BIOchemical, DEnsitometric, TEChnological and Imaging Tools to Evaluate the Bone/muscle in Children with Cerebral Palsy

NCT06672731 | Not Yet Recruiting

• 2 other identifiers: PRIN2022PRIN2002
• Study Type: interventional
• Target: 100
• Locations: 0 countries
• Sites: N/A

Brief Summary

Cerebral palsy (CP) is the most common chronic disability in childhood, burden by motor, sensation, cognition, feeding and communication impairment. A serious concern in children with CP is bone/muscle health deterioration, which negatively impacts the already reduced quality of life (QoL). Children with CP show low bone density, vitamin D deficiency, sarcopenia and high risk of fragility fractures, with heavy effects on what is already limited home, school and community life. The causes for muscle-bone impairment are low weight-bearing deambulation during skeletal formation with low bone mineralization, poor nutrition and low calcium intake, low sun exposure, use of anticonvulsant medications with a negative profile on bone. Understanding the causes affecting bone quality and setting up interventions to reduce the impact of physical disability are essential in young subjects with CP. This project combines complementary expertise and resources in the fields of Endocrinological Biochemistry, Paediatric Neurological Disorders and Neuroimaging, to allow an innovative, technology-assisted workup for bone/muscle health evaluation in young subjects with CP, which could drive novel therapeutics, nutritional and rehabilitation programs. The first aim of this project is to evaluate bone/muscle health in young subjects with CP compared with sex-age matched healthy subjects, providing i) serum biomarkers of mineral metabolism and the metabolome of Vitamin D, assessed with last generation Mass Spectrometry, ii) muscle sarcopenia markers like Irisin and other myokines, depicting the response of the muscle to exercise iii) neuronal damage and inflammatory markers, iv) densitometric data by the low-cost and safe Quantitative ultrasound (QUS) at phalanges of the hand, plus the novel and very promising Radiofrequency Echographic Multi Spectrometry (REMS) served by the software for fragility fractures risk. We also aim to correlate the previous mentioned markers of bone/muscle health with a combination of demographic, clinical, cognitive and technological parameters, the last obtained by an innovative use of wearable sensors or actigraphs, positioned at the wrists, which depicts movements, physical activity (PA), energy expenditures (EE) and, together with heart-rate monitors, metabolic data during a normal like week in subjects with CP and healthy controls. Finally, a newly validated scoring for brain lesions in subjects with CP and the production of imaging "biomarkers" of neuronal damage, will be correlated with their bone/muscle health data, PA and EE to understand the impact of brain damage on functional performance and bone metabolism.

Conditions

Cerebral Palsy Infantile

Keywords

cerebral palsy; bone health; muscle health

Outcome Measures

Primary Outcomes (3)

• Bone/Muscle Unit Health Assessment

  We will achieve the aim 1 by the following evaluations: \- 25OHD changes evaluated as plasma levels of Vitamin D, intended as hypovitaminosis D 25OHD\<20 ng/ml

  at enrollment

• Evaluation of bone mineral density as QUS

  AD-SOS (amplitude-dependent speed of sound) expressed as standard deviation compared to normal (absolute number) and BTT (bone transmission time expressed in seconds)

  at enrollment

• Evaluation of bone/muscle score at REMS

  T score and Z score at REMS are index of bone fagility and are expressed as standard deviation difference compared to normal (absolute number, cut off \<2.5). Moreover a % of fragility risk is produced (cut-off \>10%)

  at enrollment

Study Arms (2)

Children with cerebral palsy

EXPERIMENTAL

50 children with cerebral palsy in whom the intervention is intended as the application of already validated movement sensors, namely actigraphs.

Diagnostic Test: REMS: Radiofrequency Echographic Multi Spectrometry

Typically developing children

ACTIVE COMPARATOR

50 healthy children, evaluated c/o AOUP for the exclusion of other diseases, in whom for intervention is intended the application of already validated movement sensors.

Diagnostic Test: REMS: Radiofrequency Echographic Multi Spectrometry

Interventions

REMS: Radiofrequency Echographic Multi Spectrometry DIAGNOSTIC_TEST

it is a non invasive ultrasonographic evaluation of bone and muscle quality

Children with cerebral palsy; Typically developing children

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• age
• willing to sign inform content

You may not qualify if:

• bisphosphonates or bone diseases

Sponsors & Collaborators

• IRCCS Fondazione Stella Maris: lead
• University of Pisa: collaborator
• Azienda Ospedaliero, Universitaria Pisana: collaborator

Related Publications (7)

• Tinelli F, Guzzetta A, Purpura G, Pasquariello R, Cioni G, Fiori S. Structural brain damage and visual disorders in children with cerebral palsy due to periventricular leukomalacia. Neuroimage Clin. 2020;28:102430. doi: 10.1016/j.nicl.2020.102430. Epub 2020 Sep 11.

  PMID: 32980597 BACKGROUND

• Di Paola M, Gatti D, Viapiana O, Cianferotti L, Cavalli L, Caffarelli C, Conversano F, Quarta E, Pisani P, Girasole G, Giusti A, Manfredini M, Arioli G, Matucci-Cerinic M, Bianchi G, Nuti R, Gonnelli S, Brandi ML, Muratore M, Rossini M. Radiofrequency echographic multispectrometry compared with dual X-ray absorptiometry for osteoporosis diagnosis on lumbar spine and femoral neck. Osteoporos Int. 2019 Feb;30(2):391-402. doi: 10.1007/s00198-018-4686-3. Epub 2018 Sep 4.

  PMID: 30178159 BACKGROUND

• Tomai Pitinca MD, Fortini P, Gonnelli S, Caffarelli C. Could Radiofrequency Echographic Multi-Spectrometry (REMS) Overcome the Limitations of BMD by DXA Related to Artifacts? A Series of 3 Cases. J Ultrasound Med. 2021 Dec;40(12):2773-2777. doi: 10.1002/jum.15665. Epub 2021 Feb 21.

  PMID: 33615539 BACKGROUND

• Baroncelli GI. Quantitative ultrasound methods to assess bone mineral status in children: technical characteristics, performance, and clinical application. Pediatr Res. 2008 Mar;63(3):220-8. doi: 10.1203/PDR.0b013e318163a286.

  PMID: 18287958 BACKGROUND

• Colaianni G, Faienza MF, Sanesi L, Brunetti G, Pignataro P, Lippo L, Bortolotti S, Storlino G, Piacente L, D'Amato G, Colucci S, Grano M. Irisin serum levels are positively correlated with bone mineral status in a population of healthy children. Pediatr Res. 2019 Mar;85(4):484-488. doi: 10.1038/s41390-019-0278-y. Epub 2019 Jan 15.

  PMID: 30683930 BACKGROUND

• Saggese G, Vierucci F, Prodam F, Cardinale F, Cetin I, Chiappini E, De' Angelis GL, Massari M, Miraglia Del Giudice E, Miraglia Del Giudice M, Peroni D, Terracciano L, Agostiniani R, Careddu D, Ghiglioni DG, Bona G, Di Mauro G, Corsello G. Vitamin D in pediatric age: consensus of the Italian Pediatric Society and the Italian Society of Preventive and Social Pediatrics, jointly with the Italian Federation of Pediatricians. Ital J Pediatr. 2018 May 8;44(1):51. doi: 10.1186/s13052-018-0488-7.

  PMID: 29739471 BACKGROUND

• Braito I, Maselli M, Sgandurra G, Inguaggiato E, Beani E, Cecchi F, Cioni G, Boyd R. Assessment of upper limb use in children with typical development and neurodevelopmental disorders by inertial sensors: a systematic review. J Neuroeng Rehabil. 2018 Nov 6;15(1):94. doi: 10.1186/s12984-018-0447-y.

  PMID: 30400992 BACKGROUND

MeSH Terms

Conditions

Cerebral Palsy

Condition Hierarchy (Ancestors)

Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Central Study Contacts

Federica Saponaro MD, PhD, Md, Phd

CONTACT

+393204964028; federica.saponaro@unipi.it

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 30, 2024
• First Posted: November 4, 2024
• Study Start: November 1, 2024
• Primary Completion: May 31, 2025
• Study Completion: December 31, 2025
• Last Updated: November 4, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share