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

Sponsor
Assistance Publique - Hôpitaux de Paris
Study ID
NCT07571486
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - 80 Years
Healthy Volunteers
Not accepted

Interventions

  • SonoCloud-4 (SC4) device — DEVICE
    Implantation of SC4 device and 9 Blood Brain Barrier opening sessions by ultrasound without any associated drug

Study Details

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

Key Dates

First listed
May 6, 2026
Start date
Jul 15, 2026
Status verified
Aug 2026
Primary completion
Jul 15, 2030
Completion
Jul 15, 2031

Study Design

Enrollment
23 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Sonications
    Repeated US transient disruptions of the blood-brain barrier

Primary Outcome Measure

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 [ Time Frame: from inclusion up to 50 weeks ]

Central Contacts

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