NCT07388966招募中不适用
Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia Using Syde®
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- SYSNAV
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Reliability of digital endpoint derived from Syde® assessed by the Intra-Class Correlation (ICC) for each recording period timepoint.
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 3 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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/m
- •Recent upper and/or lower limbs injury (trauma/fracture or surgery) in the 6 months preceding inclusion.
- •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.
结局指标
主要结局
Reliability of digital endpoint derived from Syde® assessed by the Intra-Class Correlation (ICC) for each recording period timepoint.
时间窗: 12 months
reliability assessed by the ICC (Intra-Class Correlation)for each recording period timepoint (baseline, 6 months, 12 months)
次要结局
- 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)
- Longitudinal Change in digital endpoints derived from Syde®(12 months)
- Syde® movement reconstruction accuracy compared to Optical Motion Capture measures.(Baseline)
- Qualitative interview exploring limitations on mobility and impact on daily life(baseline)
研究者
研究点 (1)
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