Ignite Creation Date:
2025-12-25 @ 3:27 AM
Ignite Modification Date:
2026-04-09 @ 1:12 PM
Study NCT ID:
NCT00657605
Status:
COMPLETED
Last Update Posted:
2020-02-13
First Post:
2008-04-09
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Effects of Human Leptin Replacement
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009765', 'term': 'Obesity'}, {'id': 'D024821', 'term': 'Metabolic Syndrome'}, {'id': 'D003920', 'term': 'Diabetes Mellitus'}], 'ancestors': [{'id': 'D050177', 'term': 'Overweight'}, {'id': 'D044343', 'term': 'Overnutrition'}, {'id': 'D009748', 'term': 'Nutrition Disorders'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D001835', 'term': 'Body Weight'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D007333', 'term': 'Insulin Resistance'}, {'id': 'D006946', 'term': 'Hyperinsulinism'}, {'id': 'D044882', 'term': 'Glucose Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C122136', 'term': 'recombinant methionyl human leptin'}, {'id': 'C415771', 'term': 'metreleptin'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'y.p.davis@med.miami.edu', 'phone': '305-243-0494', 'title': 'Richard Bookman', 'organization': 'University of Miami'}, 'certainAgreement': {'piSponsorEmployee': True}, 'limitationsAndCaveats': {'description': 'This is a rare disease population study. To protect participant privacy and maintain confidentiality, results will not be reported.'}}, 'adverseEventsModule': {'timeFrame': '107 months', 'description': 'Adverse Events not reported due to participant confidentiality.', 'eventGroups': [{'id': 'EG000', 'title': 'Recombinant Methionyl Human Leptin', 'description': 'Participants with congenital leptin deficiency will receive the Recombinant methionyl human leptin intervention subcutaneously, once a day with a dose of 0.02 to 0.04 mg/kg (adjusted according to weight loss).', 'otherNumAtRisk': 0, 'deathsNumAtRisk': 3, 'otherNumAffected': 0, 'seriousNumAtRisk': 0, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change in Weight', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Recombinant Methionyl Human Leptin', 'description': 'Participants with congenital leptin deficiency will receive the Recombinant methionyl human leptin intervention subcutaneously, once a day with a dose of 0.02 to 0.04 mg/kg (adjusted according to weight loss).'}], 'classes': [{'categories': [{'measurements': [{'value': 'NA', 'comment': 'Results not reported due to participant confidentiality.', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline, 107 months', 'unitOfMeasure': 'lbs', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Glucose Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Recombinant Methionyl Human Leptin', 'description': 'Participants with congenital leptin deficiency will receive the Recombinant methionyl human leptin intervention subcutaneously, once a day with a dose of 0.02 to 0.04 mg/kg (adjusted according to weight loss).'}], 'classes': [{'categories': [{'measurements': [{'value': 'NA', 'comment': 'Results not reported due to participant confidentiality.', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline, 107 months', 'unitOfMeasure': 'mg/dL', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Recombinant Methionyl Human Leptin', 'description': 'Participants with congenital leptin deficiency will receive the Recombinant methionyl human leptin intervention subcutaneously, once a day with a dose of 0.02 to 0.04 mg/kg (adjusted according to weight loss).'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Recombinant Methionyl Human Leptin', 'description': 'Participants with congenital leptin deficiency will receive the Recombinant methionyl human leptin intervention subcutaneously, once a day with a dose of 0.02 to 0.04 mg/kg (adjusted according to weight loss).'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 3}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2001-06', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-02', 'completionDateStruct': {'date': '2010-05-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-02-11', 'studyFirstSubmitDate': '2008-04-09', 'resultsFirstSubmitDate': '2020-02-11', 'studyFirstSubmitQcDate': '2008-04-09', 'lastUpdatePostDateStruct': {'date': '2020-02-13', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-02-11', 'studyFirstPostDateStruct': {'date': '2008-04-14', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-02-13', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2010-05-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in Weight', 'timeFrame': 'Baseline, 107 months'}], 'secondaryOutcomes': [{'measure': 'Change in Glucose Levels', 'timeFrame': 'Baseline, 107 months'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Congenital leptin deficiency', 'Obesity', 'Metabolic syndrome', 'Diabetes'], 'conditions': ['Obesity', 'Metabolic Syndrome', 'Diabetes']}, 'referencesModule': {'references': [{'pmid': '9500540', 'type': 'BACKGROUND', 'citation': 'Strobel A, Issad T, Camoin L, Ozata M, Strosberg AD. A leptin missense mutation associated with hypogonadism and morbid obesity. Nat Genet. 1998 Mar;18(3):213-5. doi: 10.1038/ng0398-213. No abstract available.'}, {'pmid': '17986612', 'type': 'RESULT', 'citation': 'Baicy K, London ED, Monterosso J, Wong ML, Delibasi T, Sharma A, Licinio J. Leptin replacement alters brain response to food cues in genetically leptin-deficient adults. Proc Natl Acad Sci U S A. 2007 Nov 13;104(46):18276-9. doi: 10.1073/pnas.0706481104. Epub 2007 Nov 6.'}, {'pmid': '17684509', 'type': 'RESULT', 'citation': 'Licinio J, Ribeiro L, Busnello JV, Delibasi T, Thakur S, Elashoff RM, Sharma A, Jardack PM, Depaoli AM, Wong ML. Effects of leptin replacement on macro- and micronutrient preferences. Int J Obes (Lond). 2007 Dec;31(12):1859-63. doi: 10.1038/sj.ijo.0803703. Epub 2007 Aug 7.'}, {'pmid': '17517446', 'type': 'RESULT', 'citation': 'Licinio J, Milane M, Thakur S, Whelan F, Yildiz BO, Delibasi T, de Miranda PB, Ozata M, Bolu E, Depaoli A, Wong ML. Effects of leptin on intake of specific micro- and macronutrients in a woman with leptin gene deficiency studied off and on leptin at stable body weight. Appetite. 2007 Nov;49(3):594-9. doi: 10.1016/j.appet.2007.03.228. Epub 2007 Apr 6.'}, {'pmid': '15949871', 'type': 'RESULT', 'citation': 'Williamson DA, Ravussin E, Wong ML, Wagner A, Dipaoli A, Caglayan S, Ozata M, Martin C, Walden H, Arnett C, Licinio J. Microanalysis of eating behavior of three leptin deficient adults treated with leptin therapy. Appetite. 2005 Aug;45(1):75-80. doi: 10.1016/j.appet.2005.01.002.'}, {'pmid': '15713712', 'type': 'RESULT', 'citation': 'Matochik JA, London ED, Yildiz BO, Ozata M, Caglayan S, DePaoli AM, Wong ML, Licinio J. Effect of leptin replacement on brain structure in genetically leptin-deficient adults. J Clin Endocrinol Metab. 2005 May;90(5):2851-4. doi: 10.1210/jc.2004-1979. Epub 2005 Feb 15.'}, {'pmid': '15070752', 'type': 'RESULT', 'citation': "Licinio J, Caglayan S, Ozata M, Yildiz BO, de Miranda PB, O'Kirwan F, Whitby R, Liang L, Cohen P, Bhasin S, Krauss RM, Veldhuis JD, Wagner AJ, DePaoli AM, McCann SM, Wong ML. Phenotypic effects of leptin replacement on morbid obesity, diabetes mellitus, hypogonadism, and behavior in leptin-deficient adults. Proc Natl Acad Sci U S A. 2004 Mar 30;101(13):4531-6. doi: 10.1073/pnas.0308767101. Epub 2004 Mar 9."}, {'pmid': '11443202', 'type': 'RESULT', 'citation': 'Mantzoros CS, Ozata M, Negrao AB, Suchard MA, Ziotopoulou M, Caglayan S, Elashoff RM, Cogswell RJ, Negro P, Liberty V, Wong ML, Veldhuis J, Ozdemir IC, Gold PW, Flier JS, Licinio J. Synchronicity of frequently sampled thyrotropin (TSH) and leptin concentrations in healthy adults and leptin-deficient subjects: evidence for possible partial TSH regulation by leptin in humans. J Clin Endocrinol Metab. 2001 Jul;86(7):3284-91. doi: 10.1210/jcem.86.7.7644.'}, {'pmid': '10523015', 'type': 'RESULT', 'citation': 'Ozata M, Ozdemir IC, Licinio J. Human leptin deficiency caused by a missense mutation: multiple endocrine defects, decreased sympathetic tone, and immune system dysfunction indicate new targets for leptin action, greater central than peripheral resistance to the effects of leptin, and spontaneous correction of leptin-mediated defects. J Clin Endocrinol Metab. 1999 Oct;84(10):3686-95. doi: 10.1210/jcem.84.10.5999.'}, {'pmid': '21070531', 'type': 'DERIVED', 'citation': 'Paz-Filho G, Wong ML, Licinio J. The procognitive effects of leptin in the brain and their clinical implications. Int J Clin Pract. 2010 Dec;64(13):1808-12. doi: 10.1111/j.1742-1241.2010.02536.x.'}, {'pmid': '19889232', 'type': 'DERIVED', 'citation': 'Paz-Filho G, Delibasi T, Erol HK, Wong ML, Licinio J. Congenital leptin deficiency and thyroid function. Thyroid Res. 2009 Nov 4;2(1):11. doi: 10.1186/1756-6614-2-11.'}, {'pmid': '18854428', 'type': 'DERIVED', 'citation': 'Paz-Filho G, Esposito K, Hurwitz B, Sharma A, Dong C, Andreev V, Delibasi T, Erol H, Ayala A, Wong ML, Licinio J. Changes in insulin sensitivity during leptin replacement therapy in leptin-deficient patients. Am J Physiol Endocrinol Metab. 2008 Dec;295(6):E1401-8. doi: 10.1152/ajpendo.90450.2008. Epub 2008 Oct 14.'}, {'pmid': '18726828', 'type': 'DERIVED', 'citation': 'Andreev VP, Paz-Filho G, Wong ML, Licinio J. Deconvolution of insulin secretion, insulin hepatic extraction post-hepatic delivery rates and sensitivity during 24-hour standardized meals: time course of glucose homeostasis in leptin replacement treatment. Horm Metab Res. 2009 Feb;41(2):142-51. doi: 10.1055/s-0028-1082048. Epub 2008 Aug 22.'}]}, 'descriptionModule': {'briefSummary': 'This study will test the hypothesis that leptin contributes to the regulation of the dynamics of human endocrine function.', 'detailedDescription': 'The study aims to elucidate the role of leptin in the regulation of human endocrine function approached by a carefully designed, prospective clinical study of the rapidly-sampled dynamics of endocrine rhythms during the course of leptin-replacement treatment in the only three adult individuals identified in the world so far who are leptin-naive due to a functional leptin gene mutation. A study of leptin-naive subjects avoids all confounding factors and pitfalls, because the only bioactive leptin to which they will be exposed is the exogenously administered recombinant protein. Thus, this proposal will permit us to ascertain the endocrine effects of human leptin.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Congenital leptin deficiency (these are only 3 adult individuals in the world that have been identified as leptin-naïve thus far).\n\nExclusion Criteria:\n\n* Pregnant, trying to become pregnant, breast-feeding an infant or sexually active women, not using contraception.\n* Subjects with hemoglobin levels below 12 g/dl.\n* Subjects whose body contains a ferromagnetic implanted device that might produce a safety hazard during fMRI.'}, 'identificationModule': {'nctId': 'NCT00657605', 'briefTitle': 'Effects of Human Leptin Replacement', 'organization': {'class': 'OTHER', 'fullName': 'University of Miami'}, 'officialTitle': 'Effects of Human Leptin Replacement', 'orgStudyIdInfo': {'id': '20060282'}, 'secondaryIdInfos': [{'id': '1R0IDK58851-01', 'type': 'OTHER', 'domain': 'National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Recombinant methionyl human leptin', 'description': 'Participants with congenital leptin deficiency will receive the Recombinant methionyl human leptin intervention subcutaneously, once a day with a dose of 0.02 to 0.04 mg/kg (adjusted according to weight loss).', 'interventionNames': ['Drug: Recombinant methionyl human leptin']}], 'interventions': [{'name': 'Recombinant methionyl human leptin', 'type': 'DRUG', 'otherNames': ['Metreleptin', 'r-metHuLeptin'], 'description': 'Recombinant methionyl human leptin, subcutaneous, once a day, 0.02 to 0.04 mg/kg (adjusted according to weight loss), indeterminate duration.', 'armGroupLabels': ['Recombinant methionyl human leptin']}]}, 'contactsLocationsModule': {'locations': [{'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Miami Miller School of Medicine, Center on Pharmacogenomics', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}], 'overallOfficials': [{'name': 'Julio Licinio, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Miami'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Miami', 'class': 'OTHER'}, 'collaborators': [{'name': 'National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)', 'class': 'NIH'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Associate Professor', 'investigatorFullName': 'Richard Bookman', 'investigatorAffiliation': 'University of Miami'}}}}