Muscle WeaknessMDmuscleXLHNCT05312086
Muscle Abnormalities in Children With XLH
A study testing MRI in people with Muscle Weakness and XLH, run by Assistance Publique - Hôpitaux de Paris.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 17 yearsHealthy volunteers can take part
- Conditions the study is for
- Muscle Weakness, XLH
- What is being tested?
- MRI
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- No sites listed yet
- Study dates
- 15 Apr 2022to 15 Jun 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 17 years
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- 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.
- Patient suffer of X-linked hypophosphatemia rickets.
- Severity of clinical and radiological damage (pain, muscle weakness and severe bone deformities).
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- 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.
Who cannot join
- 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.
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- 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
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Prospective Analysis of Quantitative and Qualitative Muscle Abnormalities in Children With X-linked Hypophosphatemia (XLH)
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.
Read the 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.
- Conditions
- Muscle Weakness, XLH
- Being tested
- MRI
- Keywords
- XLH rickets, IMAT, intraMAT, muscle weakness, MRI
- Medical subject headings
- Muscle Weakness
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MRIOther
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.
| Study group | Role | What they receive |
|---|---|---|
| patients with X-linked hypophosphatemic | Other | 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 Receives: Other: MRI |
| healthy volunteers | Other | 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 Receives: Other: MRI |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quantify and compare muscle composition in XLH children versus healthy control children to demonstrate a link between muscle function and muscle quality.
calculation of inter-muscular adipose tissue will be done in cm2
Measured: 1 day
Secondary outcomes (2)
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.
The Outcome Measure is BMI (Kg/m²)
Measured: 1 day
Quantitative analysis of the muscles of the lower limbs (volume and length)
The Outcome Measure is BMI (Kg/m²)
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2022
- Study start15 Apr 2022 (estimated)
- Primary completion15 Jun 2023 (estimated)
- Study completion15 Jun 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscle WeaknessStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05312086
- Sponsor's protocol ID
- 2020-A01386-33
- Acronym
- MDmuscleXLH
- Status last verified
- Jan 2022
- First submitted
- 24 Jan 2022
- Last updated
- 5 Apr 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Analysis of Quantitative and Qualitative Muscle Abnormalities in Children With X-linked Hypophosphatemia (XLH)
- ClinicalTrials.gov ID
- NCT05312086
- Acronym / sponsor ID
- MDmuscleXLH / 2020-A01386-33
- Conditions
- Muscle Weakness, XLH
- Keywords
- XLH rickets, IMAT, intraMAT, muscle weakness, MRI
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 15 Apr 2022 (estimated)
- Primary completion
- 15 Jun 2023 (estimated)
- Study completion
- 15 Jun 2023 (estimated)
- First posted
- 5 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| patients with X-linked hypophosphatemic | Other | Other: MRIPatient 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 |
| healthy volunteers | Other | Other: MRIcontrol 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 |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (12 listed)
- 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.
- Patient suffer of X-linked hypophosphatemia rickets.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MRIPerforming 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. | Other | healthy volunteers, patients with X-linked hypophosphatemic |
Primary outcome
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
2 secondary outcomes
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
Quantitative analysis of the muscles of the lower limbs (volume and length)
Time frame: 1 day
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Apr 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Questions about this trial
What is this clinical trial studying?
A study testing MRI in people with Muscle Weakness and XLH, run by Assistance Publique - Hôpitaux de Paris.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Agnés LINGLART, Pr (0145217116, agnes.linglart@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Apr 2022; AzTrials last refreshed its copy on 6 Nov 2026.