GUIDed Growth of the Proximal Femur to Prevent Further Hip MigrAtion in CErebral Palsy Patients (GUIDANCE)

In recent literature, the potential of guided growth of the proximal femur to modify hip growth in patients with cerebral palsy has been shown. Using medial hemi-epiphysiodesis of the proximal femur (TMH-PF) morphology of hips at risk of symptomatic (sub)luxation in cerebral palsy (CP) can be changed, aiming to reduce further hip migration and the need for more invasive surgical treatment modalities. Further research is necessary to assess if the results of TMH-PF in combination with adductor tenotomies are significantly better than the results of the current standard of care; adductor tenotomies alone.

The investigators objective is to determine whether guided growth of the proximal femur decreases the risk of further hip migration and need for further surgery in cerebral palsy patients.

Study Overview

• Status: Recruiting
• Conditions: Cerebral Palsy; Hip Dislocation
• Intervention / Treatment: Procedure: Temporary Medial Hemi-epiphysiodesis of the proximal femur

• Study Type: Interventional
• Enrollment (Estimated): 84
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Merel Roelen, MD; Phone Number: +31639173455; Email: m.roelen@erasmusmc.nl
• Study Contact Backup: Name: Jaap Tolk, MD PhD; Email: j.tolk@erasmusmc.nl

Study Locations

• Netherlands
  • Amsterdam, Netherlands
    • Not yet recruiting
    • Amsterdam UMC
    • Contact: Melinda Witbreuk, MD PhD
  • Groningen, Netherlands
    • Recruiting
    • UMC Groningen
    • Contact: Sophie Moerman, MD PhD
  • Maastricht, Netherlands
    • Recruiting
    • Maastricht UMC
    • Contact: Adhiambo Witlox, MD PhD
  • Nijmegen, Netherlands
    • Not yet recruiting
    • Sint Maartenskliniek
    • Contact: Arno ten Ham, MD
  • Rotterdam, Netherlands
    • Recruiting
    • Erasmus MC
    • Contact: Merel Roelen, MD
    • Contact: Jaap Tolk, MD PhD
    • Contact: Renee van Stralen, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Spastic CP
• GMFCS level IV-V
• Aged 2-8 years
• At least one hip with an abduction in flexion ≤ 40 degrees
• Migration percentage of 30-50%
• Head shaft angle > 145

Exclusion Criteria:

• Not fit for surgery
• History of bony hip surgery to the affected hip
• Severe acetabular dysplasia defined as a gothic arch, an incongruent joint or an acetabular index > 30 degrees, consistent with A2 and A3 acetabular deformity according to Robin and Graham in an adequately performed radiographs

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Adductor tenotomy + Temporary Medial Hemi-epiphysiodesis of the proximal femur; Standard care + intervention	Procedure: Temporary Medial Hemi-epiphysiodesis of the proximal femur; Temporary Medial Hemi-epiphysiodesis of the proximal femur
No Intervention: Adductor tenotomy; Standard care

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment failure	The Primary study outcome is treatment failure, defined as: Need for secondary bony hip surgery to the affected hip or progression to hip migration > 50% at 5-year follow-up . An indication for secondary bony surgery will be defined as;; Migration percentage > 50% at a minimum of 1 year after surgery, OR; An increased migration > 10% in 1 year during follow-up; Increase in Acetabular index to > 30 degrees or an increase of more than 5 degrees during follow-up	from enrollment to the end of study (8 years)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Radiographic parameters	Standardized conventional radiographs, AP and Lauenstein view (frog leg lateral) Pelvic radiographs will be performed according to the standardized protocol as defined by AACPDM; Migration percentage (%), change in migration percentage per treatment arm (on baseline and follow-up); Head Shaft angle (HSA) in degrees; Acetabular index; Melbourne hip classification; Pelvic obliquity; Positioning of the hemi epiphysiodesis screw in the intervention group	from enrollment to the end of study (8 years)
3-dimensional shape analysis of proximal femur	CT-scans of the hip will be performed in the intervention group directly postoperative, at 2-year follow-up and 5-year follow-up. On these scans the following measurements will be performed: Positioning of the hemi epiphysiodesis screw; 3-dimensional proximal femoral shape analysis regarding (change in) sphericity, congruency and head-neck orientation will be performed in patients at 2-year follow-up and at 5-year follow-up.; The direction of migration of the hip and acetabular morphology will be determined according to the method described by Brunner et al.; Sphericity of the femoral head will be determined by the Mose technique.	directly postoperative, at 2-year follow-up and at 5-year follow-up
CPChild questionnaire	"Caregivers Priorities and Child Health Index of Life with Disabilities" The CPCHILD™ is a parent completed questionnaire and was developed to help clinicians to identify areas that are impairing a child's quality of life, determine what is most important to the child and caregiver, and monitor the child's progress in a way that is meaningful to the family. Minimum value is 0 and maximum value is 100, the higher the score the better the outcome.	from enrollment to the end of study (8 years)
CPG questionnaire	CPG = "Checklist Pijn Gedrag" (Dutch for Checklist Pain behaviour) A mean pain score will be calculated. A minimum value of 0 with a maximum value of 10. Higher scores mean more pain.	from enrollment to the end of study (8 years)
Secondary surgical interventions other than bony procedure	Screw revision ▪ The following will be used as indications for screw revision in the intervention group throughout the study period: < 1 thread of the screw remaining in the epiphysis, and; HSA > 100 degrees Repeated soft tissue release. ▪ The following will be used as indications for repeated soft tissue releases (adductor and/or psoas tenotomy) in both the intervention and control group throughout the study period: Abduction < 30 degrees, and; No indication for bony surgery	from enrollment to the end of study (8 years)

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Baseline measurements	Other patient related measurements that will be obtained at baseline include; Age; Weight; Height; GMFCS classification; Change in Migration Percentage in the year prior to the adductor tenotomy; Use of tone regulation; Baclofen, Artane, Baclofen pump, Selective Dorsal Rhizotomy (SDR	At the time of enrollment

Collaborators and Investigators

• Sponsor: Erasmus Medical Center
• Investigators: Principal Investigator: Jaap Tolk, MD, PhD, Erasmus Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): March 6, 2024
• Primary Completion (Estimated): April 1, 2029
• Study Completion (Estimated): December 1, 2030

Study Registration Dates

• First Submitted: October 25, 2023
• First Submitted That Met QC Criteria: November 1, 2023
• First Posted (Actual): November 7, 2023

Study Record Updates

• Last Update Posted (Actual): October 8, 2024
• Last Update Submitted That Met QC Criteria: October 7, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Wounds and Injuries; Brain Damage, Chronic; Joint Diseases; Musculoskeletal Diseases; Hip Injuries; Joint Dislocations; Cerebral Palsy; Hip Dislocation
• Other Study ID Numbers: PanamaID 10082

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Individual de-identified participant data that underlie the results from the GUIDANCE study and the study protocol will be shared if requested. Data will be available beginning 12 months and ending 5 years following publication of this paper. Data will be available for researchers who provide a methodologically sound scientific proposal, which has been approved by an ethical committee. Proof of the latter should be provided. Analyses should achieve the aims as reported in the approved proposal. Proposals for data should be directed to the corresponding author.
• IPD Sharing Time Frame: Data will be available beginning 12 months and ending 5 years following publication of this paper
• IPD Sharing Access Criteria: Data will be available for researchers who provide a methodologically sound scientific proposal, which has been approved by an ethical committee. Proof of the latter should be provided. Analyses should achieve the aims as reported in the approved proposal.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No