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This is a fundamental, prospective, multi-centres, interventional non-comparative study without the administration of a study product to patients.
A phase 0 study is particularly well adapted to early exploration of potential targeted treatments - i.e. treatments whose efficacy can only be hoped for in patients presenting specific biological characteristics in addition to being diagnosed with the targeted disease, Neuromyelitis Optica Spectrum Disorders (NMOSD) in this study. The main goal of the study will be to support the selection of Imotopes® (i.e. synthetic peptides encompassing HLA Class II T epitopes flanked by a thioreductase motif), and (i) capable of binding with class II HLA antigens of each patient and (ii) causing ex vivo the appearance of epitope -specific cytolytic CD4+ T cells.
This is a fundamental, prospective, multi-centres, interventional non-comparative study without the administration of a study product to patients.
The objective is to test in vitro the binding of different Imotopes® to class II HLA antigens on peripheral blood mononuclear cell (PBMC) isolated from patients presenting a diagnosed and stabilized neuromyelitis optica spectrum disorders, as well as their ability to generate a cytolytic response directed against the immune cells involved in the maintenance and triggering of the disease (i.e. non-cytolytic T cells recognising the same epitopes and antigen presenting cells (APC) presenting the same epitopes).
The patients will be asked to provide at least 2 peripheral blood samples of approximately 100 milliliters each time, with a minimum of 14 days interval between these 2 samples. These blood samples will be taken by a member of the healthcare staff qualified to perform this type of procedure. If the results obtained with the 2 initial samples would be of interest to the Sponsor, the patient will be asked to come back for additional samplings with a maximum of 6 additional blood draws. The total volume of blood that will be taken over a 6 months period cannot exceed 500 milliliters with a maximum of 200 milliliters over a 30-day period for patients with a weight of min 50kg. The study has no other constraint.
The list of analysis planned to be performed on blood samples are presented below:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Test of Imotopes® candidates on blood cells of patients with stabilized NMO | Experimental | To test in vitro the binding of different Imotopes® to class II HLA antigens on PBMC isolated from patients presenting a diagnosed and stabilized neuromyelitis optica spectrum disorders, as well as their ability to generate a cytolytic response directed against the immune cells involved in the maintenance and triggering of the disease (i.e. non-cytolytic T cells recognising the same epitopes and antigen presenting cells (APC) presenting the same epitopes). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Blood sampling | Procedure | The patients will be asked to provide at least 2 peripheral blood samples of approximately 100 milliliters each time, with a minimum of 14 days interval between these 2 samples.If the results obtained with the 2 initial samples would be of interest to the Sponsor, the patient will be asked to come back for additional samplings with a maximum of 6 additional blood draws |
| Measure | Description | Time Frame |
|---|---|---|
| Measure in vitro binding of Imotopes® | Characterize, in vitro, the binding of different Imotopes® to class II HLA antigens on PBMC isolated from patients with NMO | Through study completion, an average of 1 year |
| Measure Cytolytic response against Antigen-Presenting Cells (APC) | Measure the ability of Imotopes® to generate a cytolytic response directed against the immune cells involved in the maintenance and triggering of the disease (i.e. non-cytolytic T cells recognising the same epitopes and APC presenting the same epitopes) | Through study completion, an average of 1 year |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jerome De Seze, MD | Hôpital de Hautepierre, Strasbourg | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hôpital Pierre Wertheimer | Bron | 69500 | France | |||
| Hôpital de Hautepierre |
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| ID | Term |
|---|---|
| D009471 | Neuromyelitis Optica |
| ID | Term |
|---|---|
| D009188 | Myelitis, Transverse |
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| D001800 | Blood Specimen Collection |
| ID | Term |
|---|---|
| D013048 | Specimen Handling |
| D019411 | Clinical Laboratory Techniques |
| D019937 | Diagnostic Techniques and Procedures |
| D003933 | Diagnosis |
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|
| Strasbourg |
| 67200 |
| France |
| D009902 | Optic Neuritis |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D003711 | Demyelinating Diseases |
| D005128 | Eye Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D011677 | Punctures |
| D013514 | Surgical Procedures, Operative |
| D008919 | Investigative Techniques |