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

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 - NBIA-prospective

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

DRKS

Registry ID

DRKS00003106

Enrollment

Registered

2011-07-11

Start date

2009-07-01

Completion date

Unknown

Last updated

2025-04-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

NBIA with different subtypes including PKAN Neurodegeneration with Brain Iron Accumulation (NBIA), formerly known as Hallervorden-Spatz-disease NBIA dystonia G23.0

Interventions

Group 1: NBIA patients with NBIA dystonia: Documentation of epidemiological data, clinical scales, quality of life scales and standardized video preoperatively, at 3, 12 and 24 months

Eligibility

Sex/Gender

All

Age

3 Years to 80 Years

Inclusion criteria

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

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

Design outcomes

Primary

Measure	Time frame
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

Measure	Time frame
Severity of disability at 12 months, assessed with the Burke Fahn Marsden Dystonia rating Scale disability scale (BFMDRS-D); 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	—

Measure

Time frame

Severity of disability at 12 months, assessed with the Burke Fahn Marsden Dystonia rating Scale disability scale (BFMDRS-D); 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

—

Countries

Canada, France, Germany, India, Italy, Japan, Poland, South Korea, Spain, Switzerland, Turkey, United Kingdom, United States

Contacts

Public ContactAmande Pauls

Neurologie, Uniklinik Köln

amande.pauls@uk-koeln.de0221-47897818

Outcome results

None listed

Source: DRKS (via WHO ICTRP) · Data processed: Feb 4, 2026