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Completed

NCT Number: NCT01576705

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

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.

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Key information

Conditions

Down Syndrome Abnormalities, Multiple Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations

Age range

6 month–18 month

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Institut Jerome Lejeune

Paris, 75015, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

thyroid hormone and folinic acid

Drug

thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules

Primary outcomes

  1. GMDS (Griffiths Mental Development Scale)

    Time frame: 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 outcomes

  1. BL (Brunet Lezine Revised Scale)

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Institut Jerome Lejeune

Other

Collaborators

  • Central Hospital, Nancy, France
  • Hôpital Cochin

Registry information

Acronym: ACTHYF

Important dates

Study start
2012
Primary completion
2017
Study completion
2017
First posted
Apr 12, 2012
Registry last updated
May 13, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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