Measuring limb movement in people with FGFR3-related skeletal dysplasia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This prospective, longitudinal monocentric project at Hôpital Necker will collect repeated limb movement recordings from people with FGFR3-related skeletal dysplasia using Syde® wearable technology. Participants aged 3 and older with genetically confirmed achondroplasia or hypochondroplasia who can walk at least 10 meters unassisted will wear devices and complete questionnaires during four-week recording periods every six months. The main aim is to identify which Syde®-derived digital outcomes are reliable and reproducible as natural history measures. Collected data will be analyzed for measurement reliability, feasibility of remote device use, and suitability as endpoints for future studies.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1.a. Aged 2 years old or older at the time of consent (France and Belgium)

1. b. Aged between 2 years old and \< 17 years old at the time of consent (United Kingdom)
2. Written informed consent obtained:

   1. From the parent(s) or legal guardian(s) for participants under 18 years old.
   2. From participants 18 years old and older.
3. Affiliated to, or beneficiary of a social security category (not applicable for the United Kingdom)
4. Able to walk unassisted for at least 10 meters.
5. Genetically confirmed diagnosis with one pathogenic variant (ACMG class IV \& V) of achondroplasia or hypochondroplasia.
6. Participant (and caregivers for participants under 18 years old) willing and able to comply with all study procedures including: questionnaires, Syde® related procedures

Exclusion Criteria:

1. Subjects who have short stature condition other than ACH/HCH.
2. Presence of cognitive disorders that limit their understanding of the data collection process (training of device use and 4-week recording periods every 6 months, device return at the end of the study), the implication of the study and consent.
3. Presence or history of any concurrent disease or condition that could interfere with study participation, impact pediatric growth, affect motor or balance or gait function (such as neurological, endocrine, infectious, allergic, osteoarthritis, or inflammatory), assessed by the investigator.
4. Females who are pregnant, or planning to become pregnant during the study duration.
5. Body Mass Index = 35 kg/m2.
6. Recent upper and/or lower limbs injury (trauma/fracture or surgery) in the 6 months preceding inclusion.
7. Prior limb lengthening surgery or planned or expected to have limb lengthening surgery while enrolled in the study.
8. Presence of guided growth hardware (such as 8-plates) or planned orthopedic surgeries during the study.
9. Vulnerable patient (guardianship, curatorship or safeguarding of justice), unable to provide informed consent or who is unable to express their consent.

Where it's running

• Hopital Necker
  Paris, France Recruiting
• CHU de Liège
  Liège, Belgium Not yet recruiting
• Hôpital de la Citadelle
  Liège, Belgium Not yet recruiting
• Myriad Trials
  London, United Kingdom Not yet recruiting

Study contacts

• Principal investigator: Genevieve Baujat — Hopital Necker
• Study coordinator: Ferial Toumi
• Email: ferial.toumi@sysnav.fr
• Phone: +33 2 78 00 10 98

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.