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

April 30, 2020 updated by: Institut Jerome Lejeune

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

175

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Paris, France, 75015
        • Institut Jerome Lejeune

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 1 year (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Thyroxin + folinic acid
Drug: thyroid hormone and folinic acid
thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules
Active Comparator: Thyroxin+folinic acid placebo
Drug: thyroid hormone and folinic acid
thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules
Active Comparator: Thyroxin placebo+ folinic acid
Drug: thyroid hormone and folinic acid
thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules
Placebo Comparator: Thyroxin placebo+ folinic acid placebo
Drug: thyroid hormone and folinic acid
thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
GMDS (Griffiths Mental Development Scale)
Time Frame: 12 months
GMDS for testing and estimate babies psychomotor development from birth to 2 years trough six subscales : Locomotor, Personal-social, Hearing and language, Eye-Hand coordination, Performance.Sub- and General Quotients (GDQ) standard score are based on a mean of 100 and a standard deviation of 16. For children with delayed development, which is the case for children with Down Syndrome, Quotient tables could be not used because sub- and General quotient floors at 50. For each subscale, a raw score was derived from the contributing items. Total raw score was obtained by adding subscale raw scores. Sum of all subscale raw scores was converted into a development age using correspondence table. Subscale and global development quotients were computed by dividing the development age by the chronological age multiplied by 100. For preterm infants, chronological age was corrected taking into account the gestational term. Higher QD's scores show a better psychomotor development outcome
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
BL (Brunet Lezine Revised Scale)
Time Frame: 12 months
BL includes 4 subscales : Locomotor, Coordination, Language, Sociability. Subscale and global developpemental quotients were computed by dividing the developpemental age by the chronological age multiplied by 100. This kind of formula do not give a min-max outcome. Higher QD's scores show a better psychomotor developpement outcome.
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Institut Jerome Lejeune

Collaborators

Central Hospital, Nancy, France
Hôpital Cochin

Investigators

  • Principal Investigator: Clotilde MIRCHER, MD, Institut Jerome Lejeune, Paris, France
  • Study Chair: Franck STURTZ, MD, PhD, Department of Biochemistry and Molecular Genetics, Limoges University, Limoges, France

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 2, 2012

Primary Completion (Actual)

December 14, 2017

Study Completion (Actual)

December 14, 2017

Study Registration Dates

First Submitted

April 11, 2012

First Submitted That Met QC Criteria

April 11, 2012

First Posted (Estimate)

April 12, 2012

Study Record Updates

Last Update Posted (Actual)

May 13, 2020

Last Update Submitted That Met QC Criteria

April 30, 2020

Last Verified

December 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IJL-AFHT-TH10

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Down Syndrome

Clinical Trials on thyroid hormone and folinic acid

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in United Kingdom

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe