A single-arm, single-center proof-of-concept study.
Topic / Pathology
- ALS
Objectives
Primary objective:
To assess the safety of BHE opening at the level of upper motor neurons by evaluating the frequency and severity of adverse events during the study.
Secondary objective:
To assess the efficacy of the procedure on the progression of ALS over 26 weeks using the ALSFRS-R, muscle strength, and neurofilament levels.
Sponsor
Assistance Publique – Hôpitaux de Paris (AP-HP)
Investigator
Pr Gaëlle Bruneteau
Critères d'inclusion
- Age : 18 - 80 years old
- Duration of the disease ≤ 36 months
- ALSFRS-R ≥ 30
- SVC ≥ 70% of the theoretical value
- Change in ALSFRS-R between 0.35 and 1.1 points per month
- If taking riluzole, dose must have been stable for 30 days prior to study enrollment
Critères de non-inclusion
- Presence of comorbidities incompatible with the implantation or use of SonoCloud
- Cardiac pacemaker
- Allergy to gadolinium, lidocaine, cloxacillin, EMLA, or SonoVue® (contrast agent)
- Chronic, severe, or unstable life-threatening disease
- Psychiatric or neurological disease other than ALS
- Currently taking edaravone, tofersen, or another experimental drug
- Use of invasive or non-invasive ventilation
- Use of a gastrostomy or nasogastric tube
- Known right-to-left shunts
- Known severe pulmonary hypertension (pulmonary
arterial pressure > 90 mmHg), uncontrolled
systemic hypertension
- Known respiratory distress syndrome
- Women of childbearing age or sexually
active men not using contraception
- Pregnant or breastfeeding women
- History of or a positive result on an HIV screening test,
current hepatitis C infection (defined
as positive anti-HCV antibodies and detectable HCV RNA
), or current hepatitis B infection (defined
as a positive result for HBsAg and/or total anti-
HBc antibodies) . Participants with positive anti-HCV antibodies
and undetectable HCV RNA may
participate in the study. Participants who are immune to
hepatitis B as a result of a previous natural
infection (defined as HBsAg-negative, anti-HBc
positive, and anti-HBs positive) or vaccination (defined
as HBsAg negative, anti-HBc negative, and anti-HBs
positive) are eligible to participate in the study.
- Non-affiliated patients who are beneficiaries of a
social security category
Status
Recruiting
