Ignite Creation Date:
2025-12-24 @ 6:59 PM
Ignite Modification Date:
2026-01-07 @ 11:10 AM
Study NCT ID:
NCT04734457
Status:
UNKNOWN
Last Update Posted:
2021-10-05
First Post:
2021-01-11
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Final Height in Patients With CH Diagnosed by the Screening
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003409', 'term': 'Congenital Hypothyroidism'}], 'ancestors': [{'id': 'D004392', 'term': 'Dwarfism'}, {'id': 'D001848', 'term': 'Bone Diseases, Developmental'}, {'id': 'D001847', 'term': 'Bone Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D001849', 'term': 'Bone Diseases, Endocrine'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}, {'id': 'D007037', 'term': 'Hypothyroidism'}, {'id': 'D013959', 'term': 'Thyroid Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D013974', 'term': 'Thyroxine'}, {'id': 'D015997', 'term': 'Neonatal Screening'}], 'ancestors': [{'id': 'D013963', 'term': 'Thyroid Hormones'}, {'id': 'D006728', 'term': 'Hormones'}, {'id': 'D006730', 'term': 'Hormones, Hormone Substitutes, and Hormone Antagonists'}, {'id': 'D024322', 'term': 'Amino Acids, Aromatic'}, {'id': 'D000598', 'term': 'Amino Acids, Cyclic'}, {'id': 'D000596', 'term': 'Amino Acids'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D019411', 'term': 'Clinical Laboratory Techniques'}, {'id': 'D019937', 'term': 'Diagnostic Techniques and Procedures'}, {'id': 'D003933', 'term': 'Diagnosis'}, {'id': 'D008403', 'term': 'Mass Screening'}, {'id': 'D008919', 'term': 'Investigative Techniques'}, {'id': 'D006306', 'term': 'Health Surveys'}, {'id': 'D011795', 'term': 'Surveys and Questionnaires'}, {'id': 'D003625', 'term': 'Data Collection'}, {'id': 'D004812', 'term': 'Epidemiologic Methods'}, {'id': 'D003954', 'term': 'Diagnostic Services'}, {'id': 'D011314', 'term': 'Preventive Health Services'}, {'id': 'D006296', 'term': 'Health Services'}, {'id': 'D005159', 'term': 'Health Care Facilities Workforce and Services'}, {'id': 'D017531', 'term': 'Health Care Evaluation Mechanisms'}, {'id': 'D011787', 'term': 'Quality of Health Care'}, {'id': 'D017530', 'term': 'Health Care Quality, Access, and Evaluation'}, {'id': 'D011634', 'term': 'Public Health'}, {'id': 'D004778', 'term': 'Environment and Public Health'}, {'id': 'D015980', 'term': 'Public Health Practice'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'RETROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 60}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2021-11-02', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-10', 'completionDateStruct': {'date': '2022-04-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2021-10-01', 'studyFirstSubmitDate': '2021-01-11', 'studyFirstSubmitQcDate': '2021-01-29', 'lastUpdatePostDateStruct': {'date': '2021-10-05', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-02-02', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-03-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Assessment of change of Final Height in patients with Congenital Hypothyroidism diagnosed by neonatal screening', 'timeFrame': 'Since the diagnosis is established by neonatal screening during first week of life height will be assessed every 6 months with follow up throughout the study period until the study subject reaches an average of 18 years of age( final height reached).', 'description': 'The investigators assess the effect of early diagnosis and early levothyroxine start on the change in longitudinal growth in patients with Congenital Hypothyroidism.'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Final Height', 'Neonatal screening'], 'conditions': ['Congenital Hypothyroidism']}, 'referencesModule': {'references': [{'pmid': '12218379', 'type': 'BACKGROUND', 'citation': 'Bain P, Toublanc JE. Adult height in congenital hypothyroidism: prognostic factors and the importance of compliance with treatment. Horm Res. 2002;58(3):136-42. doi: 10.1159/000064489.'}, {'pmid': '17932107', 'type': 'RESULT', 'citation': 'Bassett JH, Williams AJ, Murphy E, Boyde A, Howell PG, Swinhoe R, Archanco M, Flamant F, Samarut J, Costagliola S, Vassart G, Weiss RE, Refetoff S, Williams GR. A lack of thyroid hormones rather than excess thyrotropin causes abnormal skeletal development in hypothyroidism. Mol Endocrinol. 2008 Feb;22(2):501-12. doi: 10.1210/me.2007-0221. Epub 2007 Oct 11.'}, {'pmid': '31350727', 'type': 'RESULT', 'citation': 'Bauer AJ, Wassner AJ. Thyroid hormone therapy in congenital hypothyroidism and pediatric hypothyroidism. Endocrine. 2019 Oct;66(1):51-62. doi: 10.1007/s12020-019-02024-6. Epub 2019 Jul 26.'}, {'pmid': '7639544', 'type': 'RESULT', 'citation': 'Cole TJ, Freeman JV, Preece MA. Body mass index reference curves for the UK, 1990. Arch Dis Child. 1995 Jul;73(1):25-9. doi: 10.1136/adc.73.1.25.'}, {'pmid': '9497870', 'type': 'RESULT', 'citation': 'Dickerman Z, De Vries L. Prepubertal and pubertal growth, timing and duration of puberty and attained adult height in patients with congenital hypothyroidism (CH) detected by the neonatal screening programme for CH--a longitudinal study. Clin Endocrinol (Oxf). 1997 Dec;47(6):649-54. doi: 10.1046/j.1365-2265.1997.3181148.x.'}, {'pmid': '17433800', 'type': 'RESULT', 'citation': 'Huffmeier U, Tietze HU, Rauch A. Severe skeletal dysplasia caused by undiagnosed hypothyroidism. Eur J Med Genet. 2007 May-Jun;50(3):209-15. doi: 10.1016/j.ejmg.2007.02.002. Epub 2007 Mar 12.'}, {'pmid': '16205094', 'type': 'RESULT', 'citation': 'Nilsson O, Marino R, De Luca F, Phillip M, Baron J. Endocrine regulation of the growth plate. Horm Res. 2005;64(4):157-65. doi: 10.1159/000088791. Epub 2005 Oct 4.'}, {'pmid': '11580992', 'type': 'RESULT', 'citation': 'Salerno M, Micillo M, Di Maio S, Capalbo D, Ferri P, Lettiero T, Tenore A. Longitudinal growth, sexual maturation and final height in patients with congenital hypothyroidism detected by neonatal screening. Eur J Endocrinol. 2001 Oct;145(4):377-83. doi: 10.1530/eje.0.1450377.'}, {'pmid': '5491878', 'type': 'RESULT', 'citation': "Tanner JM, Goldstein H, Whitehouse RH. Standards for children's height at ages 2-9 years allowing for heights of parents. Arch Dis Child. 1970 Dec;45(244):755-62. doi: 10.1136/adc.45.244.755."}, {'pmid': '28548569', 'type': 'RESULT', 'citation': 'Whitney GA, Kean TJ, Fernandes RJ, Waldman S, Tse MY, Pang SC, Mansour JM, Dennis JE. Thyroxine Increases Collagen Type II Expression and Accumulation in Scaffold-Free Tissue-Engineered Articular Cartilage. Tissue Eng Part A. 2018 Mar;24(5-6):369-381. doi: 10.1089/ten.TEA.2016.0533. Epub 2017 Jul 7.'}, {'pmid': '25036648', 'type': 'RESULT', 'citation': 'Williams GR. Role of thyroid hormone receptor-alpha1 in endochondral ossification. Endocrinology. 2014 Aug;155(8):2747-50. doi: 10.1210/en.2014-1527. No abstract available.'}, {'pmid': '28853052', 'type': 'RESULT', 'citation': 'Williams GR, Bassett JHD. Thyroid diseases and bone health. J Endocrinol Invest. 2018 Jan;41(1):99-109. doi: 10.1007/s40618-017-0753-4. Epub 2017 Aug 29.'}]}, 'descriptionModule': {'briefSummary': 'The aim of this study is to evaluate longitudinal growth and final height in patients with Congenital Hypothyroidism detected by neonatal screening and factors affecting it.', 'detailedDescription': 'Linear growth and final height are reported as rather normal in patients with CH diagnosed through the screening and the target height is the most important factor determining linear growth. Data on final height in patients with CH suggest that adult height is significantly correlated with parental height and the mean L-Thyroxine daily dose administered over the first 6 months of treatment. The lowest recommended dose during this period is 8.5μg/kg/day. Periodical adjustments of L-Thyroxine daily dose should be guided by clinical observation and serum free T4 levels.\n\nTo our knowledge this is the first study from our center to assess final height in patients with congenital hypothyroidism diagnosed through the Egyptian neonatal screening program.\n\nA complex network of endocrine signals stimulates the process of longitudinal growth including growth hormone, insulin-like growth factor-1, glucocorticoids, thyroid hormone, estrogen, androgens, vitamin D and leptin.\n\nThyroid hormones are essential for development and normal bone growth. Biochemical studies have shown that thyroid hormones can affect the expression of various bone markers in the serum, reflecting changes in bone remodeling which involves both osteoblastic and osteoclastic activities.\n\nThyroid hormones act through chondrocytes bearing thyroid hormone receptors (TRs) to modulate growth plate proliferation, differentiation and vascular invasion. Several mechanisms mediate these functions including direct action on the chondrocytes, osteoblasts and mast cells. It also works through interaction with other hormones and growth factors acting in endocrine, paracrine and autocrine fashions.\n\nOverall, T3 acts via TRα in chondrocytes and osteoblasts to regulate intramembranous and endochondral ossification and control the rate of linear growth, bone maturation and mineralization,T4 induces the expression of both type II and X collagen and chondrocyte hypertrophy.\n\nHypothyroidism in children causes growth arrest, delayed bone maturation, and epiphyseal dysgenesis that can result in short stature and delayed closure of the epiphyses.\n\nIn severe undiagnosed cases, complete post-natal growth arrest and skeletal dysplasia occurs with characteristic X-ray features including stippled epiphyses reflecting epiphyseal dysgenesis, congenital hip dislocation, vertebral immaturity, scoliosis, patent fontanelles and sutures and delayed eruption of teeth.\n\nPrompt treatment of patients suffering from CH with thyroid hormone replacement induces a period of rapid catch-up growth in which skeletal maturation and bone age are also accelerated. Ultimately, normal adult height can be expected.\n\nIt is controversial whether factors as the degree of hypothyroidism at diagnosis, the timing of the onset of treatment and the doses of replacement L-thyroxine (L-T4) affect linear growth in children with congenital hypothyroidism, detected by screening and treated from early infancy'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': "All the patients suffering from CH diagnosed by Egyptian neonatal screening program and have a documented history of CH who received treatment at endocrinology clinic, Children's Hospital, Ain Shams University.", 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients having a documented history of CH diagnosed by Egyptian neonatal screening program.\n* Patients receiving L-thyroxine (L-T4) since the diagnosis is established.\n\nExclusion Criteria:\n\n* Patients who are non-compliant on L-thyroxine (L-T4) and failed to follow up at the clinic on regular basis.\n* Patients who were missed from diagnosis by neonatal screening.\n* Patients with associated hormonal disorders affecting same parameters concerned in the study.'}, 'identificationModule': {'nctId': 'NCT04734457', 'briefTitle': 'Final Height in Patients With CH Diagnosed by the Screening', 'organization': {'class': 'OTHER', 'fullName': 'Ain Shams University'}, 'officialTitle': 'Final Height in Patients With Congenital Hypothyroidism Diagnosed by Neonatal Screening', 'orgStudyIdInfo': {'id': 'Final height in CH patients'}}, 'armsInterventionsModule': {'interventions': [{'name': 'Levothyroxin', 'type': 'DRUG', 'otherNames': ['neonatal screening'], 'description': 'We assess the effect of early diagnosis of CH by neonatal screening and start of treatment on the final height of the patients'}]}, 'contactsLocationsModule': {'locations': [{'zip': '1181', 'city': 'Cairo', 'state': 'Abbasiya', 'country': 'Egypt', 'facility': 'Ain Shams University', 'geoPoint': {'lat': 30.06263, 'lon': 31.24967}}], 'centralContacts': [{'name': 'Mohamed Nader, M.B., B.Ch.', 'role': 'CONTACT', 'email': 'moh.nader90@gmail.com', 'phone': '+201014397938'}], 'overallOfficials': [{'name': 'Wessam Mouharram, PhD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Ain Shams University'}, {'name': 'Rana Mahmoud, PhD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Ain Shams University'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ain Shams University', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Principal Investigator', 'investigatorFullName': 'Mohamed Nader', 'investigatorAffiliation': 'Ain Shams University'}}}}