NCT07571486RecruitingPhase 1/2

Therapeutic Approach of Repeated Transient Blood-brain Barrier Opening in Amyotrophic Lateral Sclerosis.

Therapeutic Approach of Repeated Transient Blood-brain Barrier Opening in Amyotrophic Lateral Sclerosis. A One-arm, Single-center, Proof-of-concept Study (Son-ALS)

Source: ClinicalTrials.gov · Last updated 10 Aug 2026

Brief summary (registry, in English)

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

Status (registry): Recruiting
Phase (registry): Phase 1/2
Study type: Interventional
Enrollment: 23 (estimated)
First submitted: 30 Apr 2026
Start date: 15 Jul 2026
Primary completion date: 15 Jul 2030
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Diseases: ALS (motor neurone disease)
NCT number: NCT07571486
EU CT number: 2024-513471-40-00

What you commit to

Study type: Interventional · Phase 1/2
Planned participants: 23
From 15 Jul 2026 to the primary completion date, 15 Jul 2030
1 site in 1 country
Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

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
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
History or positive test result at screening for HIV, current hepatitis C infection (defined as positive HCV antibody and detectable HCV RNA) or current hepatitis B infection (defined as positive for HBsAg and/or total anti-HBc). Participants with positive HCV antibody and undetectable HCV RNA are eligible to participate in the study. Participants with immunity to hepatitis B from previous natural infection (defined as negative HbsAg, positive anti-HBc, and positive anti-HBs) or vaccination (defined as negative HbsAg, negative anti-HBc, and positive anti-HBs) are eligible to participate in the study.

Other illness

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
Known severe pulmonary hypertension (pulmonary artery pressure \>90 mmHg), uncontrolled systemic hypertension
Known respiratory distress syndrome

Other

Invasive or non-invasive mechanical ventilation use
Gastrostomy or nasogastric tube use
Known right-to-left shunts,
Women of child-bearing potential or sexually active man, without contraception
Pregnant or breast-feeding woman
Patient not affiliated or beneficiary of a social security category

Who the study is looking for

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,
Mild functional impairment (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,
Slow Vital capacity \>= 70% of normal,
If taking riluzole, patient on stable dose for over 30 days prior to study entry,
Able and willing to follow trial procedures including site travels and visit requirements

Age in the registry: 18 years to 80 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (1)

Sites in registry order. Save your city in your profile to sort them by distance.

Site	City	Country	Site status
Assistance Publique Hopitaux de Paris Coordinator of this site? Confirm its status	Paris	France	Recruiting

This page is information, not medical advice. Discuss any trial with your doctor.