Therapeutic Approach of Repeated Transient Blood-brain Barrier Opening in Amyotrophic Lateral Scl… (NCT07571486) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
Therapeutic Approach of Repeated Transient Blood-brain Barrier Opening in Amyotrophic Lateral Sclerosis.
France23 participantsStarted 2026-06-15
Plain-language summary
This is proof-of-concept, single-arm, single-center study to assess the safety and explore the efficacy of repeated US transient disruptions of the blood-brain barrier (BBB) in Amyotrophic Lateral Sclerosis (ALS).
Phase 1:
The primary objective is to assess the safety of ultrasound induced BBB opening in the upper motor neuron area and adjacent supplementary motor area in adult patients with ALS, as assessed by adverse events frequency and severity during study (incidence of AE summarized by system organ class and/or preferred term and severity) based on the Common Terminology Criteria for Adverse Events, version 5.0 A run-in period of 12 weeks between inclusion and baseline will take place for each patient in order to evaluate precisely disease progression rate, disease severity and to collect concomitant medication. After this run-in period, the patient will be implanted with the SC4 device (baseline visit). The first sonication session will be performed two weeks after implantation. A total of 9 sonications, with no concomitant drug administration, will be performed over a period of 24 weeks.
Phase 2a:
Based on the safety outcome of the Phase 1, an expansion cohort will open to assess the first signal of efficacy of the US transient disruptions of the BBB in ALS. The primary objective will be to assess the first signal of efficacy of the procedure on disease progression over 26 weeks evaluated by the change from baseline to week 26 of neurofilament light (NfL) levels in blood.The Phase 2a will continuously include 11 additional patients. Patients will be treated according to the same schedule as in phase 1
Who can participate
Age range
18 Years – 80 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age 18-80 years,
. Able and willing to give signed and informed consent
. Confirmed diagnosis of ALS using the Gold Coast criteria with involvement of both upper and lower motor neurons in at least one body region, i.e. patients classified as "definite", "probable", "probable laboratory supported" or "possible" ALS using the El Escorial categories (patients with only lower motor neuron involvement will not be included)
. Disease duration \<= 36 months,
. ALSFRS-R ≥30,
. Change in ALSFRS-R score between 0.35 points and 1.1 points per month (both inclusive) in the period from onset of first symptoms to the Screening visit,
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Phase 1 : Adverse events frequency and severity during study to assess the safety of ultrasound induced BBB opening in the upper motor neuron area and adjacent supplementary motor area
Timeframe: from inclusion up to 50 weeks
2
Phase 2a : Neurofilament light (NfL) levels in blood to assess the first signal of efficacy of the procedure on disease progression
. If taking riluzole, patient on stable dose for over 30 days prior to study entry,
Exclusion criteria
. Patients with an uncontrolled intercurrent illness or any pre-existing comorbidities that in the Investigator's opinion may prevent the implantation of the device or may impair the ability of the patient to receive treatment with SonoCloud or may be cofounding for evaluation of the clinical trial
. Cardiac pacemaker (contraindication to MRI)
. Allergy to any drug used in the study (Gadolinium, Xylocain, Cloxacilline, EMLA, echographic contrast agent microbubbles: SonoVue®)
. Severe or instable chronic or acute disease or any life-threatening condition
. Other significant neurological or psychiatric disease in addition to ALS, including history of uncontrolled seizures
. Treatment with edaravone, tofersen or with another investigational drug or biological agent within 1 month or 5 half-lives of study agent, whichever is longer, before screening
. Invasive or non-invasive mechanical ventilation use