Efficacy assessment of systematic treatment with folinic acid and thyroid hormone on psychomotor development of Down Syndrome young children - ACTHYF

Phase 1

• Conditions: Down syndrome; MedDRA version: 14.0Level: PTClassification code 10044688Term: Trisomy 21System Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 14.0Level: LLTClassification code 10013616Term: Down's syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2010-021134-66-FR
• Lead Sponsor: Institut Jerome Lejeune

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2011-07-04
• Study Completion: 2018-05-31
• Last Update Posted: 30 November 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 175

Inclusion Criteria

-Patient with a caryotype of a homogenous trisomy 21, free or with or robertsonian translocation,
-Patient with echocardiography showing no evidence of cardiopathy or valular leak,
-Patient aged 6 to 18 months at inclusion,

Are the trial subjects under 18? yes
Number of subjects for this age range: 256
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

-patient with current or past hypothyroidy
-patient with current or past TSH> 5mUI/l,
-leucémoid reaction at birth
-past or present leukemia, West Syndrome, seizure or any other neurological instable disorder,
-past or present signs of CNS damage : stroke, hypoxia, meningitis,
-past or present severe cardiac disease or significant valvular leak at echocardiography,
- uncontrolled cardiac rythm abnormalities,
-Apgar < 7 at 5 mn of birth,
- gestationnal age < 245 jours (35 semaines),

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Psychomotor development efficacy assessment of two medications on Down's syndrome young children :<br>- systematic thyroxin treatment with clinically and biologically controlled dosing,<br>- systematic folinic acid treatment,<br>- interaction assessment between the two medications;Secondary Objective: Identify genetic and biochemical items influencing treatment outcome;Primary end point(s): GQ of GMDS (Griffiths);Timepoint(s) of evaluation of this end point: 0, 6, 12 months

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Brunet Lézine scale;Timepoint(s) of evaluation of this end point: 0, 6, 12 months