Stimulation of the Globus pallidus internus in patients with NBIA (formerly Hallervorden-Spatz-Syndrome): prospective analysis of international therapeutic outcomes and development of a therapeutic algorithm

Recruiting

• Conditions: BIA with different subtypes including PKANNeurodegeneration with Brain Iron Accumulation (NBIA), formerly known as Hallervorden-Spatz-diseaseNBIA dystonia; G23.0; Hallervorden-Spatz disease

• Registration Number: DRKS00003106
• Lead Sponsor: Medizinische Fakultaete der Universitaet zu Koeln, Uniklinik Koeln, Klinik und Poliklinik fuer Neurologie

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-07-11
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

(1)Diagnosis of NBIA
(2)Presence of dystonia
(3)Patient is appropriate candidate to undergo bilateral GPi-DBS
(4)Patient, legal representative if applicable, and attending doctor have chosen GPi-DBS as treatment
(5)Sufficient compliance to take part in the study
(6)Informed consent to take part in the study from patient or legal representative

Exclusion Criteria

(1)Patients with other severe concurrent neurological disease (e. g. brain tumor, severe brain damage due to perinatal hypoxia or trauma etc.)
(2)Contraindication for DBS surgery or surgery in general (i.e. coagulopathy etc.)
(3)Condition likely to require use of MRI in the future
(4)Pregnancy

Study & Design

• Study Type: observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
blinded ratings on the Burke-Fahn-Marsden-Dystonia-Rating Scale motor score (BFMDRS-M) 12 months postoperatively compared to preoperative status. The scale is a clinical scale and rates the severity of dystonia.

Secondary Outcome Measures

Name	Time	Method
Severity of disability at 12 months, assessed with the Burke Fahn Marsden Dystonia rating Scale disability scale (BFMDRS-D);<br>quality of life and ease of care at 12 months, measured using the Comfort, Care and Hypertonicity Questionnaire; pain assessment at 12 months assessed using Wong-Baker-Faces; Quality of life at 12 months assessed using the SF-36; Severity of dystonia at 3 and 24 months postoperativly, assessed with the Burke Fahn Marsden Dystonia Rating Scale motor score (BMFDRS-M); role of genetic status in outcome prediction of improvement in severity of dystonia as assessed using the Burke Fahn Marsden Dystonia rating scale