Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children

Phase 3

Completed

• Conditions: Down Syndrome
• Interventions: Drug: thyroid hormone and folinic acid

• Registration Number: NCT01576705
• Lead Sponsor: Institut Jerome Lejeune

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2012-04-02
• Study First Submitted: 2012-04-11
• Study First Submitted QC: 2012-04-11
• Study First Posted: 2012-04-12
• Primary Completion: 2017-12-14
• Study Completion: 2017-12-14
• Results First Submitted: 2019-10-09
• Status Verified: 2019-12
• Results First Submitted QC: 2020-04-30
• Last Update Submitted: 2020-04-30
• Results First Posted: 2020-05-13
• Last Update Posted: 2020-05-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 175

Inclusion Criteria

• 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

Exclusion Criteria

• 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)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Thyroxin + folinic acid	thyroid hormone and folinic acid	-
Thyroxin+folinic acid placebo	thyroid hormone and folinic acid	-
Thyroxin placebo+ folinic acid	thyroid hormone and folinic acid	-
Thyroxin placebo+ folinic acid placebo	thyroid hormone and folinic acid	-

Primary Outcome Measures

Name	Time	Method
GMDS (Griffiths Mental Development Scale)	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

Secondary Outcome Measures

Name	Time	Method
BL (Brunet Lezine Revised Scale)	12 months	BL includes 4 subscales : Locomotor, Coordination, Language, Sociability. Subscale and global developpemental quotients were computed by dividing the developpemental age by the chronological age multiplied by 100. This kind of formula do not give a min-max outcome. Higher QD's scores show a better psychomotor developpement outcome.

Trial Locations

Locations (1)

Institut Jerome Lejeune; 🇫🇷 Paris, France