | 24 weeks after first treament injection |
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Inclusion Criteria:
Male or female patient aged 18 years or more
Definite or probable CIDP according to the European Federation of Neurological Societies (EFNS)/Peripheral Nerve Society (PNS) guidelines 2010 clinical and neurophysiological criteria Pure motor CIDP, provided that a diagnosis of multifocal motor neuropathy has been ruled out CIDP associated with monoclonal gammopathy of undetermined significance (MGUS), provided that anti-MAG antibodies titer is lower than the used technique's negativity threshold (1000 BTU for Bühlmann ELISA technique) Lewis-Sumner syndrome
Score of at least 2 on the adjusted INCAT disability scale
Patient who either :
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Eduardo NOBILE-ORAZIO, MD | IRCCS Instituto Clinico Humanitas, Milano, Italy | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| CHU de Bordeaux - Hôpital Pellegrin | Bordeaux | France | ||||
| Hôpital général du CHU de Dijon |
59 subjects signed an informed consent in the pre-assigment period. Among them, 18 subjects were considered as a screening failure including 3 subjects re-screened and finally enrolled.
Of 44 enrolled subjects, on subject was discontinued (consent withdrawn by subject) from the study before the first administration of study drug.
Between 24 February 2015 and 22 March 2017, 59 subjects from 23 sites signed an informed consent.
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| ID | Title | Description |
|---|---|---|
| FG000 | Single Arm | This study included patients never previously treated with IgG and patients already treated with IgG but in clinical relapse following IgG therapy discontinuation. |
| 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 |
|---|---|---|---|---|---|---|---|
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 7, 2018 | Jun 29, 2020 |
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| Dijon |
| France |
| CHU de Nice - Hôpital l'Archet | Nice | France |
| CHU Paris - Hôpital Pitié salpétrière | Paris | France |
| Hôpital Pontchaillou | Rennes | France |
| CHU de saint Etienne - Hôpital nord | Saint-Etienne | France |
| Hôpital de Hautepierre | Strasbourg | France |
| IRRCS Azienda Ospedaliera Universitaria | Genova | Italy |
| IRCCS - Istituto Clinico Humanitas | Milan | Italy |
| IRRCS Istituto Nazionale Neurologico Besta | Milan | Italy |
| Ospedale San Raffaele IRCCS | Milan | Italy |
| Azienda Ospedaliere Universitaria di Padova | Padova | Italy |
| Università Cattolica del Sacro Cuore | Roma | Italy |
| Azienda Ospedaliere Universitaria san Giovanni | Torino | Italy |
| Hospital de la Santa Creu i Sant Pau | Barcelona | Spain |
| Hospital Quiron Madrid | Madrid | 28223 | Spain |
| Hospital General Universitario Gregorio | Madrid | Spain |
| Hospital clinico Universitario de Santiago | Santiago de Compostela | Spain |
| Hospital Universitario Virgen del Rocio | Seville | Spain |
| Hospital Universitario i Politècnico La Fe | Valencia | Spain |
| Hôpital Razi, La Manouba | Manouba | Tunisia |
| Hôpital Fattouma Bourguiba | Monastir | Tunisia |
| Hôpital habib Bourguiba | Sfax | Tunisia |
| Hôpital Sahloul | Sousse | Tunisia |
| Hôpital militaire de Tunis | Tunis | Tunisia |
| Ankara University medical School Neurology | Ankara | Turkey (Türkiye) |
| Hacettepe University Medical School Neurology | Ankara | Turkey (Türkiye) |
| Uludag University Medical School Neurology | Bursa | Turkey (Türkiye) |
| Istanbul UniversityCerrahpasa Medical School Neurology | Istanbul | Turkey (Türkiye) |
| Marmara Universitesi Egitim Ve Arastirma Hastanesi | Istanbul | Turkey (Türkiye) |
| St Georges | London | SW17OQT | United Kingdom |
| Southampton General Hospital | Southampton | United Kingdom |
| University Hospital of North Straffordshire | Stratford-upon-Avon | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Total Treated Set | All subjects who received at least one infusion of I10E |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| |||||||||||||||||||||||
| Age, Continuous | Median | Full Range | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| 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 | Efficacy Endpoint: Responder Rate at End of Study | Responders were defined as patients with a decrease ≥1 point in the adjusted INCAT disability score compared to baseline. Adjusted INCAT disability score can vary from 0 (normal) to 9 (maximal disability). If a patient was treated with a not-allowed treatment during the study period, then all adjusted INCAT disability score measured after the intake of these not-allowed treatments were censored. If the score at EoS visit was missing, then the Last Observation Carried Forward (LOCF) approach was applied to replace this missing value. | Posted | Count of Participants | Participants | 24 weeks after first treament injection |
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The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Total Treated Set | All subjects who received at least one infusion of I10E | 0 | 43 | 7 | 43 | 39 | 43 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Femur fracture | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Synovial rupture | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Fibrin D dimer increased | Investigations | MedDRA (17.1) | Non-systematic Assessment |
| |
| Colorectal adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (17.1) | Non-systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA (17.1) | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Chills | General disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Malaise | General disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (17.1) | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (17.1) | Non-systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical trial information desk | LFB Biotechnologies | 0033169827010 | supportqcm@lfb.fr |
| SAP_002.pdf |
| Prot | Yes | No | No | Study Protocol | May 3, 2017 | Jan 5, 2021 | Prot_003.pdf |
| ID | Term |
|---|---|
| D020277 | Polyradiculoneuropathy, Chronic Inflammatory Demyelinating |
| D010523 | Peripheral Nervous System Diseases |
| D035583 | Rare Diseases |
| ID | Term |
|---|---|
| D011129 | Polyradiculoneuropathy |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D003711 | Demyelinating Diseases |
| D011115 | Polyneuropathies |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| United Kingdom |
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| France |
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| Tunisia |
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| Spain |
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| Poland |
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