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Muscle Abnormalities in Children With XLH (MDmuscleXLH)

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ClinicalTrials.gov Identifier: NCT05312086
Recruitment Status : Not yet recruiting
First Posted : April 5, 2022
Last Update Posted : April 5, 2022
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

Study Description
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Brief Summary:
XLH rickets is a rare disease with muscle weakness. Fat parameters such as IMAT and intraMAT could be increased in this disease. IMAT and intraMAT will be calculated on MRI for 11 XLH children versus 20 typically developing children. The investigator will compare the percentage of IMAT in the XLH group versus control group and the difference concerning the intraMAT between the two groups.

Condition or disease Intervention/treatment Phase
Muscle Weakness XLH Other: MRI Not Applicable

Detailed Description:

Single-center, prospective, non-randomized, controlled study with XLH and healthy volunteer children.

This study involves performing muscle MRI in healthy child volunteers to assess and compare the structure and composition of muscles to those of muscles in children with XLH pathology (muscle MRI of XLH patients is performed as part of standard medical care).

During this study we will have two groups: XLH group and a control group with typically developing children. In the XLH group, composed of 10 patients aged between 5 ans 17 years, female or male and ongoing growth (lesser bone age than 15 years old), have XLH and the most severe radiological deformities,clinical complications (pain and muscular weakness), have already done a muscle MRI of the lower limb during their follow-up.

The control group composed of 20 healthy volunteers aged between 5 ans 17 years old, female or male and without any endocrine pathology. A muscle MRI is necessary for healthy volunteers.

Every child will be alone After signing the informed consent by the 2 parents, the patient or the healthy volunteer and his parents, the MRI assessment will be performed on the same day.

Performing the muscle MRI requires the cooperation of the child in order to remain lying down and motionless throughout the duration of the MRI, i.e. approximately 45 minutes.

Performing MRI does not require the injection of contrast products or radiopharmaceuticals.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: Prospective Analysis of Quantitative and Qualitative Muscle Abnormalities in Children With X-linked Hypophosphatemia (XLH)
Estimated Study Start Date : April 15, 2022
Estimated Primary Completion Date : June 15, 2023
Estimated Study Completion Date : June 15, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
patients with X-linked hypophosphatemic
Patient group composed of 10 patients aged between 5 to 17 years old, female or male, with X-linked hypophosphatemic (XLH) rickets, severity of clinical and radiological damage (pain, muscle weakness and severe bone deformities) and ongoing growth (lesser bone age than 15 years old), child under conventional treatment
 Other: MRI

Performing the muscle MRI requires the cooperation of the child in order to remain lying down and motionless throughout the duration of the MRI, about 45 minutes.

Performing MRI does not require the injection of contrast products or radiopharmaceuticals.
healthy volunteers
control group composed of 20 helthy volunteers aged between 5 to 17 years old, female or male, without any endocrine pathology, not suffering from XLH, matching by age (+/- 6 months) and sex
 Other: MRI

Performing the muscle MRI requires the cooperation of the child in order to remain lying down and motionless throughout the duration of the MRI, about 45 minutes.

Performing MRI does not require the injection of contrast products or radiopharmaceuticals.


Outcome Measures
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Primary Outcome Measures :
  1. Quantify and compare muscle composition in XLH children versus healthy control children to demonstrate a link between muscle function and muscle quality. [ Time Frame: 1 day ]
    calculation of inter-muscular adipose tissue will be done in cm2


Secondary Outcome Measures :
  1. Measurement of muscle strength on force platforms, during gait analysis. Power test by mechanography of the jump (bipod and monopodal). Grasping test and correlation of fatty infiltration and functional data. [ Time Frame: 1 day ]
    The Outcome Measure is BMI (Kg/m²)

  2. Quantitative analysis of the muscles of the lower limbs (volume and length) [ Time Frame: 1 day ]
    The Outcome Measure is BMI (Kg/m²)


Eligibility Criteria
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Ages Eligible for Study:   5 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria common criteria to the 2 groups:

  • Age: between 5 and 17 years.
  • Sex: male or female.
  • Informed consent, signed by both parents or holder of parental autority after being informated of the study.
  • Affiliation to a social security schema or having the right to.

Inclusion criteria for XLH Patients :

  • Patient suffer of X-linked hypophosphatemia rickets.
  • Severity of clinical and radiological damage (pain, muscle weakness and severe bone deformities).
  • Growth in progress (bone age less than 15 years).
  • Child under conventional treatment: patient responding to conventional treatment or
  • under treatment with Burosumab (patient in treatment failure under treatment conventional).

incluion criteria for helthy volunteers :

  • Healthy voluntary subjects, not suffering from XLH.
  • Matching by age (+/- 6 months) and sex.

Exclusion Criteria common to the 2 groups :

  • Not being able to stay still during the MRI examination (approximately 45 min).
  • Growth completed.
  • History of lower limb surgery.
  • Contraindications to MRI
  • Holders of parental authority under AME.
  • Holders of parental authority under tutorship / curatorship.

Exclusion criteria for Healthy volunteers :

  • Patients with endocrine, contracted or muscular pathology.
  • Patient receives a long-term treatment.
  • Patients with a high athletic level.
  • BMI-IOTF <20 or> 27.8
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05312086


Contacts
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Contact: Agnés LINGLART, Pr 0145217116 agnes.linglart@aphp.fr
Contact: Philippe WICART, Pr 0144494310 p.wicart@aphp.fr

Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
More Information
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Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT05312086    
Other Study ID Numbers: 2020-A01386-33
First Posted: April 5, 2022    Key Record Dates
Last Update Posted: April 5, 2022
Last Verified: January 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Assistance Publique - Hôpitaux de Paris:
XLH rickets
IMAT
intraMAT
muscle weakness
MRI
Additional relevant MeSH terms:
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Muscle Weakness
Paresis
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes