Movement Disorders Clinical Trials

IB1001-303

Safety and efficacy of levacetylleucine in ataxia-telangiectasia: a phase 3, randomised, double-blind, placebo-controlled crossover trial

Lancet Neurology · 2026

Bottom Line: In this first positive phase 3 trial in ataxia-telangiectasia, levacetylleucine produced a statistically significant and clinically meaningful improvement in SARA (treatment effect −1·88 points, 95% CI −2·70 to −1·06; p

CEREBELLAR ATAXIA 1

Levacetylleucine for AT

2026

In this first positive phase 3 trial in ataxia-telangiectasia, levacetylleucine produced a statistically significant and clinically meaningful improvement in SARA (treatment effect −1·88 points, 95...

Lancet Neurology

PHARMACOTHERAPY 1

LCE vs LB

2026

In 90 patients with Hoehn & Yahr stage 1-1.5 Parkinson's disease, a single dose of LCE and LB produced similar plasma dopamine elevations at 90 and 180 minutes, with no significant between-group or in...

Medicine (Baltimore)

PARKINSON'S DISEASE 5

Does staged, bilateral MRgFUS pallidothalamic tractotomy offer a favorable risk-benefit profile for advanced Parkinson's disease with motor complications?

MRGFUS

2026

Lancet Neurology

Can fixed-dose tavapadon, a selective D1/D5 agonist, improve motor function in early Parkinson disease while avoiding the adverse effects of D2/D3 agonists?

TEMPO-1

2026

JAMA Neurology

Does continuous subcutaneous infusion of levodopa or foslevodopa reduce daily OFF-time more effectively than oral levodopa in Parkinson's disease patients with motor fluctuations?

SC LEVODOPA PD

2026

European Journal of Neurology

Does adding amantadine-IR early in levodopa therapy reduce the risk of developing dyskinesia in Parkinson's disease?

PREMANDYSK

2026

Movement Disorders View Details →

Does azathioprine, a broadly acting peripheral immunosuppressant, slow clinical progression in early Parkinson's disease as measured by the MDS-UPDRS gait-axial score?

AZA-PD

2025

Lancet Neurology

DISEASE-MODIFYING THERAPY 1

REASON

2026

Intrathecal BIIB094 was generally well tolerated with no drug-related serious adverse events and produced robust, dose-related lowering of CSF LRRK2 protein (up to 59%) and phosphorylated Rab10 (up to...

Nature Medicine

NON-INVASIVE NEUROMODULATION 3

GPi tbTUS in PD

2026

Bilateral GPi tbTUS produced a clinically meaningful 4.1-point reduction in MDS-UPDRS-III at 30 min post-sonication (p=0.002), driven primarily by bradykinesia improvement; M1-only and dual-site M1+GP...

Movement Disorders

Home-Based taVNS in PD

2026

Home-based bilateral taVNS reduced MDS-UPDRS-III by 6.10 points and significantly improved quality of life, sleep, and autonomic function vs sham over 3 weeks, with elevated serum acetylcholine and re...

Neurotherapeutics

M1-iTBS in Mid-Stage PD

2026

Bilateral M1-iTBS produced a 30.7% reduction in MDS-UPDRS Part III at 2 weeks (vs 14.3% sham, p=0.036) plus large improvements in depression (-47.6%) and anxiety (-37.9%), accompanied by increased cer...

Neurotherapeutics

DISEASE-MODIFYING THERAPY 1

VIBRANT-HD

2026

Branaplam 56 mg weekly lowered CSF mHTT by 25.2% (placebo-corrected) at week 17 but caused peripheral neuropathy in 85.7% of treated participants, leading to early termination and discontinuation of N...

Nature Medicine

TOURETTE SYNDROME 1

Does ecopipam, a selective dopamine D1 receptor antagonist, maintain tic improvement and remain well tolerated for up to 24 weeks in patients with Tourette syndrome?

Ecopipam Tourette

2026

JAMA Neurology

MULTIPLE SYSTEM ATROPHY 1

Does the anti-α-synuclein monoclonal antibody amlenetug slow clinical disease progression in patients with multiple system atrophy?

AMULET

2026

The Lancet Neurology

RANDOMIZED CONTROLLED TRIAL 1

Can once-daily tavapadon, a selective D1/D5 dopamine agonist, improve motor control and reduce off-time in Parkinson disease patients with motor fluctuations on levodopa while minimizing adverse events?

TEMPO-3

2026

JAMA Neurology