Ignite Creation Date:
2025-12-25 @ 3:30 AM
Ignite Modification Date:
2026-03-06 @ 1:48 PM
Study NCT ID:
NCT01576705
Status:
COMPLETED
Last Update Posted:
2020-05-13
First Post:
2012-04-11
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D004314', 'term': 'Down Syndrome'}], 'ancestors': [{'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D025063', 'term': 'Chromosome Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D013963', 'term': 'Thyroid Hormones'}, {'id': 'D002955', 'term': 'Leucovorin'}], 'ancestors': [{'id': 'D006728', 'term': 'Hormones'}, {'id': 'D006730', 'term': 'Hormones, Hormone Substitutes, and Hormone Antagonists'}, {'id': 'D005575', 'term': 'Formyltetrahydrofolates'}, {'id': 'D013763', 'term': 'Tetrahydrofolates'}, {'id': 'D005492', 'term': 'Folic Acid'}, {'id': 'D011622', 'term': 'Pterins'}, {'id': 'D011621', 'term': 'Pteridines'}, {'id': 'D006574', 'term': 'Heterocyclic Compounds, 2-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D003067', 'term': 'Coenzymes'}, {'id': 'D045762', 'term': 'Enzymes and Coenzymes'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clotilde.mircher@institutlejeune.org', 'phone': '00 33 1 56 58 63 00', 'title': 'Dr Clotilde Mircher', 'organization': 'Institut Jérôme Lejeune'}, 'certainAgreement': {'piSponsorEmployee': True}, 'limitationsAndCaveats': {'description': 'The initial protocol planned for 256 patients, however due to difficulties in recruiting patients, the sample size was subsequently reduced to 175 patients, to obtain 140 evaluable patients.'}}, 'adverseEventsModule': {'timeFrame': '12 months', 'description': 'safety analysis were performed on all randomised patients receiving at least one dose of study treatment Safety population correspond to 174 subjects', 'eventGroups': [{'id': 'EG000', 'title': 'Thyroxin+Folinic Acid Placebo', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules', 'otherNumAtRisk': 44, 'deathsNumAtRisk': 44, 'otherNumAffected': 0, 'seriousNumAtRisk': 44, 'deathsNumAffected': 0, 'seriousNumAffected': 9}, {'id': 'EG001', 'title': 'Thyroxin + Folinic Acid', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules', 'otherNumAtRisk': 43, 'deathsNumAtRisk': 43, 'otherNumAffected': 0, 'seriousNumAtRisk': 43, 'deathsNumAffected': 1, 'seriousNumAffected': 8}, {'id': 'EG002', 'title': 'Thyroxin Placebo+ Folinic Acid', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules', 'otherNumAtRisk': 43, 'deathsNumAtRisk': 43, 'otherNumAffected': 7, 'seriousNumAtRisk': 43, 'deathsNumAffected': 0, 'seriousNumAffected': 8}, {'id': 'EG003', 'title': 'Thyroxin Placebo+ Folinic Acid Placebo', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules', 'otherNumAtRisk': 44, 'deathsNumAtRisk': 44, 'otherNumAffected': 0, 'seriousNumAtRisk': 44, 'deathsNumAffected': 0, 'seriousNumAffected': 5}], 'otherEvents': [{'term': 'Endocrine disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 43, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG003', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 20.1'}, {'term': 'Neoplasms begnin, malignant and unspecified', 'stats': [{'groupId': 'EG000', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 20.1'}, {'term': 'Nervous system disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 20.1'}], 'seriousEvents': [{'term': 'Infections and infestations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 44, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 43, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 43, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 44, 'numEvents': 7, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 20.1'}, {'term': 'Respiratory, thoracic and mediastinal disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 44, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 43, 'numEvents': 6, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 43, 'numEvents': 5, 'numAffected': 3}, {'groupId': 'EG003', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 20.1'}, {'term': 'General disorders and administration site conditions', 'stats': [{'groupId': 'EG000', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 20.1'}, {'term': 'Metabolism and nutrition disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 44, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 43, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 43, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 44, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 20.1'}, {'term': 'Surgical and medical procedures', 'stats': [{'groupId': 'EG000', 'numAtRisk': 44, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 43, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 20.1'}, {'term': 'Congenital, familial and genetic disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 44, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 43, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 20.1'}, {'term': 'Injury, poisoning and procedural complications', 'stats': [{'groupId': 'EG000', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 20.1'}, {'term': 'Neoplasm begign, maligant and unspecified', 'stats': [{'groupId': 'EG000', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 20.1'}, {'term': 'Nervous system disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 20.1'}, {'term': 'Skin and subcutnaeous tissue disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 43, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 44, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 20.1'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'GMDS (Griffiths Mental Development Scale)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '35', 'groupId': 'OG001'}, {'value': '30', 'groupId': 'OG002'}, {'value': '41', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Thyroxin + Folinic Acid', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules'}, {'id': 'OG001', 'title': 'Thyroxin+Folinic Acid Placebo', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules'}, {'id': 'OG002', 'title': 'Thyroxin Placebo+ Folinic Acid', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules'}, {'id': 'OG003', 'title': 'Thyroxin Placebo+ Folinic Acid Placebo', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules'}], 'classes': [{'categories': [{'measurements': [{'value': '51.96', 'spread': '7.88', 'groupId': 'OG000'}, {'value': '51.27', 'spread': '6.48', 'groupId': 'OG001'}, {'value': '51.66', 'spread': '6.37', 'groupId': 'OG002'}, {'value': '51.67', 'spread': '8.29', 'groupId': 'OG003'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 months', 'description': "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", 'unitOfMeasure': 'developpement quotient', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The main analysis of the primary efficacy endpoint is the adjusted change from baseline in GDQ derived from the Griffith Mental Development Scales (GMDS) at 12 months analysed by ANCOVA adjusted for covariates. Expected Outcome : six points difference in GDQ between any of the three treatment group compared to placebo at 12 months from baseline.'}, {'type': 'SECONDARY', 'title': 'BL (Brunet Lezine Revised Scale)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '35', 'groupId': 'OG001'}, {'value': '30', 'groupId': 'OG002'}, {'value': '40', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Thyroxin + Folinic Acid', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules'}, {'id': 'OG001', 'title': 'Thyroxin+Folinic Acid Placebo', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules'}, {'id': 'OG002', 'title': 'Thyroxin Placebo+ Folinic Acid', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules'}, {'id': 'OG003', 'title': 'Thyroxin Placebo+ Folinic Acid Placebo', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules'}], 'classes': [{'categories': [{'measurements': [{'value': '51.18', 'spread': '6.98', 'groupId': 'OG000'}, {'value': '50.64', 'spread': '7.25', 'groupId': 'OG001'}, {'value': '51.24', 'spread': '6.46', 'groupId': 'OG002'}, {'value': '51.36', 'spread': '8.27', 'groupId': 'OG003'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 months', 'description': "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.", 'unitOfMeasure': 'developpement quotient', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The main analysis of the secondary efficacy endpoint of the adjusted change from baseline in the BL-R GDQ at 12 months, analysed by ANCOVA adjusted for covariates, is presented in the mITT population.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Thyroxin + Folinic Acid', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg in tablets folinic acid 5 mg in capsules'}, {'id': 'FG001', 'title': 'Thyroxin+Folinic Acid Placebo', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg in tablets folinic acid placebo in capsules'}, {'id': 'FG002', 'title': 'Thyroxin Placebo+ Folinic Acid', 'description': 'thyroid hormone and folinic acid: thyroid hormone placebo in tablets folinic acid 5 mg in capsules'}, {'id': 'FG003', 'title': 'Thyroxin Placebo+ Folinic Acid Placebo', 'description': 'thyroid hormone and folinic acid: thyroid hormone placebo in tablets folinic acid placebo in capsules'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '43'}, {'groupId': 'FG001', 'numSubjects': '45'}, {'groupId': 'FG002', 'numSubjects': '43'}, {'groupId': 'FG003', 'numSubjects': '44'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '40'}, {'groupId': 'FG001', 'numSubjects': '37'}, {'groupId': 'FG002', 'numSubjects': '38'}, {'groupId': 'FG003', 'numSubjects': '41'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '8'}, {'groupId': 'FG002', 'numSubjects': '5'}, {'groupId': 'FG003', 'numSubjects': '3'}]}], 'dropWithdraws': [{'type': 'TSH > 7 mIU/L at baseline', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '7'}, {'groupId': 'FG002', 'numSubjects': '5'}, {'groupId': 'FG003', 'numSubjects': '3'}]}, {'type': 'invalid ICF', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Recruitment period : 2012-2016 First inclusion : 02/04/2012 Last inclusion : 14/12/2016\n\nLocation : Institut Jérôme Lejeune', 'preAssignmentDetails': 'As recruitment was nationwide and patients were included at a single centre, screening and randomisation were performed on the same day to minimise visits to the centre. Eligibility of randomised patients for TSH levels per exclusion criterion #8 was thus confirmed retrospectively, and patients with high TSH levels were discontinued.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '41', 'groupId': 'BG003'}, {'value': '156', 'groupId': 'BG004'}]}], 'groups': [{'id': 'BG000', 'title': 'Thyroxin + Folinic Acid', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules'}, {'id': 'BG001', 'title': 'Thyroxin+Folinic Acid Placebo', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules'}, {'id': 'BG002', 'title': 'Thyroxin Placebo+ Folinic Acid', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules'}, {'id': 'BG003', 'title': 'Thyroxin Placebo+ Folinic Acid Placebo', 'description': 'thyroid hormone and folinic acid: thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules'}, {'id': 'BG004', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Customized', 'classes': [{'title': 'Age', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '41', 'groupId': 'BG003'}, {'value': '156', 'groupId': 'BG004'}]}], 'categories': [{'measurements': [{'value': '12.3', 'spread': '3.4', 'groupId': 'BG000'}, {'value': '12.2', 'spread': '3.8', 'groupId': 'BG001'}, {'value': '12.3', 'spread': '4', 'groupId': 'BG002'}, {'value': '12.7', 'spread': '3.3', 'groupId': 'BG003'}, {'value': '12.37', 'spread': '3.58', 'groupId': 'BG004'}]}]}], 'paramType': 'MEAN', 'description': 'Age at inclusion in months', 'unitOfMeasure': 'months', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '41', 'groupId': 'BG003'}, {'value': '156', 'groupId': 'BG004'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '17', 'groupId': 'BG000'}, {'value': '16', 'groupId': 'BG001'}, {'value': '17', 'groupId': 'BG002'}, {'value': '17', 'groupId': 'BG003'}, {'value': '67', 'groupId': 'BG004'}]}, {'title': 'Male', 'measurements': [{'value': '23', 'groupId': 'BG000'}, {'value': '21', 'groupId': 'BG001'}, {'value': '21', 'groupId': 'BG002'}, {'value': '24', 'groupId': 'BG003'}, {'value': '89', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Male or Female', 'unitOfMeasure': 'Participants'}, {'title': 'Race and Ethnicity Not Collected', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Race and Ethnicity were not collected from any participant.'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'France', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '41', 'groupId': 'BG003'}, {'value': '156', 'groupId': 'BG004'}]}], 'categories': [{'measurements': [{'value': '40', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '41', 'groupId': 'BG003'}, {'value': '156', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': "Patient's height at baseline", 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '41', 'groupId': 'BG003'}, {'value': '156', 'groupId': 'BG004'}]}], 'categories': [{'measurements': [{'value': '70.86', 'spread': '3.88', 'groupId': 'BG000'}, {'value': '71.14', 'spread': '5.03', 'groupId': 'BG001'}, {'value': '71.13', 'spread': '4.15', 'groupId': 'BG002'}, {'value': '71.68', 'spread': '4.36', 'groupId': 'BG003'}, {'value': '71.21', 'spread': '4.33', 'groupId': 'BG004'}]}]}], 'paramType': 'MEAN', 'description': 'Height measured in cm', 'unitOfMeasure': 'cm', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Karyotype %', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '41', 'groupId': 'BG003'}, {'value': '156', 'groupId': 'BG004'}]}], 'categories': [{'title': 'Free trisomy 21', 'measurements': [{'value': '39', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '40', 'groupId': 'BG003'}, {'value': '154', 'groupId': 'BG004'}]}, {'title': 'Robertsonian translocation', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}, {'value': '2', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Pregnancy term', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '41', 'groupId': 'BG003'}, {'value': '156', 'groupId': 'BG004'}]}], 'categories': [{'measurements': [{'value': '38.03', 'spread': '1.37', 'groupId': 'BG000'}, {'value': '38.14', 'spread': '0.98', 'groupId': 'BG001'}, {'value': '38.29', 'spread': '1.33', 'groupId': 'BG002'}, {'value': '37.85', 'spread': '0.99', 'groupId': 'BG003'}, {'value': '38.07', 'spread': '1.18', 'groupId': 'BG004'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'weeks of amenorrhea', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Cardiac abnormalities', 'classes': [{'title': 'Isolated hear murmur', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '41', 'groupId': 'BG003'}, {'value': '156', 'groupId': 'BG004'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}, {'value': '7', 'groupId': 'BG004'}]}]}, {'title': 'Atrioventricular septal defect (AVSD)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '41', 'groupId': 'BG003'}, {'value': '156', 'groupId': 'BG004'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}, {'value': '6', 'groupId': 'BG004'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'TSH', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '41', 'groupId': 'BG003'}, {'value': '156', 'groupId': 'BG004'}]}], 'categories': [{'measurements': [{'value': '4.3', 'spread': '1.7', 'groupId': 'BG000'}, {'value': '4.32', 'spread': '1.52', 'groupId': 'BG001'}, {'value': '4.79', 'spread': '1.23', 'groupId': 'BG002'}, {'value': '4.45', 'spread': '1.49', 'groupId': 'BG003'}, {'value': '4.46', 'spread': '1.5', 'groupId': 'BG004'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'mIU/L', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': "Patient's weight at baseline", 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '41', 'groupId': 'BG003'}, {'value': '156', 'groupId': 'BG004'}]}], 'categories': [{'measurements': [{'value': '8.30', 'spread': '1.2', 'groupId': 'BG000'}, {'value': '8.19', 'spread': '1.42', 'groupId': 'BG001'}, {'value': '8.54', 'spread': '1.13', 'groupId': 'BG002'}, {'value': '8.62', 'spread': '1.6', 'groupId': 'BG003'}, {'value': '8.42', 'spread': '1.35', 'groupId': 'BG004'}]}]}], 'paramType': 'MEAN', 'description': 'Weight measured in kg', 'unitOfMeasure': 'Kg', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': "Patient's head circumference at baseline", 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '41', 'groupId': 'BG003'}, {'value': '156', 'groupId': 'BG004'}]}], 'categories': [{'measurements': [{'value': '43.5', 'spread': '1.59', 'groupId': 'BG000'}, {'value': '43.55', 'spread': '1.64', 'groupId': 'BG001'}, {'value': '43.63', 'spread': '1.55', 'groupId': 'BG002'}, {'value': '43.82', 'spread': '1.68', 'groupId': 'BG003'}, {'value': '43.63', 'spread': '1.6', 'groupId': 'BG004'}]}]}], 'paramType': 'MEAN', 'description': 'Head circumference measured in cm', 'unitOfMeasure': 'cm', 'dispersionType': 'STANDARD_DEVIATION'}], 'populationDescription': 'The modified intention-to-treat (mITT) population included all randomized patients who did not prematurely discontinue due to elevated baseline thyroid stimulating hormone (TSH)'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-11-07', 'size': 1441109, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2019-11-05T10:46', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 175}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2012-04-02', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-12', 'completionDateStruct': {'date': '2017-12-14', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-04-30', 'studyFirstSubmitDate': '2012-04-11', 'resultsFirstSubmitDate': '2019-10-09', 'studyFirstSubmitQcDate': '2012-04-11', 'lastUpdatePostDateStruct': {'date': '2020-05-13', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-04-30', 'studyFirstPostDateStruct': {'date': '2012-04-12', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-05-13', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2017-12-14', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'GMDS (Griffiths Mental Development Scale)', 'timeFrame': '12 months', 'description': "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"}], 'secondaryOutcomes': [{'measure': 'BL (Brunet Lezine Revised Scale)', 'timeFrame': '12 months', 'description': "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."}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Down syndrome', 'Young children', 'Psychomotor development', 'Thyroid hormone', 'Folinic acid', 'Psychometric tests', 'GMDS', 'Griffiths', 'Genetic factors', 'Biochemical factors'], 'conditions': ['Down Syndrome']}, 'referencesModule': {'references': [{'pmid': '20084109', 'type': 'BACKGROUND', 'citation': "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."}, {'pmid': '16889491', 'type': 'BACKGROUND', 'citation': 'van Trotsenburg AS, Kempers MJ, Endert E, Tijssen JG, de Vijlder JJ, Vulsma T. Trisomy 21 causes persistent congenital hypothyroidism presumably of thyroidal origin. Thyroid. 2006 Jul;16(7):671-80. doi: 10.1089/thy.2006.16.671.'}, {'pmid': '15755847', 'type': 'BACKGROUND', 'citation': 'van Trotsenburg AS, Vulsma T, van Rozenburg-Marres SL, van Baar AL, Ridder JC, Heymans HS, Tijssen JG, de Vijlder JJ. The effect of thyroxine treatment started in the neonatal period on development and growth of two-year-old Down syndrome children: a randomized clinical trial. J Clin Endocrinol Metab. 2005 Jun;90(6):3304-11. doi: 10.1210/jc.2005-0130. Epub 2005 Mar 8.'}, {'pmid': '18296460', 'type': 'BACKGROUND', 'citation': "Ellis JM, Tan HK, Gilbert RE, Muller DP, Henley W, Moy R, Pumphrey R, Ani C, Davies S, Edwards V, Green H, Salt A, Logan S. Supplementation with antioxidants and folinic acid for children with Down's syndrome: randomised controlled trial. BMJ. 2008 Mar 15;336(7644):594-7. doi: 10.1136/bmj.39465.544028.AE. Epub 2008 Feb 21."}, {'pmid': '34863019', 'type': 'DERIVED', 'citation': 'Sacco S, Bouis C, Gallard J, Pichot A, Blondiaux E, Marey I, Dorison N, Sturtz F, Cieuta-Walti C, Ravel A, Mircher C. Psychomotor development in infants and young children with Down syndrome-A prospective, repeated measure, post-hoc analysis. Am J Med Genet A. 2022 Mar;188(3):818-827. doi: 10.1002/ajmg.a.62587. Epub 2021 Dec 4.'}, {'pmid': '31281181', 'type': 'DERIVED', 'citation': 'Mircher C, Sacco S, Bouis C, Gallard J, Pichot A, Le Galloudec E, Cieuta C, Marey I, Greiner-Mahler O, Dorison N, Gambarini A, Stora S, Durand S, Polak M, Baruchel A, Schlumberger E, Dewailly J, Azar-Kolakez A, Gueant-Rodriguez RM, Gueant JL, Borderie D, Bonnefont-Rousselot D, Blondiaux E, Ravel A, Sturtz FG. Thyroid hormone and folinic acid in young children with Down syndrome: the phase 3 ACTHYF trial. Genet Med. 2020 Jan;22(1):44-52. doi: 10.1038/s41436-019-0597-8. Epub 2019 Jul 8.'}], 'seeAlsoLinks': [{'url': 'http://www.institutlejeune.org/', 'label': 'Institut Jerome Lejeune'}]}, 'descriptionModule': {'briefSummary': 'Evaluation of the following in very young children with Down syndrome:\n\n* the efficacy of systematic treatment with L-thyroxine at controlled doses (clinically and by ultrasensitive thyreostimulating hormone (TSH) assay),\n* the efficacy of systematic folinic acid treatment at a dose of 1 mg/kg/o.i.d,\n* any interaction between these two treatments.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '18 Months', 'minimumAge': '6 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* patient with a karyotype demonstrating homogeneous, free or Robertsonian translocation trisomy 21\n* patient having undergone a cardiac ultrasound not demonstrating any severe heart disease\n* patient aged 6 to 18 months at inclusion\n\nExclusion Criteria:\n\n* congenital hypothyroidism\n* hypothyroidism demonstrated by laboratory tests (TSH \\> 7mUI/l)\n* presenting or having presented hyperthyroidism\n* presenting or having presented leukaemia\n* presenting or having presented West syndrome or any other form of epilepsy or unstable neurological disease\n* presenting or having presented signs of central nervous system distress: stroke, postoperative hypoxia, meningitis)\n* presenting severe heart disease on cardiac ultrasound, with haemodynamic effects\n* presenting non-controlled cardiac arrhythmia\n* Apgar \\< 7 to 5 min at birth\n* Gestational age \\< 231 days (33 gestation weeks)'}, 'identificationModule': {'nctId': 'NCT01576705', 'acronym': 'ACTHYF', 'briefTitle': 'Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children', 'organization': {'class': 'OTHER', 'fullName': 'Institut Jerome Lejeune'}, 'officialTitle': 'Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children', 'orgStudyIdInfo': {'id': 'IJL-AFHT-TH10'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Thyroxin + folinic acid', 'interventionNames': ['Drug: thyroid hormone and folinic acid']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'Thyroxin+folinic acid placebo', 'interventionNames': ['Drug: thyroid hormone and folinic acid']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'Thyroxin placebo+ folinic acid', 'interventionNames': ['Drug: thyroid hormone and folinic acid']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Thyroxin placebo+ folinic acid placebo', 'interventionNames': ['Drug: thyroid hormone and folinic acid']}], 'interventions': [{'name': 'thyroid hormone and folinic acid', 'type': 'DRUG', 'description': 'thyroid hormone 25microg or placebo in tablets folinic acid 5 mg or placebo in capsules', 'armGroupLabels': ['Thyroxin + folinic acid', 'Thyroxin placebo+ folinic acid', 'Thyroxin placebo+ folinic acid placebo', 'Thyroxin+folinic acid placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '75015', 'city': 'Paris', 'country': 'France', 'facility': 'Institut Jerome Lejeune', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}], 'overallOfficials': [{'name': 'Clotilde MIRCHER, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Institut Jerome Lejeune, Paris, France'}, {'name': 'Franck STURTZ, MD, PhD', 'role': 'STUDY_CHAIR', 'affiliation': 'Department of Biochemistry and Molecular Genetics, Limoges University, Limoges, France'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Institut Jerome Lejeune', 'class': 'OTHER'}, 'collaborators': [{'name': 'Hôpital Cochin', 'class': 'OTHER'}, {'name': 'Central Hospital, Nancy, France', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}