Dystonic Cerebral PalsyPREDICT-ITBNCT06606574
Intrathecal Baclofen and Pediatric Dystonia
A study testing ITB Titration Protocol, Ventral Dorsal Rhizotomy and Deep Brain Stimulation in people with Dystonic Cerebral Palsy, run by Baylor College of Medicine at 1 site in United States.
Trial summary
The goal of this study is to better understand outcomes in children with dystonic cerebral palsy (CP) receiving intrathecal baclofen (ITB), ventral dorsal rhizotomy (VDR), or deep brain stimulation (DBS) as part of standard clinical care. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Dystonic Cerebral Palsy
- What is being tested?
- ITB Titration Protocol, Ventral Dorsal Rhizotomy and Deep Brain Stimulation
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Nov 2024to 31 Aug 2029
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Child must be large enough to have an ITB pump implanted (typically ~18 kg/4 years old)
- Child must be less than 21 years of age
- 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 >14
- 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.
- Child and family are willing to participate in full schedule of formal assessments, spanning pre-implantation, every 2 weeks until ideal intrathecal baclofen pump dosing is reached, and follow-up assessments
Show all 17Show fewer
- Child must be less than 21 years of age
- 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 >14
- Currently less than 12 months post ITB pump implant for tone management.
- Child and family are willing to participate in the 12-month outcome measure assessment.
- Child must be less than 21 years of age
- They have a diagnosis of CP
- Presence of dystonia verified with a documented Hypertonia Assessment Tool (HAT)
- They have been identified by a physician as a candidate for VDR for tone management and the family/child have agreed to proceed with surgery.
- Child must be less than 21 years of age
- They have a diagnosis of CP and have been identified by a physician as a candidate for DBS
Who cannot join
- Emergent need for ITB such as status dystonicus or paroxysmal sympathetic hyperactivity
- Botulinum injections within 3 months or phenol injections within 6 months
- Foster care placement or incarceration
- Family not willing to participate in schedule of assessments
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Texas Childrens Hospital
Houston, Texas, 77030
Principal Investigator, MD, PhD, 832-824-4945, spthomas@texaschildrens.org
About this study
Official title: Predicting Response In Children With Dystonic Cerebral Palsy to Intrathecal Baclofen (Predict-ITB), Ventral Dorsal Rhizotomy (Predict-VDR), and Deep Brain Stimulation (Predict-DBS)
The goal of this study is to better understand outcomes in children with dystonic cerebral palsy (CP) receiving intrathecal baclofen (ITB), ventral dorsal rhizotomy (VDR), or deep brain stimulation (DBS) as part of standard clinical care. The decision to proceed with ITB, VDR, or DBS is made independently of research participation.
The study aims to:
Read more
- Evaluate changes in dystonia and other clinically important outcomes;
- Identify characteristics associated with treatment response; and
- Develop a multidimensional outcome measure for children with dystonic CP.
Participants receiving ITB will follow a study-specific standardized titration protocol designed to identify a dose that provides maximum benefit with minimal side effects. Participants in all three groups will complete research assessments and questionnaires at protocol-specified timepoints to evaluate dystonia, spasticity, function, pain, stress, and quality of life.
Read the detailed description
Cerebral palsy (CP) is the most common cause of physical disability in childhood and affects ~1 in 323 in the US per the CDC. The lifetime medical costs for an individual with CP was expected to be ~$1.4 million (inflation adjusted). An effective way to improve quality of life and decrease disease burden, both in childhood and adulthood, is to significantly reduce or eliminate abnormal tone. As muscle tone subsides, it is easier to move the body both passively and actively. A reduction in tone can improve quality of life, reduce pain, ease the burden of daily care and performance of day-to-day activities, and reduce healthcare costs. Intrathecal baclofen ( ITB) is used widely to manage dystonia in CP even though the data on exact efficacy is mixed. Therefore, the goal of this study, is to understand the effects of ITB on a heterogenous population of children with dystonic CP.
The goal of the proposed research is to complete a longitudinal prospective cohort study investigating the effects of intrathecal baclofen (ITB) on children with dystonic cerebral palsy (CP). This study builds on new preliminary data and capitalizes on the expertise of an established transdisciplinary team, a high-resource academic clinical environment, and a diverse Parent-Patient Council. The study's findings are likely to have high impact to improve future treatment of children with dystonic CP, including identifying key patient characteristics associated with more or less positive outcomes and developing a sensitive, efficient multidimensional composite measure to assess treatment responsiveness. Specifically, this protocol aims to determine the 12-month effects of ITB in a cohort of 65 clinically representative, ITB naïve, children with dystonic CP using a standardized titration protocol. Additionally, this protocol aims to complete a detailed characterization of brain malformation and injury patterns in children with dystonic CP and explore potential differences in outcome patterns as related to patient characteristics and use findings to guide development of a sensitive multidimensional or composite measure that captures clinically important areas of change for the child and family.
- Conditions
- Dystonic Cerebral Palsy
- Being tested
- ITB Titration Protocol, Ventral Dorsal Rhizotomy, Deep Brain Stimulation, Research Assessments and Study Monitoring
- Keywords
- Intrathecal baclofen, Spasticity, Dystonia, Cerebral Palsy, Deep Brain Stimulation, Ventral Dorsal Rhizotomy
- Medical subject headings
- Muscle Spasticity, Dystonia, Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ITB Titration ProtocolDrug
Also called Intrathecal Baclofen
Intrathecal baclofen (ITB) is provided as part of standard clinical care, and the decision to proceed with ITB is made independently of research participation. Enrolled participants follow a study-specific standardized titration protocol. ITB dose is increased according to prespecified steps based on persistent hypertonia and/or potential improvement on the D-FIS, provided there are no detrimental side effects. Titration continues until dystonia and spasticity are eliminated or no longer deemed functionally impairing by the medical team, side effects limit further titration, or a maximum dose of 2,000 mcg/day is reached. If detrimental side effects occur, the dose is reduced to the previous step as needed until side effects are controlled. The final tolerated dose becomes the maintenance dose for the remainder of the study.
Ventral Dorsal RhizotomyProcedure or surgery
Ventral dorsal rhizotomy (VDR) is performed as part of standard clinical care. The decision to proceed with VDR is made independently of research participation. The study does not assign participants to VDR or modify the VDR procedure or clinical follow-up.
Deep Brain StimulationProcedure or surgery
Deep brain stimulation (DBS) is performed as part of standard clinical care. The decision to proceed with DBS is made independently of research participation. The study does not assign participants to DBS or modify the DBS procedure or clinical follow-up.
Research Assessments and Study MonitoringOther
Participants complete protocol-specified clinician or therapy assessments, patient-reported outcome measures, and study-monitoring activities at baseline and 3, 6, and 12 months. For participants receiving ITB, selected assessments and safety-monitoring activities also occur during study-specific titration visits.
| Study group | Role | What they receive |
|---|---|---|
| Intrathecal Baclofen | Experimental | Participants in this arm receive intrathecal baclofen (ITB) as part of standard clinical care. The decision to proceed with ITB is made independently of research participation. Enrolled participants follow a study-specific ITB titration protocol and complete research assessments and study monitoring at protocol-specified timepoints. Receives: Drug: ITB Titration Protocol; Other: Research Assessments and Study Monitoring |
| Ventral Dorsal Rhizotomy | Other | Participants in this arm undergo ventral dorsal rhizotomy (VDR) as part of standard clinical care. The decision to proceed with VDR is made independently of research participation. The study does not assign participants to VDR or modify the VDR procedure or clinical follow-up. Enrolled participants complete research assessments and study monitoring at baseline and 3, 6, and 12 months. Receives: Procedure: Ventral Dorsal Rhizotomy; Other: Research Assessments and Study Monitoring |
| Deep Brian Stimulation | Other | Participants in this arm undergo deep brain stimulation (DBS) as part of standard clinical care. The decision to proceed with DBS is made independently of research participation. The study does not assign participants to DBS or modify the DBS procedure or clinical follow-up. Enrolled participants complete research assessments and study monitoring at baseline and 3, 6, and 12 months. Receives: Procedure: Deep Brain Stimulation; Other: Research Assessments and Study Monitoring |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Secondary outcomes (10)
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.
Measured: Baseline (pre-intervention), 3 months, 6 months, and 12 months
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.
Measured: Baseline (pre-intervention), 3 months, 6 months, and 12 months
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.
Measured: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Gross Motor Family Report (GMFR)
The Gross Motor Function Report (GMFR) is a patient- or caregiver-reported measure that evaluates gross motor abilities and functional mobility in everyday activities. The GMFR assesses performance across a range of movement domains, including sitting, standing, transfers, walking, running, and participation in daily physical activities. Higher scores indicate better gross motor function and mobility.
Measured: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Box and Blocks Test
This test is a quick, simple way to assess manual dexterity. This test cannot be used in a child with severe upper extremity impairment or severe cognitive impairment, therefore, not all participants in this study will be able to complete this task. A child is asked to move as many blocks as possible from one side of a divided box to the other in 60 seconds. It has excellent interrater reliability and MCID of 5.29- 6.46 seconds.
Measured: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Performance Quality Rating Scale (PQRS)
The PQRS is an observational measure of performance quality of a parent/patient-selected meaningful activity. Occupational therapists use a video recording of a child performing the task of interest and then rate the quality. It has high interrater reliability from 0.83-0.93 and test-retest reliability \>0.80.
Measured: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Canadian Occupational Performance Measure (COPM)
The COPM focuses on occupational performance in all areas of life, including self-care, leisure, and productivity. It consists of a semi- structured interview to determine areas of concern for the parent and child. Together with an occupational therapist, they score performance on the particular concerns at the set timepoints in the study. We will have families and children set 2-3 goals that are relevant to ITB treatment. The COPM is an excellent tool for this study as it is very sensitive to change and has an established MCID of 2 points.
Measured: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Caregiver Priorities & Child Health Index of Life with Disabilities (CPCHILD©)
The CPCHILD© measures a child with severe disability's overall health, comfort, function, and QoL in relationship to their medical conditions. It has 37 questions answered by the child or parent about activities of daily living, positioning, transferring and mobility, comfort and emotions, communications and social interaction, health, and overall QoL. It was initially validated in children with CP and traumatic brain injuries. Prospective longitudinal cohort studies are underway to assess the measure's responsiveness/sensitivity to change.
Measured: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Patient-Reported Outcomes Measurement Information System (PROMIS®) Questionnaires
NIH's Office of Strategic Coordination - Common Fund's PROMIS® developed a validated item bank of patient-reported outcomes for clinical research and practice. The PROMIS offers many short form questionnaires that are simple and quick for parents and children to complete. We will use the following to assess pain, anxiety, depression, and stress:
(1a) PROMIS® Pediatric Item Bank v1.0 - Pain Behavior 8a
(1b) PROMIS® Parent Proxy Bank v1.0 - Pain Behavior 8a. (2a) PROMIS® Pediatric Item Bank v2.0 - Pediatric Anxiety - Short Form 8a (2b) Parent Proxy Bank v2.0 - Parent Proxy Anxiety - Short Form 8a (3a) PROMIS® Pediatric Item Bank v2.0 - Pediatric Depressive Symptoms - Short Form 8a (3b) Parent Proxy Bank v2.0 - Parent Proxy Depressive Symptoms - Short Form 6a. (4) PROMIS® Parent Proxy Bank v1.0 - Psychological Stress Experiences - Short Form 4a.
(5) PROMIS® Parent Proxy Bank v1.0 - Physical Stress
Measured: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Goal Attainment Scale (GAS)
The GAS is a unique outcome measurement tool with some controversy about its standardization. It selects a single important goal to be tracked before, during, and after an intervention. GAS was created to account for the heterogeneity of the rehabilitation population. In addition to choosing the goal(s), the child and their family, with guidance from their care team, choose how to define a successful outcome. GAS is tailored to each child and has been shown to be as sensitive to change over time as other standard outcome measurement tools, including the GMFM (discussed above) and the Pediatric Evaluation of Disability Inventory. The single RCT IDYS trial used the GAS as their 1o outcome measure. By including this, we can directly compare results.
Measured: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2024
- Study start1 Nov 2024
- Primary completion30 Jun 2029 (estimated)
- Study completion31 Aug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Dystonic Cerebral PalsyStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 65 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Responsible party
- Principal investigator, Sruthi P. Thomas, MD, PhD, Baylor College of Medicine
- Investigators
- Sruthi P Thomas, MD, PhD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06606574
- Sponsor's protocol ID
- H-54449
- Other IDs
- R01HD112563
- Acronym
- PREDICT-ITB
- Status last verified
- Nov 2026
- First submitted
- 18 Sep 2024
- Last updated
- 5 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Individual Participant data and final study results may be reviewed with the participant(s) and their families. Data de-identified data may be summarized into graphs showing the changes in all of the measures at 0, 3, 6, and 12 months.
Publications
Thomas SP, Ramey S, Conaway M, Kralik S, Arp R, Raskin JS, Gadgil N, Marupudi N, Fehlings D. Investigating the effects of intrathecal baclofen and ventral-dorsal rhizotomy on dystonia in children with cerebral palsy (PREDICT-Dystonia): protocol for a prospective observational clinical trial. BMJ Open. 2026 Oct 6;16(10):e123926. doi: 10.1136/bmjopen-2026-123926.
PubMed 42838585Thomas SP, Fehlings D, Ramey S, Conaway M, Kralik S, Raskin J. PREDICT-ITB: Predicting response in children with dystonic CP to ITB - study protocol. medRxiv [Preprint]. 2025 Jul 8:2025.07.08.25331080. doi: 10.1101/2025.07.08.25331080.
PubMed 40672491
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Predicting Response In Children With Dystonic Cerebral Palsy to Intrathecal Baclofen (Predict-ITB), Ventral Dorsal Rhizotomy (Predict-VDR), and Deep Brain Stimulation (Predict-DBS)
- ClinicalTrials.gov ID
- NCT06606574
- Acronym / sponsor ID
- PREDICT-ITB / H-54449
- Conditions
- Dystonic Cerebral Palsy
- Keywords
- Intrathecal baclofen, Spasticity, Dystonia, Cerebral Palsy, Deep Brain Stimulation, Ventral Dorsal Rhizotomy
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 65 participants (estimated)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Study start
- 1 Nov 2024 (actual)
- Primary completion
- 30 Jun 2029 (estimated)
- Study completion
- 31 Aug 2029 (estimated)
- First posted
- 23 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this study is to better understand outcomes in children with dystonic cerebral palsy (CP) receiving intrathecal baclofen (ITB), ventral dorsal rhizotomy (VDR), or deep brain stimulation (DBS) as part of standard clinical care. The decision to proceed with ITB, VDR, or DBS is made independently of research participation.
The study aims to:
- Evaluate changes in dystonia and other clinically important outcomes;
- Identify characteristics associated with treatment response; and
- Develop a multidimensional outcome measure for children with dystonic CP.
Participants receiving ITB will follow a study-specific standardized titration protocol designed to identify a dose that provides maximum benefit with minimal side effects. Participants in all three groups will complete research assessments and questionnaires at protocol-specified timepoints to evaluate dystonia, spasticity, function, pain, stress, and quality of life.
Detailed description
Cerebral palsy (CP) is the most common cause of physical disability in childhood and affects ~1 in 323 in the US per the CDC. The lifetime medical costs for an individual with CP was expected to be ~$1.4 million (inflation adjusted). An effective way to improve quality of life and decrease disease burden, both in childhood and adulthood, is to significantly reduce or eliminate abnormal tone. As muscle tone subsides, it is easier to move the body both passively and actively. A reduction in tone can improve quality of life, reduce pain, ease the burden of daily care and performance of day-to-day activities, and reduce healthcare costs. Intrathecal baclofen ( ITB) is used widely to manage dystonia in CP even though the data on exact efficacy is mixed. Therefore, the goal of this study, is to understand the effects of ITB on a heterogenous population of children with dystonic CP.
The goal of the proposed research is to complete a longitudinal prospective cohort study investigating the effects of intrathecal baclofen (ITB) on children with dystonic cerebral palsy (CP). This study builds on new preliminary data and capitalizes on the expertise of an established transdisciplinary team, a high-resource academic clinical environment, and a diverse Parent-Patient Council. The study's findings are likely to have high impact to improve future treatment of children with dystonic CP, including identifying key patient characteristics associated with more or less positive outcomes and developing a sensitive, efficient multidimensional composite measure to assess treatment responsiveness. Specifically, this protocol aims to determine the 12-month effects of ITB in a cohort of 65 clinically representative, ITB naïve, children with dystonic CP using a standardized titration protocol. Additionally, this protocol aims to complete a detailed characterization of brain malformation and injury patterns in children with dystonic CP and explore potential differences in outcome patterns as related to patient characteristics and use findings to guide development of a sensitive multidimensional or composite measure that captures clinically important areas of change for the child and family.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Intrathecal Baclofen | Experimental | Drug: ITB Titration Protocol, Other: Research Assessments and Study MonitoringParticipants in this arm receive intrathecal baclofen (ITB) as part of standard clinical care. The decision to proceed with ITB is made independently of research participation. Enrolled participants follow a study-specific ITB titration protocol and complete research assessments and study monitoring at protocol-specified timepoints. |
| Ventral Dorsal Rhizotomy | Other | Procedure: Ventral Dorsal Rhizotomy, Other: Research Assessments and Study MonitoringParticipants in this arm undergo ventral dorsal rhizotomy (VDR) as part of standard clinical care. The decision to proceed with VDR is made independently of research participation. The study does not assign participants to VDR or modify the VDR procedure or clinical follow-up. Enrolled participants complete research assessments and study monitoring at baseline and 3, 6, and 12 months. |
| Deep Brian Stimulation | Other | Procedure: Deep Brain Stimulation, Other: Research Assessments and Study MonitoringParticipants in this arm undergo deep brain stimulation (DBS) as part of standard clinical care. The decision to proceed with DBS is made independently of research participation. The study does not assign participants to DBS or modify the DBS procedure or clinical follow-up. Enrolled participants complete research assessments and study monitoring at baseline and 3, 6, and 12 months. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (17 listed)
- Child must be large enough to have an ITB pump implanted (typically ~18 kg/4 years old)
- Child must be less than 21 years of age
- 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 >14
- 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.
Key exclusion criteria (4 listed)
- Emergent need for ITB such as status dystonicus or paroxysmal sympathetic hyperactivity
- Botulinum injections within 3 months or phenol injections within 6 months
- Foster care placement or incarceration
- Family not willing to participate in schedule of assessments
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ITB Titration ProtocolAlso: Intrathecal BaclofenIntrathecal baclofen (ITB) is provided as part of standard clinical care, and the decision to proceed with ITB is made independently of research participation. Enrolled participants follow a study-specific standardized titration protocol. ITB dose is increased according to prespecified steps based on persistent hypertonia and/or potential improvement on the D-FIS, provided there are no detrimental side effects. Titration continues until dystonia and spasticity are eliminated or no longer deemed functionally impairing by the medical team, side effects limit further titration, or a maximum dose of 2,000 mcg/day is reached. If detrimental side effects occur, the dose is reduced to the previous step as needed until side effects are controlled. The final tolerated dose becomes the maintenance dose for the remainder of the study. | Drug | Intrathecal Baclofen |
| Ventral Dorsal RhizotomyVentral dorsal rhizotomy (VDR) is performed as part of standard clinical care. The decision to proceed with VDR is made independently of research participation. The study does not assign participants to VDR or modify the VDR procedure or clinical follow-up. | Procedure or surgery | Ventral Dorsal Rhizotomy |
| Deep Brain StimulationDeep brain stimulation (DBS) is performed as part of standard clinical care. The decision to proceed with DBS is made independently of research participation. The study does not assign participants to DBS or modify the DBS procedure or clinical follow-up. | Procedure or surgery | Deep Brian Stimulation |
| Research Assessments and Study MonitoringParticipants complete protocol-specified clinician or therapy assessments, patient-reported outcome measures, and study-monitoring activities at baseline and 3, 6, and 12 months. For participants receiving ITB, selected assessments and safety-monitoring activities also occur during study-specific titration visits. | Other | Deep Brian Stimulation, Intrathecal Baclofen, Ventral Dorsal Rhizotomy |
Primary outcome
Barry-Albright Dystonia Scale (BADS)
Time frame: Baseline (pre-intervention), 3 months, 6 months, and 12 months
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
10 secondary outcomes
Dyskinesia Impairment Scale-II (DIS-II)
Time frame: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Australian Spasticity Assessment Scale (ASAS) and Composite Measure
Time frame: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Modified Ashworth Scale (MAS)
Time frame: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Gross Motor Family Report (GMFR)
Time frame: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Box and Blocks Test
Time frame: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Performance Quality Rating Scale (PQRS)
Time frame: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Canadian Occupational Performance Measure (COPM)
Time frame: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Caregiver Priorities & Child Health Index of Life with Disabilities (CPCHILD©)
Time frame: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Patient-Reported Outcomes Measurement Information System (PROMIS®) Questionnaires
Time frame: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Goal Attainment Scale (GAS)
Time frame: Baseline (pre-intervention), 3 months, 6 months, and 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- Derived from this study: PubMed 42838585Thomas SP, Ramey S, Conaway M, Kralik S, Arp R, Raskin JS, Gadgil N, Marupudi N, Fehlings D. Investigating the effects of intrathecal baclofen and ventral-dorsal rhizotomy on dystonia in children with cerebral palsy (PREDICT-Dystonia): protocol for a prospective observational clinical trial. BMJ Open. 2026 Oct 6;16(10):e123926. doi: 10.1136/bmjopen-2026-123926.
- Derived from this study: PubMed 40672491Thomas SP, Fehlings D, Ramey S, Conaway M, Kralik S, Raskin J. PREDICT-ITB: Predicting response in children with dystonic CP to ITB - study protocol. medRxiv [Preprint]. 2025 Jul 8:2025.07.08.25331080. doi: 10.1101/2025.07.08.25331080.
- Individual participant dataSharing planned: Undecided. Individual Participant data and final study results may be reviewed with the participant(s) and their families. Data de-identified data may be summarized into graphs showing the changes in all of the measures at 0, 3, 6, and 12 months.
Related trials for Dystonic Cerebral Palsy
RecruitingObservational study
Outcomes of Neurosurgical Tone Management
Spastic/Dystonic Quadriplegia, Cerebral Palsy (CP)
RecruitingPhase 1
Pharmacogenomic Contributions to Trihexyphenidyl Biotransformation and Response in Children With Dystonic Cerebral Palsy
Pediatric Disorder, Genetic Predisposition
Recruiting
Focused Ultrasound Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects
Cerebral Palsy, Dyskinetic, Secondary Dystonia
Recruiting
Cerebellar Deep Brain Stimulation for Movement Disorders in Cerebral Palsy in Children and Young Adults
Dyskinetic Cerebral Palsy, Dystonic Cerebral Palsy
Not yet recruitingObservational study
Enhancing Sensorimotor Processing in Children With Dystonia
Dystonia, Dystonic Cerebral Palsy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Sruthi P Thomas, MD, PhD principal investigator
Baylor College of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing ITB Titration Protocol, Ventral Dorsal Rhizotomy and Deep Brain Stimulation in people with Dystonic Cerebral Palsy, run by Baylor College of Medicine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sruthi Thomas, MD, PhD (832-824-4945, spthomas@texaschildrens.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Barry-Albright Dystonia Scale (BADS) (time frame: Baseline (pre-intervention), 3 months, 6 months, and 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06606574, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2026.