Dyskinetic Cerebral Palsy Due to Perinatal Hypoxia Clinical Trial
— STIM-CPOfficial title:
Effect of Deep Brain Stimulation in the Globus Pallidus Internus on Quality of Life in Young Patients With Dyston-dyskinetic Cerebral Palsy
| Verified date | May 2021 |
| Source | University Hospital of Cologne |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Observational |
There are limited therapeutical options for patients with secondary dystonia due to cerebral palsy. Pharmacotherapy is often without effect, or side effects are severe. Meanwhile deep brain stimulation (DBS) has proven to be a safe and effective therapy for patients with parkinson´s disease or primary / idiopathic dystonia. Experiences with DBS in patients with dyskinetic cerebral palsy are limited with heterogeneous data. With STIM-CP we investigate the effect of DBS on quality of life in young patients with a dyskinetic movement disorder (dyskinetic cerebral palsy) due to perinatal hypoxic brain injury. Additionally, the effect of DBS on motor development, speech, memory, attention, cognition and pain perception will be assessed.
| Status | Active, not recruiting |
| Enrollment | 16 |
| Est. completion date | December 31, 2022 |
| Est. primary completion date | March 10, 2020 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 7 Years to 18 Years |
| Eligibility | Inclusion Criteria: - The treating physician has chosen GPi-DBS for the treatment of the secondary dystonia caused by cere-bral palsy in this patient - Patient and/or legal representative, if the patient is underaged or not capable to give consent himself, have chosen GPi-DBS as treatment - The consent to participate in the trial of the underaged patient, if he is capable to understand the study requirements, is required - Age at enrolment 7-18 years - Diagnosis of secondary dystonia due to cerebral palsy caused by perinatal hypoxic injury - Anti-dystonic pharmacotherapy insufficient (e.g. Jankovic J. Medical treatment of dystonia. Movement disorders, Vol. 28, No. 7, 2013) 67 - Stable anti-dystonic medication over the last 30 days - Globus pallidus internus (pars posterior) and thalamus (motor part) intact on MRI (not older than 2 years - if possible) - No fixed severe skeletal deformations with loss of function, which need immediate orthopaedic surgical intervention - Sufficient compliance of the patient or the legal representative if the patient is underaged or not capable to give consent himself to take part in the study - Informed consent to take part in the study from patient and/or legal representative if the patient is underaged or not capable to give consent himself - Patient and/or legal representative if the patient is underaged or not capable to give consent himself, understands the study requirements and the treatment procedures and provides written informed consent before any study-specific tests or procedures are performed Exclusion Criteria: - • Patients with known primary (e.g. DYT1) or idiopathic dystonia - Severe axial hypotonia with total loss of head control (e.g. absence of control at "upper thoracic level" in the SATCo score) (medication effect excluded) - Fixed hemi-dystonia - Severe spasticity in knee- and elbow-flexors and -extensors (Modified Ashworth Scale >3) - Fixed severe skeletal contractions with loss of function which require immediate orthopaedic surgical intervention - Patients with other severe concurrent neurological disease (e. g. brain tumor, neurodegenerative diseases, trauma etc.) - Condition likely to require use of MRI in the future - Any intracranial abnormality or medical condition that would contraindicate DBS surgery - Any findings in neuropsychological screening assessments that would contraindicate DBS surgery - Any current drug and / or alcohol abuse - Any history of frequent grand-mal seizures without response to anticonvulsive treatment - Any other active implanted device (e.g. Cochlear implant, pacemaker), whether turned on or off, would be allowed provided that they do not interfere with functioning of the device. - Any previous brain surgery that would interfere with the placement of the leads or the functioning of the device. - A history of neurostimulation intolerance in any area of the body. - Currently on any anticoagulant medications that cannot be discontinued during perioperative period. - Any significant medical condition that is likely to interfere with study procedures or likely to confound evaluation of study endpoints, including any terminal illness with survival <12 months. - Participation in another drug, device, or biologics trial concurrently or within the preceding 30 days; any other trial participation should be approved by the Principal Investigator. - A female that is breastfeeding or of child bearing potential with a positive urine pregnancy test or - if a person is sexually active - not using sufficient contraception with a Pearl Index of less than 1% including all forms of hormonal contraception ("antibaby-pill", hormonal plaster, NuvaRing®, Implanon®, hormonal depot injections, contraceptive coil), the tubal ligature (female sterilization). Alternatively, the female of child bearing potential is sexually abstinent. |
| Country | Name | City | State |
|---|---|---|---|
| Germany | University Hospital Cologne | Cologne | Northern Westfalia |
| Lead Sponsor | Collaborator |
|---|---|
| University Hospital of Cologne | Boston Scientific Corporation, Hannover Medical School, Schoen Klinik Vogtareuth, University Hospital Berlin Charite, University Hospital of Duesseldorf, University Hospital of Freiburg, University Hospital of Kiel, University Hospital of Munich, University Hospital of Tübingen, University Hospital of Würzburg, University Hospital Schleswig Holstein Campus Luebeck, University Hsopital of Magdeburg |
Germany,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) | Difference in CPCHILD before and 36 months on DBS (response=improvement > 10%) | CPCHILD 12 months after DBS | |
| Secondary | Burke-Fahn-Marsden-Dystonia Rating Scale movement and disability | Assessment of the severity of dystonia | 0, 6, 12, 24 and 36 months after DBS | |
| Secondary | Dyskinesia Impairment Scale | Assessment of the severity of chorea and dystonia | 0, 12, 24 and 36 months | |
| Secondary | Tardieu Scale | Assessment of the severity of spasticity | 0 and 12 months after DBS | |
| Secondary | Frenchay Dysarthria Assessment | Assessment of speech and swallowing | 0, 12, 24 and 36 months after DBS | |
| Secondary | SF-36 | Assessment of Quality of life | 0, 6, 12, 24 and 36 months after DBS | |
| Secondary | Strengths and Difficulties Questionnaire | Assessment of mood and attention | 0, 6, 12, 24 and 36 months after DBS | |
| Secondary | Snijders-Oomen-Non-Verbal-Intelligence Test (SON-R) | Assessment of Cognition | 0 and 12 months | |
| Secondary | Attentional Network Test (ANT) | Assessment of Attention | 0 and 12 months after DBS | |
| Secondary | Non-Verbal-Learning Test (NVLT) | Assessment of cognition | 0 and 12 months after DBS | |
| Secondary | Wong Baker Faces | Assessment of pain | 0, 6, 12, 24 and 36 months after DBS | |
| Secondary | Family Scale (FaBel) | Assessment of the burden for caregivers | 0, 6, 12, 24 and 36 months after DBS | |
| Secondary | CPCHILD | Assessment of quality of life | 0, 6, 24 and 36 months after DBS | |
| Secondary | Canadian Occupational Performance Measure (COPM) | Assessment of activities of daily living | COPM 0 and 12 months after DBS | |
| Secondary | Gross Motor Function Measure (GMFM-66) | Assessment of physical disability | GMFM-66 0 and 12 months after DBS | |
| Secondary | Gross Motor Function Classification System (GMFCS) | Degree of physical impairment | GMFCS 0, 12, 24 and 36 months after DBS |