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?
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
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
Start Date: April 2012
Completed Date: December 14, 2017
Phase: Phase 3
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)
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.