Cerebral PalsyNCT03789786
Long-term Outcomes of Selective Dorsal Rhizotomy Among Individuals With Cerebral Palsy
An observational study of Cerebral Palsy, run by Gillette Children's Specialty Healthcare at 3 sites in United States.
Trial summary
Spasticity affects up to 80% of individuals diagnosed with cerebral palsy. Selective dorsal rhizotomy (SDR) is a surgical method used by some hospitals to permanently reduce spasticity in order to prevent further morbidities. Read more about this study
- Who is it for?
- Ages 21 to 45 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- Gait and Motion Analysis, Gross Motor Function Measure (GMFM) and Surveys
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 78 participants
- Where
- 3 sitesUnited States
- Study dates
- 1 Nov 2018to 31 Jan 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
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 21 to 45 years
- All sexes
- No healthy volunteers
- Adult
Study population: Former/current patients at Gillette Children's Specialty Healthcare, Shriners Hospitals for Children - Salt Lake City, or Shriners Hospitals for Children - Spokane
Who may be able to join
- Able to speak and read English
- Diagnosed with bilateral cerebral palsy (i.e. no hemiplegics)
- Minimum age of 21 years presently
- Had a baseline gait and motion analysis
- Controls (-SDR):
- No SDR
Show all 14Show fewer
- No history of intrathecal baclofen (ITB) pump implantation for > 1 year
- No ITB pump at time of long-term follow-up (explant > or = 6 months)
- No history of oral baclofen for > 1 year
- No oral baclofen use at time of long-term follow-up
- No more than 10 sessions on botulinum toxin, phenol, or alcohol injection
- Cases (+SDR):
- History of SDR > 5 years ago
- Had a baseline gait analysis < or = 18 months before SDR
Who cannot join
- none
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101
Shriners Hospitals for Children - Salt Lake City
Salt Lake City, Utah, 84103
Shriners Hospitals for Childrens - Spokane
Spokane, Washington, 99204
About this study
Official title: Long-term Outcomes of Selective Dorsal Rhizotomy Among Individuals With Cerebral Palsy Compared to a Matched Control Group
Spasticity affects up to 80% of individuals diagnosed with cerebral palsy. Selective dorsal rhizotomy (SDR) is a surgical method used by some hospitals to permanently reduce spasticity in order to prevent further morbidities. Better understanding of the long-term outcomes of SDR is essential for clinicians and families. The results of this study will have direct clinical impact by equipping providers with the necessary information to counsel families during medical decision making.
Read the detailed description
Spasticity affects up to 80% of individuals diagnosed with cerebral palsy. Excessive spasticity is thought to be uncomfortable, reduce function, cause gait deviations (e.g. equinus), and contribute toward musculoskeletal deformity and an elevated energy cost while walking. As such, SDR is a surgical method used by some hospitals to permanently reduce spasticity in order to prevent the aforementioned morbidities.
Treatment philosophies differ widely in regards to how aggressively to manage spasticity. Some centers (e.g. Gillette) aggressively treat spasticity early in life through a variety of measures such as SDR, intrathecal baclofen pumps, and botulinum toxin injections. Other centers (e.g. Shriners Hospitals for Children - Salt Lake City and Spokane) offer little in the way of spasticity reduction treatments.
There are several compelling reasons to conduct the proposed research study. First, emerging evidence suggests that the elimination of spasticity during childhood via SDR does not prevent contractures and only partially explains poor gross motor function, both previously thought to be clear outcomes of the surgery. Additionally, many of the longitudinal cohort studies that examined SDR outcomes have shown many outcome measures peak 1-3 years after surgery, and then decline toward baseline (i.e. pre-SDR) levels. Lastly, the quality of the SDR outcome literature is poor. Rarely are outcomes looked at in context of a proper control group. Either a control group is absent or comprised of typically developing children. This limits our ability to understand how patients with cerebral palsy may age without undergoing an SDR.
Better understanding of the long-term outcomes of SDR is essential for clinicians and families. The surgery, in general, is costly to families (time, expense, risk, etc.) and clinicians should have every confidence in the intended outcomes for any intervention.
- Conditions
- Cerebral Palsy
- Being tested
- Gait and Motion Analysis, Gross Motor Function Measure (GMFM), Surveys
- Keywords
- Selective Dorsal Rhizotomy, Outcomes
- Medical subject headings
- Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gait and Motion AnalysisDiagnostic test
Gait and motion analysis is comprised of 3-dimensional kinematics and kinetics, electromyography, energy expenditure, and physical exam (range of motion, strength, spasticity, etc.)
Gross Motor Function Measure (GMFM)Diagnostic test
Gross Motor Function Measure (GMFM) is an assessment used to evaluate gross motor function over time in individuals with cerebral palsy. The assessment is comprised of movement activities like standing, walking, running, and jumping
SurveysBehavioural
Six surveys are used to assess function, activity, participation, pain, quality of life, and treatment history
| Study group | Role | What they receive |
|---|---|---|
| Cases (+SDR) | Patients with cerebral palsy that underwent an SDR Receives: Diagnostic Test: Gait and Motion Analysis; Diagnostic Test: Gross Motor Function Measure (GMFM); Behavioral: Surveys | |
| Controls (-SDR) | Matched patients with cerebral palsy but did not undergo an SDR Receives: Diagnostic Test: Gait and Motion Analysis; Diagnostic Test: Gross Motor Function Measure (GMFM); Behavioral: Surveys |
What is being measured
The primary outcome is the main question the study is designed to answer.
Three-dimensional gait and motion analysis
Compare three-dimensional gait kinematics and kinetics across groups
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Energy expenditure
Compare energy expenditure across groups
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Spasticity
Compare spasticity, measured by Modified Ashworth Score (0 no increase in tone - 4 rigid in flexion or extension), across groups
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Passive range of motion
Compare passive range of motion, measured by lower extremity physical exam, across groups
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Selective motor control
Compare selective motor control (0 patterned movement - 2 complete isolated movement) across groups
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Lower extremity strength
Compare lower extremity strength, measured by the manual muscle test, across groups
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Gross Motor Function Measure (GMFM-66)
Assess function using portions of the GMFM-66 (0 low function - 100 high function) and compare across groups
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Gillette Functional Assessment Questionnaire (Gillette FAQ)
Assess function and activity using the Gillette FAQ (self-reported survey, 0 low function - 10 high function) and compare across groups
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Functional Mobility Scale (FMS)
Assess function and activity using the FMS (self-reported survey, 1 uses wheelchair - 6 independent) and compare across groups
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Participation Enfranchisement survey
Assess participation using the Participation Enfranchisement survey (self-reported survey, true/false) and compare across groups
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Diener Satisfaction with Life Scale
Assess satisfaction using the Diener Satisfaction with Life Scale (self-reported survey, 5 dissatisfied- 35 satisfied) and compare across groups
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
World Health Organization (WHO) Quality of Life Scale
Assess satisfaction using the WHO Quality of Life Scale (self-reported survey, 0 low quality of life - 100 high quality of life) and compare across groups
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Multiple Sclerosis Spasticity Scale (MSSS-88)
Assess pain using portions of the MSSS-88 (self-reported survey, 21 not at all bothered - 84 extremely bothered) and compare across groups
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Secondary outcomes (3)
Change in gait and motion analysis
Compare change in gait kinematics and kinetics within groups and if the change is similar between groups
Measured: Baseline (qualifying exam for cases and controls) compared to long-term follow-up research visit (on average 10 years post-baseline)
Incidence of prior surgery and anti-spastic treatments
Incidence of prior surgery and anti-spastic treatments
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Cost of prior surgery and anti-spastic treatments
Cost of prior surgery and anti-spastic treatments
Measured: Time of long-term follow-up research visit (on average 10 years post-baseline)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2018
- Study start1 Nov 2018
- Primary completion31 Jan 2020
- Study completion31 Jan 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Case-control
- Time perspective
- Other
- Enrolment
- 78 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gillette Children's Specialty Healthcare
- Sponsor type
- University, hospital or other
- Collaborators
- Shriners Hospitals for Children
- Responsible party
- Principal investigator, Michael H Schwartz, PhD, Gillette Children's Specialty Healthcare
- Investigators
- Michael Schwartz, PhD, Principal investigator, Gillette Children's Specialty Healthcare
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03789786
- Sponsor's protocol ID
- STUDY00002353
- Status last verified
- Oct 2023
- First submitted
- 20 Nov 2018
- Last updated
- 13 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Munger ME, Chen BP, MacWilliams BA, McMulkin ML, Schwartz MH. Comparing the effects of two spasticity management strategies on the long-term outcomes of individuals with bilateral spastic cerebral palsy: a multicentre cohort study protocol. BMJ Open. 2019 Jun 20;9(6):e027486. doi: 10.1136/bmjopen-2018-027486.
PubMed 31227534
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Outcomes of Selective Dorsal Rhizotomy Among Individuals With Cerebral Palsy Compared to a Matched Control Group
- ClinicalTrials.gov ID
- NCT03789786
- Acronym / sponsor ID
- STUDY00002353
- Conditions
- Cerebral Palsy
- Keywords
- Selective Dorsal Rhizotomy, Outcomes
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 78 participants (actual)
- Lead sponsor
- Gillette Children's Specialty HealthcareUniversity, hospital or other
- Collaborators
- Shriners Hospitals for Children
- Study start
- 1 Nov 2018 (actual)
- Primary completion
- 31 Jan 2020 (actual)
- Study completion
- 31 Jan 2020 (actual)
- First posted
- 31 Dec 2018 (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.
Spasticity affects up to 80% of individuals diagnosed with cerebral palsy. Selective dorsal rhizotomy (SDR) is a surgical method used by some hospitals to permanently reduce spasticity in order to prevent further morbidities. Better understanding of the long-term outcomes of SDR is essential for clinicians and families. The results of this study will have direct clinical impact by equipping providers with the necessary information to counsel families during medical decision making.
Detailed description
Spasticity affects up to 80% of individuals diagnosed with cerebral palsy. Excessive spasticity is thought to be uncomfortable, reduce function, cause gait deviations (e.g. equinus), and contribute toward musculoskeletal deformity and an elevated energy cost while walking. As such, SDR is a surgical method used by some hospitals to permanently reduce spasticity in order to prevent the aforementioned morbidities.
Treatment philosophies differ widely in regards to how aggressively to manage spasticity. Some centers (e.g. Gillette) aggressively treat spasticity early in life through a variety of measures such as SDR, intrathecal baclofen pumps, and botulinum toxin injections. Other centers (e.g. Shriners Hospitals for Children - Salt Lake City and Spokane) offer little in the way of spasticity reduction treatments.
There are several compelling reasons to conduct the proposed research study. First, emerging evidence suggests that the elimination of spasticity during childhood via SDR does not prevent contractures and only partially explains poor gross motor function, both previously thought to be clear outcomes of the surgery. Additionally, many of the longitudinal cohort studies that examined SDR outcomes have shown many outcome measures peak 1-3 years after surgery, and then decline toward baseline (i.e. pre-SDR) levels. Lastly, the quality of the SDR outcome literature is poor. Rarely are outcomes looked at in context of a proper control group. Either a control group is absent or comprised of typically developing children. This limits our ability to understand how patients with cerebral palsy may age without undergoing an SDR.
Better understanding of the long-term outcomes of SDR is essential for clinicians and families. The surgery, in general, is costly to families (time, expense, risk, etc.) and clinicians should have every confidence in the intended outcomes for any intervention.
Study design
- Observational model
- Case-control
- Time perspective
- Other
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cases (+SDR) | – | Diagnostic Test: Gait and Motion Analysis, Diagnostic Test: Gross Motor Function Measure (GMFM), Behavioral: SurveysPatients with cerebral palsy that underwent an SDR |
| Controls (-SDR) | – | Diagnostic Test: Gait and Motion Analysis, Diagnostic Test: Gross Motor Function Measure (GMFM), Behavioral: SurveysMatched patients with cerebral palsy but did not undergo an SDR |
Who may qualify
- Ages
- 21 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Former/current patients at Gillette Children's Specialty Healthcare, Shriners Hospitals for Children - Salt Lake City, or Shriners Hospitals for Children - Spokane
Key inclusion criteria (14 listed)
- Able to speak and read English
- Diagnosed with bilateral cerebral palsy (i.e. no hemiplegics)
- Minimum age of 21 years presently
- Had a baseline gait and motion analysis
- No SDR
Key exclusion criteria (1 listed)
- none
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 |
|---|---|---|
| Gait and Motion AnalysisGait and motion analysis is comprised of 3-dimensional kinematics and kinetics, electromyography, energy expenditure, and physical exam (range of motion, strength, spasticity, etc.) | Diagnostic test | Cases (+SDR), Controls (-SDR) |
| Gross Motor Function Measure (GMFM)Gross Motor Function Measure (GMFM) is an assessment used to evaluate gross motor function over time in individuals with cerebral palsy. The assessment is comprised of movement activities like standing, walking, running, and jumping | Diagnostic test | Cases (+SDR), Controls (-SDR) |
| SurveysSix surveys are used to assess function, activity, participation, pain, quality of life, and treatment history | Behavioural | Cases (+SDR), Controls (-SDR) |
Primary outcome
Three-dimensional gait and motion analysis
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Compare three-dimensional gait kinematics and kinetics across groups
Energy expenditure
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Compare energy expenditure across groups
Spasticity
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Compare spasticity, measured by Modified Ashworth Score (0 no increase in tone - 4 rigid in flexion or extension), across groups
Passive range of motion
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Compare passive range of motion, measured by lower extremity physical exam, across groups
Selective motor control
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Compare selective motor control (0 patterned movement - 2 complete isolated movement) across groups
Lower extremity strength
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Compare lower extremity strength, measured by the manual muscle test, across groups
Gross Motor Function Measure (GMFM-66)
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Assess function using portions of the GMFM-66 (0 low function - 100 high function) and compare across groups
Gillette Functional Assessment Questionnaire (Gillette FAQ)
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Assess function and activity using the Gillette FAQ (self-reported survey, 0 low function - 10 high function) and compare across groups
Functional Mobility Scale (FMS)
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Assess function and activity using the FMS (self-reported survey, 1 uses wheelchair - 6 independent) and compare across groups
Participation Enfranchisement survey
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Assess participation using the Participation Enfranchisement survey (self-reported survey, true/false) and compare across groups
Diener Satisfaction with Life Scale
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Assess satisfaction using the Diener Satisfaction with Life Scale (self-reported survey, 5 dissatisfied- 35 satisfied) and compare across groups
World Health Organization (WHO) Quality of Life Scale
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Assess satisfaction using the WHO Quality of Life Scale (self-reported survey, 0 low quality of life - 100 high quality of life) and compare across groups
Multiple Sclerosis Spasticity Scale (MSSS-88)
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Assess pain using portions of the MSSS-88 (self-reported survey, 21 not at all bothered - 84 extremely bothered) and compare across groups
3 secondary outcomes
Change in gait and motion analysis
Time frame: Baseline (qualifying exam for cases and controls) compared to long-term follow-up research visit (on average 10 years post-baseline)
Incidence of prior surgery and anti-spastic treatments
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Cost of prior surgery and anti-spastic treatments
Time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2023.
- Derived from this study: PubMed 31227534Munger ME, Chen BP, MacWilliams BA, McMulkin ML, Schwartz MH. Comparing the effects of two spasticity management strategies on the long-term outcomes of individuals with bilateral spastic cerebral palsy: a multicentre cohort study protocol. BMJ Open. 2019 Jun 20;9(6):e027486. doi: 10.1136/bmjopen-2018-027486.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Michael Schwartz, PhD principal investigator
Gillette Children's Specialty Healthcare
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cerebral Palsy, run by Gillette Children's Specialty Healthcare at 3 sites 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 14 inclusion and 1 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: finished, as of the sponsor's last update on 13 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Three-dimensional gait and motion analysis (time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)). The study lists 13 primary outcomes in all. 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 NCT03789786, which also lists 1 linked publication.
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 13 Oct 2023.