The HD-DBS Study – Recruiting
Deep Brain Stimulation (DBS) of the Globus pallidus (GP) in Huntington’s disease (HD): A prospective, randomised, controlled, international, multi-centre study.
PI: Prof. Dr. med. Jan Vesper, Co-I: Prof. Dr. med. Alfons Schnitzler
Single case reports have revealed beneficial effects of pallidal DBS on the debilitating motor symptoms of HD. This multi-centre trial aims at proving the efficacy and safety of pallidal DBS in 50 HD patients. Sample size is based on our previous controlled randomised pilot study, which provided strong evidence for the optimal target point to be within the GPi/GPe border zone, and a simulation analysis performed to access the risk that the trial could be underpowered.
The objective of this trial is to demonstrate the efficacy and safety of pallidal DBS in HD patients, and show superiority of DBS on motor function in the stimulation group compared to stimulation-off group and that DBS is a good alternative to medication therapy. For ethical reasons all patients (including the controls) will receive best contact stimulation after assessment of the primary endpoint for 3 months and a final follow up is planned 6 month after study inclusion. All patients will receive a bilateral stereotactic insertion of quadripolar electrodes (Medtronic Inc, 3387) into the Globus pallidus (GP) for chronic high-frequency stimulation with the upper two contacts reaching the external GP (GPe) and the lower two contacts placed in the internal GP (GPi). The implantation of the stimulator (ACTIVA®PC) will take place in the same procedure. This approach has been successfully used in previous studies of DBS in dystonia.
What is the HD-DBS Study?
The HD-DBS Study is a prospective, randomised, double blind, parallel group, sham-controlled, multi-centre superiority trial. It is the first study on Deep Brain Stimulation (DBS) of the pallidum in HD which includes the following novel aspects:
- The implantation of neurostimulation devices for the treatment of neurodegenerative disorders is a powerful neurotechnological tool, as shown over the last two decades. Its clinical potential, efficacy and safety for neurodegenerative diseases has been fully elucidated for other disorders, and will be the primary focus of this HD study.
- All participating European centres in the study follow the same clinical study protocol, neurosurgical techniques and evaluation protocol, allowing the analysis of comparable data in a much shorter time period on a substantial number of patients.
The aim of the study is to prove the efficacy and safety of pallidal DBS in HD patients, to show superiority of DBS on motor function in the stimulation group compared to stimulation-off group and to show, that DBS is a good alternative to medication therapy.
Who is eligible to participate in the HD-DBS trial?
- Age ≥18 years old
- Able and willing to provide written informed consent prior to any trial-related procedure
- Clinically symptomatic and genetically confirmed HD (number of CAG repeats ≥ 36)
- Moderate stage of the disease (UHDRS motor score ≥ 30)
- Chorea despite best medical treatment (UHDRS chorea subscore ≥ 10)
- Mattis Dementia Rating Scale ≥ 120
- Stable medication 6 weeks prior to inclusion and not participating in any interventional clinical trial within 2 months before screening
- Juvenile HD (Westphal variant) or predominant bradykinesia
- Postural instability with UHDRS retropulsion score > 2
- No implanted pacemaker or defibrillator
Who is running the HD-DBS Trial?
The HD-DBS study is funded by the DFG and has EHDN endorsement. The devices will be provided by Medtronic and the George-Huntington-Institut GmbH in Münster will support the study with Q-Motor-devices.
80 patients are expected to be assessed for eligibility and 50 patients will be randomised in total in 8 European trial sites. Patients will receive treatment of bilateral stereotactic insertion of quadripolar electrodes (Medtronic Inc., 3387) into the GP and stimulator implantation (ACTIVA® PC) for chronic high-frequency stimulation. The duration of intervention per patient will be 6 months (12 weeks parallel group, 12 weeks open follow-up).
How can I learn more about the HD-DBS trial?
If you are a clinician who may have eligible patients for the study, please contact Pauline Kleger (firstname.lastname@example.org or email@example.com), EHDN Language Area Coordinator, for further information.
ClinicalTrials.gov identifier: NCT02535884