NCT06584851 3D-Microscopic Muscle Architecture in Cerebral Palsy

Eligibility & Interventions

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What to Expect as a Participant

This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.

This trial targets 130 participants in total. It began in 2019-05-02 with a primary completion date of 2028-12.

Brief Summary

The focus of this study is to understand and define the mechanisms of the altered muscle development and growth on a microscopic level within a long-term perspective in children with cerebral palsy and to relate these findings to muscle macroscopic properties defined by muscle imaging, to neuromuscular symptoms and to treatment. This study aims to (1) evaluate intrinsic microscopic muscle properties of young growing children with CP, and (2) to evaluate these muscle properties in relation to macroscopic properties, neuromuscular symptoms and to treatment. Improved understanding of changes in microscopic muscle properties, and how they relate to macroscopic properties and to the neuromuscular symptoms as well as how they are influenced by treatment, has the potential to delineate CP phenotypes prone to intervention and to optimize treatment protocols or develop new treatments, leading to new avenues for improving function in CP. The method to study microscopic muscle properties involves analysis of muscle biopsies (histological / immunohistochemistry analysis, SC and IC culture, gene expression). Biopsies will be collected using the minimally invasive percutaneous needle microbiopsy technique, suitable for collecting repeated samples over time in the same individual while still leading to sufficient tissue of good quality for subsequent analysis1,2. For the children with CP, the local hospital's tradition of applying general anesthesia for delivering BTX injections will be exploited to collect the muscle samples prior to the BTX session and the one-year follow-up, or general anesthesia planned for orthopedic surgery or diagnostic imaging such as MRI, etc. For the collection of the samples 3 months before the BTX session, as well as 3 months and 6 months after BTX injections, the common approach for microbiospy collection will be applied, with local sedation on the skin (Rapydan©) and fascia (Xylocaine©) and local anesthesia by using Kalinox© (nitrous oxide in oxygen) under supervision of the University Hospital PROSA team. Biopsies of TD muscles will be collected in children with no history of neurological disorder, nor musculoskeletal problems at the level of the gastrocnemius or semitendinosus, at the time of upper limb orthopedic or trauma surgery and thus always under general anesthesia. Ultrasound guided percutaneous muscle biopsy has been performed in children (2 months-18 years)3,4 and has proven to be safe and well-tolerated. A pilot study (S61110) was conducted to confirm that the microbiopsy technique is suitable for the analysis of microscopic muscle properties and is well-tolerated in children with CP. Two specific research goals are planned, with hypotheses emerging from literature.

Eligibility Criteria

CHILDREN WITH CP Inclusion criteria * Children (boys/girls) diagnosed with predominantly spastic type of CP * Uni- or bilateral involvement * Gross Motor Function Classification Scale (GMFCS) Level I-III50 * 2 to 9 years of age * Planned for an orthopedic intervention that requires general anesthesia (botulinum toxin injections, orthopedic surgery, or diagnostic imaging such as Magnetic Resonance Imaging \[MRI\], etc.) Exclusion criteria * Presence of dystonia or ataxia * Previous surgery less than 6 months at the investigated muscles * Severe co-morbidities (that are likely to prevent proper assessment, such as severe cognitive problems) TYPICALY DEVELOPING CHILDREN Inclusion criteria * Children (boys/girls) * 2 to 9 years of age * Planned for a surgical intervention that requires general anesthesia (pure pediatric upper limb orthopedic surgery or trauma surgery, or ophthalmic or ear-nose-throat surgery) Exclusion criteria * History of neurological problems * History of orthopedic problems at the gastrocnemius or semitendinosus * Trauma at the level of the lower limbs * Involvement in an elite or high-performance sporting program (Children performing sports for \> 3 5 hours/week will be excluded) ADOSESCENTS WITH HSP of 12-18 years old and ADULTS WITH HPS Inclusion criteria * Adolescents (boys/girls) or adults (male/female) diagnosed with HSP, SPG3a or SPG4 * Gross Motor Function Classification Scale (GMFCS) Level I-III50 * Adolescents 12 to 18 years or adults 18-40 years of age Exclusion criteria * Presence of dystonia or ataxia * Previous surgery less than 6 months at the investigated muscles * Severe co-morbidities (that are likely to prevent proper assessment, such as severe cognitive problems) TYPICALLY DEVELOPING ADOLESCENTS of 12-18 years old and HEALTHY ADULTS (age- and gender-matched with the recruited HSP patients) Inclusion criteria * Typically developing adolescents (boys/girls) and healthy adults (male/female), who are age and gender matched with the recruited HSP participants * Adolescents 12-18 years of age or adults 18-40 years of age Exclusion criteria * History of neurological problems * History of orthopedic or muscular problems at the gastrocnemius * Trauma at the level of the lower limbs * Involvement in an elite or high-performance sporting program (participants performing sports for \> 5 hours/week will be excluded) * Sport session less than 72 hours prior to the biopsy collection

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