Effects of Leptin Replacement in Children

NCT ID: NCT00659828

Last Updated: 2020-02-13

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-06-30

Study Completion Date

2010-04-16

Brief Summary

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To assess the endocrine and immune effects of leptin replacement in leptin-deficient children, from a consanguineous family. The hypothesis is that leptin replacement will have significant effects on endocrine function.

Detailed Description

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The proposed study of the treatment of a child with congenital leptin deficiency will permit to elucidate key aspects of human endocrine function, and will give new insights on the role of leptin in human endocrine regulation.

Conditions

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Obesity Metabolic Syndrome Diabetes

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Recombinant Methionyl Human Leptin

Recombinant methionyl human leptin: Recombinant methionyl human leptin, subcutaneous, once a day, 0.02 to 0.04 mg/kg (adjusted according to weight loss).

Group Type EXPERIMENTAL

Recombinant methionyl human leptin

Intervention Type DRUG

Recombinant methionyl human leptin, subcutaneous, once a day, 0.02 to 0.04 mg/kg (adjusted according to weight loss), indeterminate duration.

Interventions

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Recombinant methionyl human leptin

Recombinant methionyl human leptin, subcutaneous, once a day, 0.02 to 0.04 mg/kg (adjusted according to weight loss), indeterminate duration.

Intervention Type DRUG

Other Intervention Names

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Metreleptin r-metHuLeptin

Eligibility Criteria

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Inclusion Criteria

* Children with a functional leptin gene mutation from a consanguineous Turkish family. Only one leptin-naïve child from this family is alive and eligible.

Exclusion Criteria

* N/A
Minimum Eligible Age

5 Years

Maximum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NIH

Sponsor Role collaborator

University of Miami

OTHER

Sponsor Role lead

Responsible Party

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Richard Bookman

Associate Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Julio Licinio, MD

Role: PRINCIPAL_INVESTIGATOR

University of Miami

Locations

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University of Miami, Center on Pharmacogenomics

Miami, Florida, United States

Site Status

Countries

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United States

References

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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.

Reference Type BACKGROUND
PMID: 17986612 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 17684509 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 17517446 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 15949871 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 15713712 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 15070752 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 11443202 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 10523015 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 9500540 (View on PubMed)

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.

Reference Type DERIVED
PMID: 21070531 (View on PubMed)

Paz-Filho GJ, Delibasi T, Erol HK, Wong ML, Licinio J. Cellular immunity before and after leptin replacement therapy. J Pediatr Endocrinol Metab. 2009 Nov;22(11):1069-74. doi: 10.1515/jpem.2009.22.11.1069.

Reference Type DERIVED
PMID: 20101893 (View on PubMed)

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.

Reference Type DERIVED
PMID: 19889232 (View on PubMed)

Paz-Filho GJ, Babikian T, Asarnow R, Delibasi T, Esposito K, Erol HK, Wong ML, Licinio J. Leptin replacement improves cognitive development. PLoS One. 2008 Aug 29;3(8):e3098. doi: 10.1371/journal.pone.0003098.

Reference Type DERIVED
PMID: 18769731 (View on PubMed)

Other Identifiers

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2R01DK058851-03

Identifier Type: NIH

Identifier Source: secondary_id

View Link

20060295

Identifier Type: -

Identifier Source: org_study_id

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