2024-514852-34-01招募中3 期
Prospective pilot study of L-dopa treatment in patients with a neurodevelopmental disorder linked to a pathogenic variant of the CTNNB1 gene
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 7
- 试验地点
- 1
- 主要终点
- GMFM-88 motor score before treatment (Day 1) and at 6 months
研究概览
简要总结
Evaluate the effect of treatment on patients' motor skills by assessing their motor skills before and after treatment with the 88-item Gross Motor Function Measure (GMFM-88).
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Assessment Of Physical And Cognitive Abilities
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Ages 1 to 15 inclusive
- •Carrier of a pathogenic variant of CTNNB1.
排除标准
- •Contraindication to treatment with L-dopa and carbidopa or one of its excipients
- •Current treatment with L-dopa, dopamine agonist or dopamine blocker,
- •Failure to obtain informed consent signed by both parents or legal guardians, and to obtain the child's assent if possible.
- •Patient not enrolled in or benefiting from a social security scheme
- •Person participating in another research project with an exclusion period still in progress
结局指标
主要结局
GMFM-88 motor score before treatment (Day 1) and at 6 months
GMFM-88 motor score before treatment (Day 1) and at 6 months
次要结局
- GMFM-88 motor score before treatment (Day 1) and at 12 months
- Changes in language/development/cognition assessment scores (Bayley III, WPPSI, WISC, Vineland), before treatment and at 1 year after treatment
- Changes in quality-of-life scores (Caregiver Priorities & Child Health Index of Life with Disabilities (CP-CHILD), Clinical Global Impression of severity scale (CGI)) before treatment, at 6 months and at 1 year after initiation of treatment
- Tolerance assessment (number and severity of adverse events, clinical follow-up, clinical, neurological and osteoarticular examination, patient logbook).
- GMFM-88 motor score between 6 and 12 months of treatment
研究者
Agathe ROUBERTIE
Scientific
Centre Hospitalier Universitaire De Montpellier
研究点 (1)
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