Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 175
- 试验地点
- 1
- 主要终点
- GMDS (Griffiths Mental Development Scale)
研究概览
简要总结
Evaluation of the following in very young children with Down syndrome:
- the efficacy of systematic treatment with L-thyroxine at controlled doses (clinically and by ultrasensitive thyreostimulating hormone (TSH) assay),
- the efficacy of systematic folinic acid treatment at a dose of 1 mg/kg/o.i.d,
- any interaction between these two treatments.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Months 至 18 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •patient with a karyotype demonstrating homogeneous, free or Robertsonian translocation trisomy 21
- •patient having undergone a cardiac ultrasound not demonstrating any severe heart disease
- •patient aged 6 to 18 months at inclusion
排除标准
- •congenital hypothyroidism
- •hypothyroidism demonstrated by laboratory tests (TSH > 7mUI/l)
- •presenting or having presented hyperthyroidism
- •presenting or having presented leukaemia
- •presenting or having presented West syndrome or any other form of epilepsy or unstable neurological disease
- •presenting or having presented signs of central nervous system distress: stroke, postoperative hypoxia, meningitis)
- •presenting severe heart disease on cardiac ultrasound, with haemodynamic effects
- •presenting non-controlled cardiac arrhythmia
- •Apgar < 7 to 5 min at birth
- •Gestational age < 231 days (33 gestation weeks)
研究组 & 干预措施
Thyroxin + folinic acid
干预措施: thyroid hormone and folinic acid (Drug)
Thyroxin+folinic acid placebo
干预措施: thyroid hormone and folinic acid (Drug)
Thyroxin placebo+ folinic acid
干预措施: thyroid hormone and folinic acid (Drug)
Thyroxin placebo+ folinic acid placebo
干预措施: thyroid hormone and folinic acid (Drug)
结局指标
主要结局
GMDS (Griffiths Mental Development Scale)
时间窗: 12 months
GMDS for testing and estimate babies psychomotor development from birth to 2 years trough six subscales : Locomotor, Personal-social, Hearing and language, Eye-Hand coordination, Performance.Sub- and General Quotients (GDQ) standard score are based on a mean of 100 and a standard deviation of 16. For children with delayed development, which is the case for children with Down Syndrome, Quotient tables could be not used because sub- and General quotient floors at 50. For each subscale, a raw score was derived from the contributing items. Total raw score was obtained by adding subscale raw scores. Sum of all subscale raw scores was converted into a development age using correspondence table. Subscale and global development quotients were computed by dividing the development age by the chronological age multiplied by 100. For preterm infants, chronological age was corrected taking into account the gestational term. Higher QD's scores show a better psychomotor development outcome
次要结局
- BL (Brunet Lezine Revised Scale)(12 months)