Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age2-65
SponsorSYSNAV
About this trial
The study aims to identify which Syde®-derived digital outcomes are reliable in FGFR3-related Skeletal Dysplasia. This requires to set-up a natural history study to measure limb movements in patients with ACH or HCH.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
b. Aged between 2 years old and < 17 years old at the time of consent (United Kingdom)
From the parent(s) or legal guardian(s) for participants under 18 years old.
From participants 18 years old and older.
Affiliated to, or beneficiary of a social security category (not applicable for the United Kingdom)
Disqualifiers
Subjects who have short stature condition other than ACH/HCH.
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.
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.
Females who are pregnant, or planning to become pregnant during the study duration.
Trial population
France: Participants will be recruited from the patient registry of the Centre de Référence des Maladies Osseuses Constitutionnelles located at Necker Hospital / IMAGINE Institute. The APPT association (Association des Personnes de Petite Taille) will also help with the recruitment through flyers distribution. All visits will take place at Necker Hospital. Belgium: Participants will be recruited from the Maladies Rares Génétiques Ostéo-articulaires (MaRGOs) patient registry located at Centre Hospitalier Universitaire (CHU) de Liège, with additional recruitment through Centre Hospitalier Régional (CHR) de Liège and support from the Solidarité Wallonne des Personnes de Petite Taille association through flyer distribution. All visits will take place at CHU de Liège and CHR de Liège. United Kingdom: Participants will be recruited from the Site Research Waiting List
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
Reliability of digital endpoint derived from Syde® assessed by the Intra-Class Correlation (ICC) for each recording period timepoint.
reliability assessed by the ICC (Intra-Class Correlation)for each recording period timepoint (baseline, 6 months, 12 months)
Time frame
12 monthsSecondary outcomes
1
number of participants reaching 180 hours of recording during the recording periods defined in the protocol
Number of patients reaching 180 hours of recordings during the recording periods defined in the protocol (supposed optimal threshold for Syde®-derived digital endpoint computation) at baseline, 6 months, 12 months
Time frame
12 months2
System Usability Score (F-SUS) score at 6 months of Syde® use
System Usability Score (SUS) at 6 months. The System Usability Scale is a 10-item questionnaire . It is a simple tool to measure the usability of an interactive system and it is widely used. For each item, the user is asked to express their level of agreement or disagreement from 1 (strongly disagree) and 5 (strongly agree).
Time frame
6 months3
Correlation between digital endpoints derived from Syde® and 6-minutes walk test (6MWT)
Correlation at baseline and at different timepoints between Syde® derived digital endpoint and 6MWT
Time frame
12 months4
Correlation between digital endpoints derived from Syde® and growth parameters
Correlation at baseline and at different timepoints between Syde® derived digital endpoints and annualized growth velocity, height Z-score, body proportion ratios
Time frame
12 months Source ClinicalTrials.gov