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

Information source: Institut Jerome Lejeune
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Down Syndrome

Intervention: thyroid hormone and folinic acid (Drug)

Phase: Phase 2/Phase 3

Status: Recruiting

Sponsored by: Institut Jerome Lejeune

Official(s) and/or principal investigator(s):
Clotilde MIRCHER, MD, Principal Investigator, Affiliation: Institut Jerome Lejeune, Paris, France
Franck STURTZ, MD, PhD, Study Chair, Affiliation: Department of Biochemistry and Molecular Genetics, Limoges University, Limoges, France
Yann GRATTAU, MD, Study Director, Affiliation: Institut Jerome Lejeune, Paris, France

Overall contact:
Yann GRATTAU, MD, Phone: +3315658, Ext: 6325, Email: yann.grattau@institutlejeune.org


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.

Clinical Details

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

Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Primary outcome: GMDS ( Griffiths Mental Development Scale)

Secondary outcome: BL (Brunet Lezine revised scale)


Minimum age: 6 Months. Maximum age: 18 Months. Gender(s): Both.


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


- patient aged 6 to 18 months at inclusion

Exclusion Criteria:

- congenital hypothyroidism

- hypothyroidism demonstrated by laboratory tests

- 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 < 245 days (35 weeks)

Locations and Contacts

Yann GRATTAU, MD, Phone: +3315658, Ext: 6325, Email: yann.grattau@institutlejeune.org

Institut Jerome Lejeune, Paris 75015, France; Recruiting
Clotilde MIRCHER, MD, Phone: +3315658, Ext: 6300, Email: clotilde.mircher@institutlejeune.org
Aime RAVEL, MD, Phone: +3315658, Ext: 6300, Email: aime.ravel@institutlejeune.org
Clotilde MIRCHER, MD, Principal Investigator
Aime RAVEL, MD, Sub-Investigator
Nathalie DORISON, MD, Sub-Investigator
Oliver GREINER-MAHLER, MD, Sub-Investigator
Marie-Odile RETHORE, MD, Sub-Investigator
Additional Information

Institut Jerome Lejeune

Related publications:

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.

van Trotsenburg AS, Kempers MJ, Endert E, Tijssen JG, de Vijlder JJ, Vulsma T. Trisomy 21 causes persistent congenital hypothyroidism presumably of thyroidal origin. Thyroid. 2006 Jul;16(7):671-80.

van Trotsenburg AS, Vulsma T, van Rozenburg-Marres SL, van Baar AL, Ridder JC, Heymans HS, Tijssen JG, de Vijlder JJ. The effect of thyroxine treatment started in the neonatal period on development and growth of two-year-old Down syndrome children: a randomized clinical trial. J Clin Endocrinol Metab. 2005 Jun;90(6):3304-11. Epub 2005 Mar 8.

Ellis JM, Tan HK, Gilbert RE, Muller DP, Henley W, Moy R, Pumphrey R, Ani C, Davies S, Edwards V, Green H, Salt A, Logan S. Supplementation with antioxidants and folinic acid for children with Down's syndrome: randomised controlled trial. BMJ. 2008 Mar 15;336(7644):594-7. doi: 10.1136/bmj.39465.544028.AE. Epub 2008 Feb 21.

Starting date: April 2012
Last updated: December 10, 2012

Page last updated: August 23, 2015

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