AchondroplasiaSKY-MOT3NCT07388966
Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia
An observational study of Achondroplasia and Hypochondroplasia, run by SYSNAV at 4 sites in 3 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 to 65 years
- Conditions the study is for
- Achondroplasia, Hypochondroplasia
- How many people
- 140 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 6 Feb 2026to Dec 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 2 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study 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
Who may be able to join
- 1.a. Aged 2 years old or older at the time of consent (France and Belgium)
- b. Aged between 2 years old and < 17 years old at the time of consent (United Kingdom)
- Written informed consent obtained:
- 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)
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- Able to walk unassisted for at least 10 meters.
- Genetically confirmed diagnosis with one pathogenic variant (ACMG class IV \& V) of achondroplasia or hypochondroplasia.
- Participant (and caregivers for participants under 18 years old) willing and able to comply with all study procedures including: questionnaires, Syde® related procedures
Who cannot join
- 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.
- Body Mass Index = 35 kg/m2.
- Recent upper and/or lower limbs injury (trauma/fracture or surgery) in the 6 months preceding inclusion.
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- Prior limb lengthening surgery or planned or expected to have limb lengthening surgery while enrolled in the study.
- Presence of guided growth hardware (such as 8-plates) or planned orthopedic surgeries during the study.
- Vulnerable patient (guardianship, curatorship or safeguarding of justice), unable to provide informed consent or who is unable to express their consent.
Trial locations and contacts
4 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
4 sites in 3 countries
Belgium2 sites, 2 recruiting
- Not yet recruiting
CHU de Liège
Liège, 4000
Julie HARVENGT
- Not yet recruiting
Hôpital de la Citadelle
Liège, 4000
Julie HARVENGT
France1 site, 1 recruiting
- Recruiting
Hopital Necker
Paris, 75015
Na An, +33 1 71 19 64 73, na.an@aphp.fr
Genevieve Baujat
United Kingdom1 site, 1 recruiting
- Not yet recruiting
Myriad Trials
London
Melita IRVING
About this study
Official title: Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia Using Syde®
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.
- Conditions
- Achondroplasia, Hypochondroplasia
- Medical subject headings
- Achondroplasia, Hypochondroplasia
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 12 months
Secondary outcomes (8)
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
Measured: 12 months
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).
Measured: 6 months
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
Measured: 12 months
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
Measured: 12 months
Correlation between digital endpoints derived from Syde® and Patient-reported Outcomes (PROs)
Correlation at baseline and at different timepoints between Syde® derived digital endpoints and PROs
Measured: 12 months
Longitudinal Change in digital endpoints derived from Syde®
Change from baseline in digital endpoints derived from Syde® at 6 and 12 months
Measured: 12 months
Syde® movement reconstruction accuracy compared to Optical Motion Capture measures.
Stride detection accuracy of Syde®, stride length and stride velocity accuracy of Syde® and upper limb measures, compared to Optical Motion Capture measures
Measured: Baseline
Qualitative interview exploring limitations on mobility and impact on daily life
caregiver and/or participant qualitative interview exploring limitations on mobility daily life
Measured: baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2026
- Study start6 Feb 2026
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AchondroplasiaStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 140 participants (estimated)
Sponsor and investigators
- Lead sponsor
- SYSNAV
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Genevieve Baujat, Principal investigator, Hopital Necker
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07388966
- Sponsor's protocol ID
- PR5030-108
- Other IDs
- 2025-A01608-41
- Acronym
- SKY-MOT3
- Status last verified
- Sep 2026
- First submitted
- 28 Jan 2026
- Last updated
- 23 Sep 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia Using Syde®
- ClinicalTrials.gov ID
- NCT07388966
- Acronym / sponsor ID
- SKY-MOT3 / PR5030-108
- Conditions
- Achondroplasia, Hypochondroplasia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 140 participants (estimated)
- Lead sponsor
- SYSNAVIndustry (pharma, biotech, devices)
- Study start
- 6 Feb 2026 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 5 Feb 2026 (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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 2 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study 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
Key inclusion criteria (9 listed)
- 1.a. Aged 2 years old or older at the time of consent (France and Belgium)
- b. Aged between 2 years old and < 17 years old at the time of consent (United Kingdom)
- Written informed consent obtained:
- Affiliated to, or beneficiary of a social security category (not applicable for the United Kingdom)
- Able to walk unassisted for at least 10 meters.
Key exclusion criteria (9 listed)
- 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.
- Body Mass Index = 35 kg/m2.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Reliability of digital endpoint derived from Syde® assessed by the Intra-Class Correlation (ICC) for each recording period timepoint.
Time frame: 12 months
reliability assessed by the ICC (Intra-Class Correlation)for each recording period timepoint (baseline, 6 months, 12 months)
8 secondary outcomes
number of participants reaching 180 hours of recording during the recording periods defined in the protocol
Time frame: 12 months
System Usability Score (F-SUS) score at 6 months of Syde® use
Time frame: 6 months
Correlation between digital endpoints derived from Syde® and 6-minutes walk test (6MWT)
Time frame: 12 months
Correlation between digital endpoints derived from Syde® and growth parameters
Time frame: 12 months
Correlation between digital endpoints derived from Syde® and Patient-reported Outcomes (PROs)
Time frame: 12 months
Longitudinal Change in digital endpoints derived from Syde®
Time frame: 12 months
Syde® movement reconstruction accuracy compared to Optical Motion Capture measures.
Time frame: Baseline
Qualitative interview exploring limitations on mobility and impact on daily life
Time frame: baseline
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2026.
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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.
Genevieve Baujat principal investigator
Hopital Necker
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Achondroplasia and Hypochondroplasia, run by SYSNAV at 4 sites in 3 countries.
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 9 inclusion and 9 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: looking for participants now, as of the sponsor's last update on 23 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Belgium, France, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ferial Toumi (+33 2 78 00 10 98, ferial.toumi@sysnav.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Reliability of digital endpoint derived from Syde® assessed by the Intra-Class Correlation (ICC) for each recording period timepoint. (time frame: 12 months). 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 23 Sep 2026.