Acetyl-leucine to improve balance after posterior-circulation ischemic stroke

The Safety and Efficacy of Acetyl-leucine in Post-stroke Ataxia: a Randomized Placebo-controlled Trial

It will test whether taking 4 grams of acetyl-leucine daily for three months helps people with gait and coordination problems after a posterior-circulation ischemic stroke.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 200 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Kafrelsheikh University (other)
• Locations: 1 site (Kafr ash Shaykh)
• Trial ID: NCT07275749 on ClinicalTrials.gov

What this trial studies

Two hundred adults with first-ever posterior-circulation ischemic stroke and measurable gait ataxia will be randomized within 24 hours to receive either 4 g/day acetyl-leucine or matching placebo for 90 days in a double-blind, placebo-controlled, parallel-group design. All participants receive standard antiplatelet therapy (loading doses of aspirin and clopidogrel, then daily aspirin 100 mg and clopidogrel 75 mg) and undergo baseline CT/CTA and follow-up MRI per protocol. The main outcomes are balance and ataxia scores (Berg Balance Scale, SARA) and functional status (mRS) over the three-month treatment period, with safety monitoring for adverse events. The single-center trial is planned at Kafr Elsheikh University Hospital between January and July 2026.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18–80 with a first-ever posterior-circulation ischemic stroke, symptom onset within 24 hours, measurable gait ataxia (≥1 point on key SARA items) and a Berg Balance Scale score of 47 or less.

Not a fit: Patients with very severe baseline disability (mRS ≥5), major cognitive or communication impairments, non-posterior strokes, prior disabling stroke, or those presenting beyond the 24-hour enrollment window are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, treatment could improve balance and reduce disability from post-stroke ataxia, helping patients regain mobility faster.

How similar studies have performed: Small open-label and early-phase reports of acetyl-leucine in cerebellar and genetic ataxias have reported symptomatic improvements, but a large randomized Phase 3 trial for post-stroke ataxia is novel.

Eligibility criteria

Inclusion Criteria:

* the investigators included both males and females, aged between 18 and 80 years, who experienced their first-ever posterior-circulation ischaemic stroke and presented with gait ataxia and score at least 1 point on the items gait, stance, trunk or heel-shin-slide of the Scale for the Assessment and Rating of Ataxia (SARA) and 47 points or less on the Berg Balance Scale (BBS).
* All of our patients underwent randomisation during the first 24 hours of the symptoms' onset, and within the first 24 hours from the time at which the patient's condition was last reported to be normal for wake-up stroke patients.

Exclusion Criteria:

* the investigators excluded patients with modified Rankin Scale (mRS) score of 5 or more, physical or mental conditions that would not allow safe participation in the study or would influence the assessment of outcomes (e.g., dementia, disturbed conscious level, severe aphasia, etc.).
* the investigators ruled out participants who suffered from neurological diseases associated with recurrent neurological deficits, such as (epilepsy, multiple sclerosis, head trauma followed by neurological deficit).
* the investigators excluded participants who underwent intravenous thrombolysis, carotid, cerebrovascular, or coronary revascularization during the first week of the trial to avoid clouding of efficacy and safety analysis.
* the investigators excluded patients who experienced recurrent ischemic stroke detected from their clinical data and/or magnetic resonance imaging (MRI) brain findings. In addition, we excluded participants who experienced hypersensitivity to the study treatment
* the investigators excluded participants who experienced organ failure such as renal failure and liver cell failure, active malignancies, and patients with a known bleeding diathesis or coagulation disorder, a history of intracerebral hemorrhage, gastrointestinal bleeding within the past 6 months, or major surgery within 30 days before randomisation .

the investigators excluded pregnant and lactating women, patients with cerebral venous thrombosis, and patients with stroke associated with cardiac arrest.

Where this trial is running

Kafr ash Shaykh

• Kafr Elsheikh University Hospital — Kafr ash Shaykh, Egypt (RECRUITING)

Study contacts

• Study coordinator: Mohamed G. Zeinhom, MD

Conditions: Ischemic Stroke, Acetyl-leucine, post-stroke ataxia, posterior-circulation stroke