NCT07388966

Brief 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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
40

participants targeted

Target at P25-P50 for all trials

Timeline
21mo left

Started Feb 2026

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress13%
Feb 2026Feb 2028

First Submitted

Initial submission to the registry

January 28, 2026

Completed
8 days until next milestone

First Posted

Study publicly available on registry

February 5, 2026

Completed
1 day until next milestone

Study Start

First participant enrolled

February 6, 2026

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2028

Last Updated

February 20, 2026

Status Verified

February 1, 2026

Enrollment Period

2 years

First QC Date

January 28, 2026

Last Update Submit

February 18, 2026

Conditions

AchondroplasiaHypochondroplasia

Outcome Measures

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)

    12 months

Secondary Outcomes (8)

  • number of participants reaching 180 hours of recording during the recording periods defined in the protocol

    12 months

  • French System Usability Score (F-SUS) score at 6 months of Syde® use

    6 months

  • Correlation between digital endpoints derived from Syde® and 6-minutes walk test (6MWT)

    12 months

  • Correlation between digital endpoints derived from Syde® and growth parameters

    12 months

  • Correlation between digital endpoints derived from Syde® and Patient-reported Outcomes (PROs)

    12 months

  • +3 more secondary outcomes

Eligibility Criteria

Age3 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Participants will be recruited from the patient registry of the Centre de Référence des Maladies Osseuses Constitutionnelles located at NeckerHospital / 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.

You may qualify if:

  • Aged 3 years old or older at the time of consent.
  • 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
  • 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

You may not qualify if:

  • 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.
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hopital Necker

Paris, 75015, France

RECRUITING

MeSH Terms

Conditions

AchondroplasiaHypochondroplasia

Condition Hierarchy (Ancestors)

DwarfismBone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesOsteochondrodysplasiasGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Genevieve Baujat

    Hopital Necker

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Ferial Toumi

CONTACT

+33 2 78 00 10 98ferial.toumi@sysnav.fr

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 28, 2026

First Posted

February 5, 2026

Study Start

February 6, 2026

Primary Completion (Estimated)

February 1, 2028

Study Completion (Estimated)

February 1, 2028

Last Updated

February 20, 2026

Record last verified: 2026-02

Locations

FR(1)