Nervous System DiseasesEXPLAINEURNCT04698421
Collection of Biological Samples From Patients With Rare Neurological Diseases
An observational study of Nervous System Diseases, run by University Hospital, Toulouse at 1 site in France.
Trial summary
The aim of this project is to improve biological collections of patients presenting rare neurological disorders with known or suspected autoimmune origin. This collection will provide appropriate biological samples to identify new biomarkers and to be accessible to the medical, scientific and industrial communities… Read more about this study
- Who is it for?
- Ages 6 to 99 years
- Conditions the study is for
- Nervous System Diseases
- What is being tested?
- Blood collection on admission and longitudinally
- How many people
- 1,000 participants
- Where
- 1 siteFrance
- Study dates
- 12 Oct 2020to 3 Sep 2030
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: patients followed at the Toulouse University Hospital and presenting rare neurological disorders with known or suspected autoimmune origin
Who may be able to join
- all patients with neurological disorders, with known or probable autoimmune involvement. This includes adults and children and peripheral and/or central nervous system symptoms.
- Social coverage up to date.
Who cannot join
- Patients with neurological damage from which the autoimmune character can be excluded.
- Known anemia and hemoglobin <10 g / dl
- Patients under protective supervision (guardianship, curators)
- Pregnant or breastfeeding woman
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Purpan University Hospital
Toulouse, 31059
Chloé Bost, PharmD, PhD, 5 61 77 61 44 ext. 0033, bost.c@chu-toulouse.fr
About this study
Official title: Prospective Collection of Biological Samples From Patients With Rare Neurological Diseases
The aim of this project is to improve biological collections of patients presenting rare neurological disorders with known or suspected autoimmune origin. This collection will provide appropriate biological samples to identify new biomarkers and to be accessible to the medical, scientific and industrial communities for the identification of new therapeutic strategies.
Read the detailed description
Neuroimmunology is a rapidly expanding field since major advances have been made in basic immunology and numerous new clinical entities have been identified in the last 10 years. Even if these discoveries have led to major advances in patient's management and treatment, a lot of work needs to be done to improve the diagnosis and prognostic biomarkers. It is widely known that the immune system is implicated in a variety of neurological disorders such as infections, encephalitis or multiple sclerosis. Numerous neurological disorders affecting the central and peripheral nervous system can be attributed to the immune system and need to be recognized as some of them can be cured by appropriate immunotherapy. These neurological disorders include autoimmune encephalitis and paraneoplastic neurological syndromes but also myasthenia, chronic demyelinating inflammatory polyneuropathy and other neuromuscular pathologies.
These neurological disorders are characterized by the presence of autoantibodies in the patient's sera or cerebral spinal fluid (CSF). These autoantibodies are generally highly specific and necessary to make the diagnosis. However, in some cases, despite strong clinical arguments for a neuroimmunological disorder, we do not identify autoantibodies, leading to inappropriate treatment and a blind follow-up considering the risk of recurrence or of associated tumor. Furthermore, even if the specific role of some autoantibodies or of immune T cells in some of these pathologies are suspected or already documented, for most of them the exact mechanism is still unknown. We need to explore the sera and CSF of these patients to identify new diagnosis and prognosis biomarker. Moreover, the availability of immune cells isolated from these patients will help us to decipher the pathophysiological mechanisms to create new therapeutic strategies. For this, animal models are already available in Centre Physiopathology Toulouse and in the French reference center in Lyon. As genetic susceptibilities may underlie, at least in part, the variability of the clinical manifestations and of the response to treatment, DNA from patients will be collected and immune genes sequencing will be compared to other control groups, included international database.
- Conditions
- Nervous System Diseases
- Being tested
- Blood collection on admission and longitudinally
- Keywords
- autoimmune encephalitis, paraneoplastic neurological syndrome, myasthenia, paraneoplastic cerebellar degeneration, rare neuropathologies with known/suspected autoimmune origin
- Medical subject headings
- Nervous System Diseases, Autoimmune Diseases of the Nervous System, Paraneoplastic Cerebellar Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood collection on admission and longitudinallyBiological
Biological samples will be collected in the normal diagnosis and follow-up process. Only blood will be taken in larger quantity (8 tubes of 7mL).
| Study group | Role | What they receive |
|---|---|---|
| patients with rare autoimmune neurological diseases | Not described Receives: Biological: Blood collection on admission and longitudinally |
What is being measured
The primary outcome is the main question the study is designed to answer.
Building a collection of biological samples and clinical-biological data from patients with rare autoimmune neurological diseases
Blood sampling
Measured: Day 0 and through study completion, an average of 1 year
Secondary outcomes (3)
Identification of new autoantibodies.
ELISA
Measured: Day 0 and through study completion, an average of 1 year
Identification of biomarkers regarding the severity (such as cytokines, axonal damages...) in order to help the therapeutic decisions.
Analysis of the phenotypic profiling of blood immune cells by multicolor fluorescence-activated cell sorter (FACS) analysis and of the transcriptomic profiling of blood immune cells by RNA sequencing
Measured: Day 0 and through study completion, an average of 1 year
Exploration of the pathophysiological mechanisms of rare autoimmune neurological pathologies.
Knock-out or knock-in animal models for one specific protein will be used to determine in vivo if the pathophysiological mechanisms of rare autoimmune neurological disorder can be induced by the abnormal expression of this protein.
Measured: Day 0 and through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jan 2021
- Study start12 Oct 2020
- Primary completion1 Jan 2030 (estimated)
- Study completion3 Sep 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Nervous System DiseasesStudies of the same condition, open and under way.
- More trials run by University Hospital, ToulouseThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Toulouse
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Chloé Bost, PharmD, PhD, Principal investigator, University Hospital, Toulouse
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04698421
- Sponsor's protocol ID
- RC31/20/0150
- Acronym
- EXPLAINEUR
- Status last verified
- Mar 2026
- First submitted
- 23 Dec 2020
- Last updated
- 19 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Collection of Biological Samples From Patients With Rare Neurological Diseases
- ClinicalTrials.gov ID
- NCT04698421
- Acronym / sponsor ID
- EXPLAINEUR / RC31/20/0150
- Conditions
- Nervous System Diseases
- Keywords
- autoimmune encephalitis, paraneoplastic neurological syndrome, myasthenia, paraneoplastic cerebellar degeneration, rare neuropathologies with known/suspected autoimmune origin
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- University Hospital, ToulouseUniversity, hospital or other
- Study start
- 12 Oct 2020 (actual)
- Primary completion
- 1 Jan 2030 (estimated)
- Study completion
- 3 Sep 2030 (estimated)
- First posted
- 6 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this project is to improve biological collections of patients presenting rare neurological disorders with known or suspected autoimmune origin. This collection will provide appropriate biological samples to identify new biomarkers and to be accessible to the medical, scientific and industrial communities for the identification of new therapeutic strategies.
Detailed description
Neuroimmunology is a rapidly expanding field since major advances have been made in basic immunology and numerous new clinical entities have been identified in the last 10 years. Even if these discoveries have led to major advances in patient's management and treatment, a lot of work needs to be done to improve the diagnosis and prognostic biomarkers. It is widely known that the immune system is implicated in a variety of neurological disorders such as infections, encephalitis or multiple sclerosis. Numerous neurological disorders affecting the central and peripheral nervous system can be attributed to the immune system and need to be recognized as some of them can be cured by appropriate immunotherapy. These neurological disorders include autoimmune encephalitis and paraneoplastic neurological syndromes but also myasthenia, chronic demyelinating inflammatory polyneuropathy and other neuromuscular pathologies.
These neurological disorders are characterized by the presence of autoantibodies in the patient's sera or cerebral spinal fluid (CSF). These autoantibodies are generally highly specific and necessary to make the diagnosis. However, in some cases, despite strong clinical arguments for a neuroimmunological disorder, we do not identify autoantibodies, leading to inappropriate treatment and a blind follow-up considering the risk of recurrence or of associated tumor. Furthermore, even if the specific role of some autoantibodies or of immune T cells in some of these pathologies are suspected or already documented, for most of them the exact mechanism is still unknown. We need to explore the sera and CSF of these patients to identify new diagnosis and prognosis biomarker. Moreover, the availability of immune cells isolated from these patients will help us to decipher the pathophysiological mechanisms to create new therapeutic strategies. For this, animal models are already available in Centre Physiopathology Toulouse and in the French reference center in Lyon. As genetic susceptibilities may underlie, at least in part, the variability of the clinical manifestations and of the response to treatment, DNA from patients will be collected and immune genes sequencing will be compared to other control groups, included international database.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| patients with rare autoimmune neurological diseases | – | Biological: Blood collection on admission and longitudinally |
Who may qualify
- Ages
- 6 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- patients followed at the Toulouse University Hospital and presenting rare neurological disorders with known or suspected autoimmune origin
Key inclusion criteria (2 listed)
- all patients with neurological disorders, with known or probable autoimmune involvement. This includes adults and children and peripheral and/or central nervous system symptoms.
- Social coverage up to date.
Key exclusion criteria (4 listed)
- Patients with neurological damage from which the autoimmune character can be excluded.
- Known anemia and hemoglobin <10 g / dl
- Patients under protective supervision (guardianship, curators)
- Pregnant or breastfeeding woman
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Blood collection on admission and longitudinallyBiological samples will be collected in the normal diagnosis and follow-up process. Only blood will be taken in larger quantity (8 tubes of 7mL). | Biological | patients with rare autoimmune neurological diseases |
Primary outcome
Building a collection of biological samples and clinical-biological data from patients with rare autoimmune neurological diseases
Time frame: Day 0 and through study completion, an average of 1 year
Blood sampling
3 secondary outcomes
Identification of new autoantibodies.
Time frame: Day 0 and through study completion, an average of 1 year
Identification of biomarkers regarding the severity (such as cytokines, axonal damages...) in order to help the therapeutic decisions.
Time frame: Day 0 and through study completion, an average of 1 year
Exploration of the pathophysiological mechanisms of rare autoimmune neurological pathologies.
Time frame: Day 0 and through study completion, an average of 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Chloé Bost, PharmD, PhD principal investigator
University Hospital, Toulouse
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Nervous System Diseases, run by University Hospital, Toulouse at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chloé Bost, PharmD, PhD (5 61 77 61 44 ext. 0033, bost.c@chu-toulouse.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Building a collection of biological samples and clinical-biological data from patients with rare autoimmune neurological diseases (time frame: Day 0 and through study completion, an average of 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Mar 2026.