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

Down Syndrome research study

What is the primary objective of this study?

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.

Who is eligible to participate?

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)

Which medical condition, disease, disorder, syndrome, illness, or injury is researched?

Down Syndrome

Study Interventions

Interventions can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available or noninvasive approaches such as surveys, education, and interviews.

Drug:thyroid hormone and folinic acidthyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules

Study Arms

Research studies and clinical trials typically have two or more research arms. An arm is a group of people who receive the same treatment in the study.

Thyroxin + folinic acid

Thyroxin+folinic acid placebo

Thyroxin placebo+ folinic acid

Thyroxin placebo+ folinic acid placebo

Study Status

Completed

Start Date: April 2012

Completed Date: December 14, 2017

Phase: Phase 3

Type: Interventional

Design:

Primary Outcome: GMDS ( Griffiths Mental Development Scale)

Secondary Outcome: BL (Brunet Lezine revised scale)

Study sponsors, principal investigator, and references

Principal Investigator: Clotilde MIRCHER, MD

Lead Sponsor: Institut Jerome Lejeune

Collaborator: Ascopharm (CRO)

More information:https://clinicaltrials.gov/show/NCT01576705

Blehaut H, Mircher C, Ravel A, Conte M, de Portzamparc V, Poret G, de Kermadec FH, Rethore MO, Sturtz FG. Effect of leucovorin (folinic acid) on the developmental quotient of children with Down's syndrome (trisomy 21) and influence of thyroid status. PLoS One. 2010 Jan 11;5(1):e8394. doi: 10.1371/journal.pone.0008394.

Discuss Levothyroid