Clinical trial NCT01987882: Cerebral Palsy Hip Outcomes Project - International Multi-centre Study
Official title: Outcomes of Hip Interventions for Children With Cerebral Palsy - An International Multi-centre Prospective Comparative Cohort Study
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Cerebral Palsy
Keywords: Outcomes research, treatment effectiveness, quality of life
Lead sponsor: Holland Bloorview Kids Rehabilitation Hospital
Sponsor class: OTHER
Collaborators: name: The Hospital for Sick Children
First posted: 2013-11-19
Enrollment: 500
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children with severe, non-ambulant cerebral palsy (GMFCS level IV and V) and their parent / primary caregiver will be enrolled in the study.

Brief summary: The primary purpose of the project is to evaluate the effectiveness of different intervention strategies to prevent or relieve symptoms associated with hip instability in children with severe cerebral palsy, using the validated Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD©) questionnaire as the primary outcome measure of health-related quality of life for this population.

Detailed description: Background: Children with severe cerebral palsy (CP) are at high risk for dislocating their hips. These hips are associated with contractures and pain, which can interfere with care-giving, seating, positioning, mobility and quality of life. The primary purpose of this project (Aim 2) is to evaluate the effectiveness of different intervention strategies to prevent or relieve the symptoms associated with hip instability in children with severe non-ambulatory CP, using the validated Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD©) questionnaire as the primary outcome measure of health related quality of life (HRQL) for this population. Secondarily, this project will also measure the impact of hip displacement on HRQL of these children. This project will be the first of its kind and this scale, which will systematically study the impact of hip instability and its management in children with severe CP, using a meaningful outcome measure that was developed specifically for this purpose. The international network of investigators/sites and the infrastructure established for this project will facilitate the long term follow-up of the participants in this study, as well as the conduct of other multi-centre clinical trials and cohort studies to evaluate the effectiveness of current and future interventions aimed at improving the quality of life of children with severe disabilities.

Study Design \& Participants: International multi-centre prospective longitudinal cohort study of children with severe (non-ambulant) cerebral palsy (GMFCS levels IV \& V) from ages 3 to 18 who have radiographic evidence of hip displacement \[Reimer's Migration Percentage (MP) ≥ 30%\].

Measures: Detailed demographic information, and prognostic factors, including co-morbid conditions will be recorded at baseline, in addition to self-administered parental reports of HRQL as measured by the CPCHILD. Hip status will be classified using standardized radiographic measures of Reimer's MP and acetabular index (AI). The primary outcome measure CPCHILD, as well as the MP \& AI will be measured at 6, 12 and 24 months following initial intervention.

Aim 1: Measure the impact of increasing hip displacement in children with severe (non-ambulant) CP on their HRQL as measured by the CPCHILD questionnaire.

Aim 2: (Primary Purpose): Measure the effectiveness of different strategies of interventions for hip displacement in children with severe (non-ambulant) CP in a prospective longitudinal comparative cohort study using the CPCHILD as the primary outcome measure of HRQL.

Aim 3: Compare the types and rates of adverse events and complications associated with each of the treatment cohorts.

Methods: Observational study of usual (site/surgeon specific) clinical practice. Investigators at each site will enroll eligible participants and assign each to one of the following 5 cohorts based on individual treating doctor's \&/or parental preferences:

A. "Natural" history or watchful waiting (N=100)

B. Serial botulinum toxin injections +/- abduction bracing (N=100)

C. Adductor (+/- psoas) muscle releases alone (N=100)

D. Hip reconstructive surgery (N=100)

E. Salvage hip surgery (N=100)

The baseline MP and CPCHILD scores for all participants will be analyzed cross-sectionally to evaluate the correlation between hip displacement and the CPCHILD scores to serve Aim 1. For Aim 2, children undergoing interventions for hip instability (Groups B, C, D, \& E) will be compared with each other as well as with their respective matched counterparts of untreated children (Group A), using repeated measures of analysis of covariance (ANOCOVA) to measure the mean change in scores from baseline at 6, 12 and 24 months after intervention.

Timelines: 500 participants will be recruited in 24 months, and followed for 24 months. The analysis, reporting of results, manuscript development and knowledge transfer will take 12 months. In total, the study will take 5 years to complete.

Eligibility criteria: Inclusion Criteria:

* Children with a working diagnosis of cerebral palsy or cerebral palsy-like condition
* Between 2 to 18 years of age
* Has a migration percentage ≥ 30%
* Non-ambulatory; the primary mode of mobility is a wheelchair
* Parent/primary caregiver must understand one of the languages in which the CPCHILD has been translated, culturally adapted and validated.

Exclusion Criteria:

* History of prior hip surgery
* Received botulinum toxin injection within the previous 3 months. Recruitment will be delayed until at least 3 months after prior botulinum toxin injection.

Primary outcomes:
measure: CPCHILD questionnaire: Total Score (0-100)
timeFrame: Change from baseline to 12 months after treatment
Secondary outcomes:
measure: CPCHILD Questionnaire: Total Score (0-100)
timeFrame: Change from baseline to 24 months after treatment
measure: Reimer's Migration Percentage (MP)
timeFrame: Change from baseline to 24 months after treatment
measure: Acetabular Index (AI) in degrees
timeFrame: Change from baseline to 24 months after treatment
measure: Morphological Hip Classification in Cerebral Palsy (MHC)
timeFrame: Change from baseline to 24 months after treatment
measure: CPCHILD Questionnaire: Subscale Scores (0 - 100)
timeFrame: Change from baseline to 24 months after treatment
Other outcomes:
measure: Adverse Events and Complications
timeFrame: Adverse events will be recorded up to 24 months after treatment
Locations:
city: Sacramento
country: United States
facility: Shriners Hospitals for Children - Northern California
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Wilmington
country: United States
facility: Alfred I. Dupont Institute
state: Delaware
city: Boston
country: United States
facility: Children's Hospital Boston
state: Massachusetts
city: Saint Paul
country: United States
facility: Gillette Children's Specialty Healthcare
state: Minnesota
city: Jackson
country: United States
facility: Children's of Mississippi
state: Mississippi
city: New York
country: United States
facility: Hospital for Special Surgery
state: New York
city: New York
country: United States
facility: New York-Presbyterian Morgan Stanley Children's Hospital
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Melbourne
country: Australia
facility: Royal Children's Hospital
state: Victoria
city: Vancouver
country: Canada
facility: BC Children's Hospital
state: British Columbia
city: Toronto
country: Canada
facility: Holland Bloorview Kids Rehabilitation Hospital
state: Ontario
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Montreal
country: Canada
facility: Shriners Hospital for Children - Canada
state: Quebec
city: Aarhus
country: Denmark
facility: Aarhus University Hospital
city: Tel Hashomer
country: Israel
facility: Safra Hospital for Children
state: Ramat Gan
city: Seongnam
country: Korea, Republic of
facility: Seoul National University Bundang Hospital
state: Gyeonggi-do
city: Auckland
country: New Zealand
facility: Starship Children's Hospital of New Zealand
city: Poznan
country: Poland
facility: K. Marcinkowski Medical University
city: Lund
country: Sweden
facility: Lund University Hospital
city: Stockholm
country: Sweden
facility: Astrid Lindgren's Children's Hospital
city: Coventry
country: United Kingdom
facility: University Hospitals Coventry & Warwickshire NHS Trust
state: England
city: Liverpool
country: United Kingdom
facility: Alder Hey Children's NHS Foundation Trust
state: England
city: London
country: United Kingdom
facility: Royal National Orthopaedic Hospital
state: England
city: London
country: United Kingdom
facility: The Royal London and St. Bartholomew's Hospitals
state: England
city: Oxford
country: United Kingdom
facility: Nuffield Orthopaedic Centre NHS Trust
state: England
city: Edinburgh
country: United Kingdom
facility: Royal Hospital for Sick Children
state: Scotland
Study officials:
affiliation: Holland Bloorview Kids Rehab and The Hospital for Sick Children, Toronto, Canada
name: Dr. Unni Narayanan, MBBS, MSc, FRCSC
role: PRINCIPAL_INVESTIGATOR
References:
citation: Narayanan UG, Fehlings D, Weir S, Knights S, Kiran S, Campbell K. Initial development and validation of the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD). Dev Med Child Neurol. 2006 Oct;48(10):804-12. doi: 10.1017/S0012162206001745.