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

This study is currently recruiting participants. (see Contacts and Locations)
Verified December 2012 by Institut Jerome Lejeune
Sponsor:
Collaborators:
Ascopharm Groupe Novasco
Effi-stat
Medi.Axe
LC2
BIOMNIS
Information provided by (Responsible Party):
Institut Jerome Lejeune
ClinicalTrials.gov Identifier:
NCT01576705
First received: April 11, 2012
Last updated: December 10, 2012
Last verified: December 2012
  Purpose

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.

Condition Intervention Phase
Down Syndrome
Drug: thyroid hormone and folinic acid
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children

Resource links provided by NLM:


Further study details as provided by Institut Jerome Lejeune:

Primary Outcome Measures:
  • GMDS ( Griffiths Mental Development Scale) [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • BL (Brunet Lezine revised scale) [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 256
Study Start Date: April 2012
Estimated Study Completion Date: October 2017
Estimated Primary Completion Date: May 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   6 Months to 18 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  • 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 < 245 days (35 weeks)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01576705

Contacts
Contact: Yann GRATTAU, MD +3315658 ext 6325 yann.grattau@institutlejeune.org
Contact: Clotilde MIRCHER, MD +3315658 ext 6331 clotilde.mircher@institutlejeune.org

Locations
France
Institut Jerome Lejeune Recruiting
Paris, France, 75015
Contact: Clotilde MIRCHER, MD    +3315658 ext 6300    clotilde.mircher@institutlejeune.org   
Contact: Aime RAVEL, MD    +3315658 ext 6300    aime.ravel@institutlejeune.org   
Principal Investigator: Clotilde MIRCHER, MD         
Sub-Investigator: Aime RAVEL, MD         
Sub-Investigator: Nathalie DORISON, MD         
Sub-Investigator: Oliver GREINER-MAHLER, MD         
Sub-Investigator: Marie-Odile RETHORE, MD         
Sponsors and Collaborators
Institut Jerome Lejeune
Ascopharm Groupe Novasco
Effi-stat
Medi.Axe
LC2
BIOMNIS
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
Study Director: Yann GRATTAU, MD Institut Jerome Lejeune, Paris, France
  More Information

Additional Information:
Publications:
Responsible Party: Institut Jerome Lejeune
ClinicalTrials.gov Identifier: NCT01576705     History of Changes
Other Study ID Numbers: IJL-AFHT-TH10
Study First Received: April 11, 2012
Last Updated: December 10, 2012
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by Institut Jerome Lejeune:
Down syndrome
Young children
Psychomotor development
Thyroid hormone
Folinic acid
Psychometric tests
GMDS
Griffiths
Genetic factors
Biochemical factors

Additional relevant MeSH terms:
Down Syndrome
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Hormones
Leucovorin
Folic Acid
Levoleucovorin
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions
Vitamin B Complex
Vitamins
Micronutrients
Growth Substances
Antidotes
Protective Agents
Hematinics
Hematologic Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on July 24, 2014