Establishment of a CSF Bank for the Development of Biomarkers of Smooth Muscle Cell (SMC) Damage in Monogenic Cerebral Small Vessel Disease

NCT ID: NCT05793424

Last Updated: 2023-03-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Total Enrollment

90 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-03-31

Study Completion Date

2025-04-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The main objective of this research is to obtain biological markers of smooth muscle cells dysfunction or degeneration in cerebral small vessel diseases.

The aim of this research is therefore to build up a biocollection of CSF and blood samples from 1) patients with CADASIL disease (the most common form of cSVD) responsible for an accumulation of the NOTCH3 protein in the microvessel wall, 2) patients with other forms of monogenic cSVD (rarer) which are not responsible for an accumulation of this protein despite the damage to the smooth muscle cells of the vessel wall and 3) control patients without cSVD, collected in the context of care.

This bio-collection will allow the identification and assay of markers testifying to the damage of the smooth muscle cells (SMC) in different types of cSVD of hereditary origin, the first of which will be the soluble NOTCH3 protein.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cerebral Small Vessel Diseases CADASIL (Diagnosis)

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

CASE_CONTROL

Study Time Perspective

OTHER

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Patients with cerebral small vessel disease ( cSVD)

Cerebrospinal fluid (CSF) sample and additional blood samples

Intervention Type OTHER

Lumbar puncture (Spinal Tap) and additional blood samples

Control patients

No interventions assigned to this group

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Cerebrospinal fluid (CSF) sample and additional blood samples

Lumbar puncture (Spinal Tap) and additional blood samples

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Patients aged between 18 and 80 years at the time of inclusion
* Diagnosis confirmed by detection of a pathogenic mutation in the NOTCH3 gene characteristic of CADASIL, or in another gene responsible for other forms of monogenic cSVD (such as COL4A1, COL4A2, HTRA1).
* Beneficiary of a social security system
* Having given their written consent


* Contraindication to lumbar puncture:
* Haemostasis disorder (severe thrombocytopenia \<60,000/mm3, PT abnormalities, INR\>1.5 and/or aPTT\>1.5) or anticoagulant use.
* Spinal plaque in or near the lumbar region (surgery) that may interfere with CSF collection
* Behavioural disorder that may interfere with the sampling process
* Intracranial process, intracranial hypertension or risk of involvement on imaging
* Skin lesions (inflammation or infection of any kind) or developmental abnormality (myelomeningocele) adjacent to the puncture site
* Person referred to in articles L. 1121-5 to L. 1121-8 and L. 1122-12 of the public health code, defined as :
* Pregnant, parturient or breastfeeding woman
* Person deprived of liberty by judicial or administrative decision
* Person hospitalised without consent and not subject to a legal protection measure, and person admitted to a health or social establishment for purposes other than research
* Minor
* Person of full age subject to a legal protection measure (guardianship, curators or safeguard of justice), person of full age unable to express their consent and not subject to a protection measure
* Person subject to an exclusion period for another research
* Patients participating in other interventional research
* Contraindication to lumbar puncture:
* Haemostasis disorder (severe thrombocytopenia \<60,000/mm3, PT abnormalities, INR\>1.5 and/or aPTT\>1.5) or anticoagulant use.
* Spinal plaque in or near the lumbar region (surgery) that may interfere with CSF collection
* Spinal plaque (surgery)
* Behavioural disorder that may interfere with the sampling process
* Intracranial process, intracranial hypertension or risk of involvement on imaging
* Skin lesions (psoriasis, infection....)inflammation or infection of any kind) or developmental abnormality (myelomeningocele) adjacent to the puncture site
* Person referred to in articles L. 1121-5 to L. 1121-8 and L. 1122-12 of the public health code, defined as
* Pregnant, parturient or breastfeeding woman
* Person deprived of liberty by judicial or administrative decision
* Person hospitalised without consent and not subject to a legal protection measure, and person admitted to a health or social establishment for purposes other than research
* Minor
* Person of full age subject to a legal protection measure (guardianship, curators or safeguard of justice), person of full age unable to express their consent and not subject to a protection measure
* Person subject to an exclusion period for another research
* Patients participating in other interventional research

For control patients ( selection after CSF sampling):


* Subject aged 18-80 years at the time of inclusion
* Imaging done: strictly normal CT scan OR MRI showing no abnormality suggestive of cSVD
* Beneficiary of a social security system
* Having had CSF and blood samples taken as part of the treatment
* Agreement to use blood and CSF samples remainders for research purposes (information and non-opposition).
* Women who were pregnant at the time the samples were taken will not be offered to participate in the study


* Any previously identified intracranial vascular pathology requiring specific management (vasculitis, acute reversible angiopathy, severe intracranial atheroma)
* Acute or chronic infectious disease (meningitis or meningoencephalitis)
* Known pregnancy
* Persons participating in other interventional research
* Preparation and freezing time for blood and CSF samples is more than 4 hours
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

CHABRIAT Hugues, Pr

Role: CONTACT

Phone: +33 1 49 95 25 93

Email: [email protected]

Matthieu RESCHE-RIGON, Pr

Role: CONTACT

Phone: +33 1 42 49 97 42

Email: [email protected]

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

APHP220744

Identifier Type: -

Identifier Source: org_study_id