Phase 3
N=175
Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children
Down Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01576705 ↗Enrolled (actual)
175
Serious AEs
17.2%
Results posted
May 2020
Primary outcome: Primary: GMDS (Griffiths Mental Development Scale) — 51.96; 51.27; 51.66; 51.67 developpement quotient
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- thyroid hormone and folinic acid (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Institut Jerome Lejeune
- Primary completion
- Dec 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY GMDS (Griffiths Mental Development Scale) |
51.96; 51.27; 51.66; 51.67 | — |
| SECONDARY BL (Brunet Lezine Revised Scale) |
51.18; 50.64; 51.24; 51.36 | — |
Summary
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.
Eligibility Criteria
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)
Data sourced from ClinicalTrials.gov (NCT01576705). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.