Efficacy Study of Folinic Acid to Improve Mental Development of Children With Down Syndrome

Summary

The purpose of this study is to determine whether folinic acid can improve developmental quotient of young Down syndrome patients, given that these present signs of folate deficiency which are known to cause reversible neurological, psychiatric and cognitive disorders.

Timeline

Start

2000-10

Primary completion

—

Completion

2003-12

Outcome

Missed primary endpoint

paper The intent-to-treat analysis (113 patients) did not show a positive effect of leucovorin treatment. PMID 20084109 ↗

Publications

• Background Botez MI, Botez T, Leveille J, Bielmann P, Cadotte M. Neuropsychological correlates of folic acid deficiency: facts and hypotheses. In: Botez MI, Reynolds EH, eds. Folic acid in neurology, psychiatry and internal medicine. New York, Raven Press, 1979:435-461.
• Background Lejeune J. [Pathogenesis of mental impairment in trisomy 21]. Ann Genet. 1991;34(2):55-64. French.
• 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.

Drugs

Indications