Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children
NCT ID: NCT01576705
Last Updated: 2020-05-13
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
175 participants
INTERVENTIONAL
2012-04-02
2017-12-14
Brief Summary
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* 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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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Thyroxin + folinic acid
thyroid hormone and folinic acid
thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules
Thyroxin+folinic acid placebo
thyroid hormone and folinic acid
thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules
Thyroxin placebo+ folinic acid
thyroid hormone and folinic acid
thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules
Thyroxin placebo+ folinic acid placebo
thyroid hormone and folinic acid
thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules
Interventions
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thyroid hormone and folinic acid
thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules
Eligibility Criteria
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Inclusion Criteria
* patient having undergone a cardiac ultrasound not demonstrating any severe heart disease
* patient aged 6 to 18 months at inclusion
Exclusion Criteria
* 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)
6 Months
18 Months
ALL
No
Sponsors
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Hôpital Cochin
OTHER
Central Hospital, Nancy, France
OTHER
Institut Jerome Lejeune
OTHER
Responsible Party
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Principal Investigators
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Clotilde MIRCHER, MD
Role: PRINCIPAL_INVESTIGATOR
Institut Jerome Lejeune, Paris, France
Franck STURTZ, MD, PhD
Role: STUDY_CHAIR
Department of Biochemistry and Molecular Genetics, Limoges University, Limoges, France
Locations
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Institut Jerome Lejeune
Paris, , France
Countries
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References
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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. doi: 10.1089/thy.2006.16.671.
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. doi: 10.1210/jc.2005-0130. 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.
Sacco S, Bouis C, Gallard J, Pichot A, Blondiaux E, Marey I, Dorison N, Sturtz F, Cieuta-Walti C, Ravel A, Mircher C. Psychomotor development in infants and young children with Down syndrome-A prospective, repeated measure, post-hoc analysis. Am J Med Genet A. 2022 Mar;188(3):818-827. doi: 10.1002/ajmg.a.62587. Epub 2021 Dec 4.
Mircher C, Sacco S, Bouis C, Gallard J, Pichot A, Le Galloudec E, Cieuta C, Marey I, Greiner-Mahler O, Dorison N, Gambarini A, Stora S, Durand S, Polak M, Baruchel A, Schlumberger E, Dewailly J, Azar-Kolakez A, Gueant-Rodriguez RM, Gueant JL, Borderie D, Bonnefont-Rousselot D, Blondiaux E, Ravel A, Sturtz FG. Thyroid hormone and folinic acid in young children with Down syndrome: the phase 3 ACTHYF trial. Genet Med. 2020 Jan;22(1):44-52. doi: 10.1038/s41436-019-0597-8. Epub 2019 Jul 8.
Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Related Links
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Institut Jerome Lejeune
Other Identifiers
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IJL-AFHT-TH10
Identifier Type: -
Identifier Source: org_study_id
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