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Cerebral Palsy Hip Outcomes Project - International Multi-centre Study

Conditions
Cerebral Palsy
Registration Number
NCT01987882
Lead Sponsor
Holland Bloorview Kids Rehabilitation Hospital
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.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
500
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.

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
CPCHILD questionnaire: Total Score (0-100)Change from baseline to 12 months after treatment

The primary outcome measure will be based at the 12 month assessment. The CPCHILD questionnaire is a validated outcome measure of health-related quality of life specifically developed for children with severe, non-ambulant cerebral palsy (GMFCS level IV and V) to evaluate the effectiveness of different treatment options.

Secondary Outcome Measures
NameTimeMethod
Reimer's Migration Percentage (MP)Change from baseline to 24 months after treatment

The Reimer's Migration Percentage (MP) quantifies the magnitude of the displacement of the femoral head that is uncovered by the acetabulum.

CPCHILD Questionnaire: Total Score (0-100)Change from baseline to 24 months after treatment

The CPCHILD questionnaire is a validated outcome measure of health-related quality of life specifically developed for children with severe, non-ambulant cerebral palsy (GMFCS level IV and V) to evaluate the effectiveness of different treatment options.

Acetabular Index (AI) in degreesChange from baseline to 24 months after treatment

The Acetabular Index (AI) in degrees quantifies the magnitude of acetabular dysplasia.

Morphological Hip Classification in Cerebral Palsy (MHC)Change from baseline to 24 months after treatment

The Morphological Hip Classification in Cerebral Palsy (MHC) describes the sphericity of the femoral head.

CPCHILD Questionnaire: Subscale Scores (0 - 100)Change from baseline to 24 months after treatment

The Subscale scores of the CPCHILD© questionnaire represent the following domains:

1. Activities of Daily Living/Personal Care (9 items);

2. Positioning, Transferring \& Mobility (8);

3. Comfort \& Emotions (9);

4. Communication \& Social Interaction (7);

5. Health (3);

6. Overall Quality of Life (1).

Standardized scores are generated on a scale of 0 (worst) to 100 (best) for each of the 6 domains.

Trial Locations

Locations (27)

Shriners Hospitals for Children - Northern California

🇺🇸

Sacramento, California, United States

Alfred I. Dupont Institute

🇺🇸

Wilmington, Delaware, United States

Children's Hospital Colorado

🇺🇸

Aurora, Colorado, United States

Children's Hospital Boston

🇺🇸

Boston, Massachusetts, United States

Children's of Mississippi

🇺🇸

Jackson, Mississippi, United States

Gillette Children's Specialty Healthcare

🇺🇸

Saint Paul, Minnesota, United States

Hospital for Special Surgery

🇺🇸

New York, New York, United States

New York-Presbyterian Morgan Stanley Children's Hospital

🇺🇸

New York, New York, United States

Royal Children's Hospital

🇦🇺

Melbourne, Victoria, Australia

Cincinnati Children's Hospital Medical Center

🇺🇸

Cincinnati, Ohio, United States

BC Children's Hospital

🇨🇦

Vancouver, British Columbia, Canada

Aarhus University Hospital

🇩🇰

Aarhus, Denmark

Holland Bloorview Kids Rehabilitation Hospital

🇨🇦

Toronto, Ontario, Canada

The Hospital for Sick Children

🇨🇦

Toronto, Ontario, Canada

Shriners Hospital for Children - Canada

🇨🇦

Montreal, Quebec, Canada

Starship Children's Hospital of New Zealand

🇳🇿

Auckland, New Zealand

K. Marcinkowski Medical University

🇵🇱

Poznan, Poland

University Hospitals Coventry & Warwickshire NHS Trust

🇬🇧

Coventry, England, United Kingdom

Alder Hey Children's NHS Foundation Trust

🇬🇧

Liverpool, England, United Kingdom

Lund University Hospital

🇸🇪

Lund, Sweden

Safra Hospital for Children

🇮🇱

Tel Hashomer, Ramat Gan, Israel

Seoul National University Bundang Hospital

🇰🇷

Seongnam, Gyeonggi-do, Korea, Republic of

Royal National Orthopaedic Hospital

🇬🇧

London, England, United Kingdom

Astrid Lindgren's Children's Hospital

🇸🇪

Stockholm, Sweden

The Royal London and St. Bartholomew's Hospitals

🇬🇧

London, England, United Kingdom

Nuffield Orthopaedic Centre NHS Trust

🇬🇧

Oxford, England, United Kingdom

Royal Hospital for Sick Children

🇬🇧

Edinburgh, Scotland, United Kingdom

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