Intrathecal Baclofen and Pediatric Dystonia
ConditionDystonic Cerebral Palsy
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
AgeUp to 17
SponsorBaylor College of Medicine
About this trial
The goal of this clinical trial is to better understand the effects of intrathecal baclofen (ITB) on children with dystonic cerebral palsy (CP).
The main questions this study aims to answer are:
(1) Determine if ITB reduces dystonia while identifying other potential benefits, (2) Identify the characteristics of children with the best response to ITB (3) Develop a holistically representative composite outcome measure for dystonic CP.
This study will evaluate patient improvement by using a standardized titration, or medication management, protocol to gradually increase the childs ITB dosages over a 12-month period until they achieve maximum benefit with minimal to no side effects. This titration protocol mimics what is currently done through routine care but with more precision. This study will also directly measure the global effects of ITB, taking into account spasticity, known dystonia triggers (e.g. pain), and patterns of CNS injury that cause dystonia.
Participants will:
1. Complete a total of 4 additional clinic visits outside usual care. These appointments will be with physical and occupational therapists as well as the study PI to complete evaluations for dystonia, spasticity, and function. 2. Complete several questionnaires at these visits. The total duration of the study for an individual child will be 12 months.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Child must be large enough to have an ITB pump implanted (typically ~18 kg/4 years old)
They have a diagnosis of CP
Presence of dystonia verified with a documented Hypertonia Assessment Tool (HAT) and they must have a Barry-Albright Dystonia Scale (BADS) score >15
They have been identified by a physician for ITB treatment for tone management and the family/child have agreed to proceed with implantation; note: an ITB trial dose is not necessary to qualify
Disqualifiers
Emergent need for ITB such as status dystonicus or paroxysmal sympathetic hyperactivity
Botulinum injections within 3 months or phenol injections within 6 months of enrollment
Foster care placement or incarceration
Trial design
Design model
Single group
Treatments tested in this trial
Titration protocol
Other interventionA standardized titration protocol for ITB will be used and the ITB dose will be increased to the next step if 1) there is persistent hypertonia identified on the dystonia screener or ASAS total spasticity, or 2) there is room for improvement on the D-FIS, and 3) there are no detrimental side effects (e.g., worsening head control). This will be continued until 1) dystonia and spasticity have been eliminated or no longer deemed as functionally impairing by the medical team, 2) side effects prevent further titration, or 3) a maximum dose of 2000 mcg/day has been reached. Once steady state is reached, this new dose becomes the maintenance dose for the rest of the trial. If there are detrimental side effects, the child will be seen weekly to confirm resolution of side effects. If side effects persist, the child will be reduced at each visit to the previous step. Once side effects are controlled, this new dose becomes the child's maintenance dose for the rest of the trial.
Treatment groups
65 Participants
are divided into 1 treatment groupGroup A: Titration protocolOther 1 intervention
Trial outcomes
Primary outcomes
1
Barry-Albright Dystonia Scale (BADS)
The BADS was created based on the Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS), a tool originally designed to detect cervical dystonia in adults.The BADS tailors this scale for detecting dystonia in CP and acquired brain injury in individuals with limitations in cognition and physical ability. Scoring focuses on posturing and involuntary dystonic movements to detect potential changes in dystonia that affect ease of care and comfort. It does not assess functional tasks. It has moderate internal consistency and inter-rater reliability. It has effectively detected change with ITB. The BADS was also found to have the greatest clinical utility for children with dystonic CP
Time frame
Baseline (pre-ITB initiation) , 3 months, 6 months, 12 months post ITB initiationSecondary outcomes
1
Dyskinesia Impairment Scale-II (DIS-II)
The DIS-II is one of the newest scales to measure dystonia and choreoathetosis at rest and during activity. It is unique in separating choreoathetosis from dystonia in this population. It has moderate internal consistency and fair interrater reliability. We include this measure because it was a key measure in the IDYS trial by Bonouvrie LA et al., the single RCT trial, thus permitting direct comparison across our studies.
Time frame
Baseline (pre-ITB initiation), 3 months, 6 months, 12 months post ITB initiation2
Australian Spasticity Assessment Scale (ASAS) and Composite Measure
The ASAS combines the best aspects of the MTS with the simplicity of the MAS's scoring system. It has clear instructions on how the limb must be tested while supine to increase reproducibility. Its interrater reliability is very high with a weighted kappa of 0.87, making it ideal for a large study with multiple time points. We will generate a composite score (as well have individual scores) for key muscle groups.
Time frame
Baseline (pre-ITB initiation), 3 months, 6 months, 12 months post ITB initiation3
Modified Ashworth Scale (MAS)
While our study relies strongly on the ASAS, we will also measure MAS so our data can be compared across studies.
Time frame
Baseline (pre-ITB initiation), 3 months, 6 months, 12 months post ITB initiation4
Gross Motor Function Measure (GMFM)
The GMFM is an assessment tool to measure changes in gross motor function with interventions in children with CP. Given that children enrolled in this study will fall in the GMFCS IV-V category, we will not score section E: walking, running, and jumping. As a widely used clinical and research tool, it has an excellent test-retest reliability and excellent inter- and intrarater reliability. The minimal clinically important difference (MCID) is \~0.7-1.2 points in ambulatory children with CP but unfortunately a level has not been determined for non-ambulatory children.
Time frame
Baseline (pre-ITB initiation) , 3 months, 6 months, 12 months post ITB initiationSponsors and contacts
Click on the lead sponsor to view all of their trials.
Baylor College of Medicine
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Collaborator
Source ClinicalTrials.gov