Association of Insulin Resistance and FGF21 on Cardiac Function in Pediatric Dilated Cardiomyopathy

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

NA

Total Enrollment

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-10-07

Study Completion Date

2021-06-30

Brief Summary

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This study will investigate whether there is an association between insulin resistance and cardiac function in children with dilated or hypertrophic cardiomyopathy. This study will also investigate whether there is an association between FGF21 and cardiac function in children with dilated or hypertrophic cardiomyopathy and whether this is mediated through greater insulin resistance and/or through independent effects.

Detailed Description

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Although pediatric cardiomyopathy is rare, the condition is severe and life-threatening. The main focus of this proposed study will examine whether insulin resistance is correlated with decreased cardiac function which will hopefully pave the way for future clinical trials using medications that sensitize insulin such as metformin or glucagon-like peptide-1 (GLP-1 agonists) as possible therapeutic agents. The exploratory piece of this study will investigate a novel therapeutic target by determining whether FGF21 has any direct effects on cardiac function and whether it interacts with insulin resistance in altering cardiac function. Patients with cardiomyopathy normally undergo ECHO as part of routine evaluation and follow up and is standard of care. At this time, there are no official guidelines for pediatric patients with cardiomyopathy to undergo oral glucose tolerance testing (OGTT) and thus it is not part of the standard of care. Based on findings from this study, the investigators hope to justify performing an OGTT on pediatric patients with dilated or hypertrophic cardiomyopathy and incorporate the procedure in future practice guidelines.

Conditions

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Cardiomyopathy, Dilated Cardiomyopathy, Hypertrophic Insulin Resistance

Keywords

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pediatrics

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

SCREENING

Blinding Strategy

NONE

Study Groups

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cardiomyopathy

only one arm, all participants undergo oral glucose tolerance testing and results are used to evaluate association with degree of cardiac dysfunction

Group Type OTHER

Oral glucose tolerance test

Intervention Type DIAGNOSTIC_TEST

measure insulin, glucose and FGF21 levels in response to oral glucose challenge

Interventions

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Oral glucose tolerance test

measure insulin, glucose and FGF21 levels in response to oral glucose challenge

Intervention Type DIAGNOSTIC_TEST

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of dilated or hypertrophic cardiomyopathy
* Pubertal (Tanner 2 breast in females or testicular volume ≥ 4mL in males)
* Permission by the primary cardiologist of the patient for enrollment in the study

Exclusion Criteria

* Prior diagnosis of diabetes and treatment with anti-diabetes medication
* Neuromuscular disorder
* Inborn error of metabolism
* Malformation syndrome
* Clinically unstable based on the assessment of the primary cardiologist caring for the patient
* inability of parent/legal guardian to provide informed consent
* non-English speaking

Minimum Eligible Age

13 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Responsible Party

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Daniel Mak, MD

Principal Investigator

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Daniel Mak, MD

Role: PRINCIPAL_INVESTIGATOR

Le Bonheur/UTHSC Pediatric Endocrine fellowship

Locations

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Le Bonheur Children's Hospital

Memphis, Tennessee, United States

Site Status

Countries

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United States

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19-06748-FB

Identifier Type: -

Identifier Source: org_study_id

Association of Insulin Resistance and FGF21 on Cardiac Function in Pediatric Dilated Cardiomyopathy

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Keywords

Study Design

Study Groups

cardiomyopathy

Oral glucose tolerance test

Interventions

Oral glucose tolerance test

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

Le Bonheur Children's Hospital

Responsible Party

Daniel Mak, MD

Principal Investigators

Daniel Mak, MD

Locations

Le Bonheur Children's Hospital

Countries

References

Towbin JA, Lowe AM, Colan SD, Sleeper LA, Orav EJ, Clunie S, Messere J, Cox GF, Lurie PR, Hsu D, Canter C, Wilkinson JD, Lipshultz SE. Incidence, causes, and outcomes of dilated cardiomyopathy in children. JAMA. 2006 Oct 18;296(15):1867-76. doi: 10.1001/jama.296.15.1867.

Riehle C, Abel ED. Insulin Signaling and Heart Failure. Circ Res. 2016 Apr 1;118(7):1151-69. doi: 10.1161/CIRCRESAHA.116.306206.

Fisher FM, Maratos-Flier E. Understanding the Physiology of FGF21. Annu Rev Physiol. 2016;78:223-41. doi: 10.1146/annurev-physiol-021115-105339. Epub 2015 Nov 19.

Camporez JP, Jornayvaz FR, Petersen MC, Pesta D, Guigni BA, Serr J, Zhang D, Kahn M, Samuel VT, Jurczak MJ, Shulman GI. Cellular mechanisms by which FGF21 improves insulin sensitivity in male mice. Endocrinology. 2013 Sep;154(9):3099-109. doi: 10.1210/en.2013-1191. Epub 2013 Jun 13.

Shah A, Shannon RP. Insulin resistance in dilated cardiomyopathy. Rev Cardiovasc Med. 2003;4 Suppl 6:S50-7.

Davila-Roman VG, Vedala G, Herrero P, de las Fuentes L, Rogers JG, Kelly DP, Gropler RJ. Altered myocardial fatty acid and glucose metabolism in idiopathic dilated cardiomyopathy. J Am Coll Cardiol. 2002 Jul 17;40(2):271-7. doi: 10.1016/s0735-1097(02)01967-8.

Nikolaidis LA, Sturzu A, Stolarski C, Elahi D, Shen YT, Shannon RP. The development of myocardial insulin resistance in conscious dogs with advanced dilated cardiomyopathy. Cardiovasc Res. 2004 Feb 1;61(2):297-306. doi: 10.1016/j.cardiores.2003.11.027.

Swan JW, Anker SD, Walton C, Godsland IF, Clark AL, Leyva F, Stevenson JC, Coats AJ. Insulin resistance in chronic heart failure: relation to severity and etiology of heart failure. J Am Coll Cardiol. 1997 Aug;30(2):527-32. doi: 10.1016/s0735-1097(97)00185-x.

Witteles RM, Tang WH, Jamali AH, Chu JW, Reaven GM, Fowler MB. Insulin resistance in idiopathic dilated cardiomyopathy: a possible etiologic link. J Am Coll Cardiol. 2004 Jul 7;44(1):78-81. doi: 10.1016/j.jacc.2004.03.037.

Other Identifiers

19-06748-FB