SUNDYS: A Multicenter, Randomized, Double-blind, Sham-controlled, Parallel-group Trial

Summary

Dystonia is a group of movement disorders characterized by twisting, repetitive movements, or abnormal postures caused by involuntary muscle contractions and is characterized by a young age of onset and a high disability rate. Early intervention can reduce disability incidence, improve the patient's quality of life, and reduce the burden on families and society. Multiple international guidelines on dystonia have found deep brain stimulation (DBS) to be a safe and effective treatment for refractory dystonia. The globus pallidal internus (GPi) is the mostly widely used target for dystonia. However, there are limitations on the GPi DBS treatment, including slow onset of beneficial effects, poor improvement of axis symptoms, and potential stimulation-related side effects. Previous studies have described the highly successful use of subthalamic nucleus deep brain stimulation (STN DBS) in patients with refractory dystonia, suggesting that STN DBS is an effective and persisting alternative to pallidal deep brain stimulation. However, all STN DBS treated cases have been analyzed in open-label uncontrolled cohort studies, leading to limited data with a high level of evidence on the STN DBS in dystonia. Further, the investigators hypothesized STN has potentially more effectiveness when compared with GPi, and may be more power-saving and quick-acting. In this study, the investigators will organize a prospective randomized, double-blind, parallel-group, multicenter study comparing active versus sham stimulation in isolated segmental or generalized dystonia to evaluate the effectiveness and safety of STN DBS by measuring the impact on motor status, mental status, quality of life, the rate of response of the patients (the number of patients with ≥30% improvement in the movement score on the Burke-Fahn-Marsden Dystonia Rating Scale) and the rate of adverse events during the trial.

Conditions

• Dystonia

Interventions

PROCEDURE

Deep brain stimulation

Deep brain stimulation (DBS) has been in use to treat patients with movement disorders since 1989, with many thousands of publications showing its effectiveness. DBS for dystonia received the US FDA mark in 2003 and China FDA mark in 2016. In this study, the DBS system devices are manufactured and donated by SceneRay (Suzhou, China). The Stimulator System is implanted by a qualified neurosurgeon and consists of three implantable components: the leads, the extension wires and the neurostimulator. The DBS programming will start within 1 week after the surgery completed.

Sponsors & Collaborators

• Renmin Hospital of Wuhan University

  collaborator OTHER

• Shanghai Tongji Hospital, Tongji University School of Medicine

  collaborator OTHER

• Second Affiliated Hospital of Soochow University

  collaborator OTHER

• West China Hospital

  collaborator OTHER

• Ruijin Hospital

  lead OTHER

Principal Investigators

• Bomin Sun · Ruijin Hospital

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

14 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2021-01-01

Primary Completion

2021-12-01

Completion

2022-01-01

Countries

• China

Study Locations