Repeated ultrasound-assisted blood-brain barrier opening in ALS

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a proof-of-concept, single-arm study conducted at one center to examine the safety and potential signs of efficacy of repeated low-intensity ultrasound–induced BBB opening in people with amyotrophic lateral sclerosis (ALS). Phase 1 focuses on safety, monitoring adverse events after implanting the SonoCloud-4 device and performing initial BBB openings in motor cortex regions; phase 2a aims to observe early efficacy signals based on motor function and disease progression. Participants undergo a 12-week run-in period for baseline progression assessment, followed by device implantation and scheduled sonication sessions. Clinical evaluations include motor function tests, pulmonary function, and MRI to plan positioning and monitor safety. The approach leverages transient BBB disruption to potentially enhance therapeutic effects or modulate CNS immune responses in ALS.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Age 18-80 years,
2. Able and willing to give signed and informed consent
3. 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)
4. Disease duration \<= 36 months,
5. Mild functional impairment (ALSFRS-R ≥30),
6. 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,
7. Slow Vital capacity \>= 70% of normal,
8. If taking riluzole, patient on stable dose for over 30 days prior to study entry,
9. Able and willing to follow trial procedures including site travels and visit requirements

Exclusion Criteria:

1. 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
2. Cardiac pacemaker (contraindication to MRI)
3. Allergy to any drug used in the study (Gadolinium, Xylocain, Cloxacilline, EMLA, echographic contrast agent microbubbles: SonoVue®)
4. Severe or instable chronic or acute disease or any life-threatening condition
5. Other significant neurological or psychiatric disease in addition to ALS, including history of uncontrolled seizures
6. 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
7. Invasive or non-invasive mechanical ventilation use
8. Gastrostomy or nasogastric tube use
9. Known right-to-left shunts,
10. Known severe pulmonary hypertension (pulmonary artery pressure \>90 mmHg), uncontrolled systemic hypertension
11. Known respiratory distress syndrome
12. Women of child-bearing potential or sexually active man, without contraception
13. Pregnant or breast-feeding woman
14. 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.
15. Patient not affiliated or beneficiary of a social security category

Where it's running

• Hôpital Pitié-Salpêtrière
  Paris, France Recruiting

Study contacts

• Study coordinator: Gaelle BRUNETEAU, Pr
• Email: gaelle.bruneteau@aphp.fr
• Phone: 1 42 16 24 72

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.