Association of Insulin Resistance and FGF21 on Cardiac Fu... | NCT04222101

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

NCT04222101

Sponsor

Le Bonheur Children's Hospital

Status

Terminated

Last Update Posted

Dec 20, 2021Actual

Enrollment

9Actual

Phase

Not Applicable

Conditions

Cardiomyopathy, Dilated
Cardiomyopathy, Hypertrophic
Insulin Resistance

Interventions

Oral glucose tolerance test

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Dec 2021

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

COVID-19 pandemic

Expanded Access Info

Start Date

Oct 7, 2019Actual

Primary Completion Date

Jun 30, 2021Actual

Completion Date

Jun 30, 2021Actual

First Submitted Date

Dec 19, 2019

First Submission Date that Met QC Criteria

Jan 6, 2020

First Posted Date

Jan 9, 2020Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 6, 2021

Last Update Posted Date

Dec 20, 2021Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Daniel Mak, MD, Principal Investigator, Le Bonheur Children's HospitalPrincipal Investigator

Lead Sponsor

Le Bonheur Children's HospitalOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

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

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

9Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
cardiomyopathy	Other	only one arm, all participants undergo oral glucose tolerance testing and results are used to evaluate association with degree of cardiac dysfunction	Diagnostic Test: Oral glucose tolerance test

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Oral glucose tolerance test	Diagnostic Test	measure insulin, glucose and FGF21 levels in response to oral glucose challenge	cardiomyopathy

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Whole body insulin sensitivity index	correlate whole body insulin sensitivity index with left ventricular ejection fraction	baseline

Secondary Outcomes

Measure	Description	Time Frame
FGF21 level	correlate FGF21 levels with whole body sensitivity index and left ventricular ejection fraction	baseline

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

13 Years

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Daniel Mak, MD	Le Bonheur/UTHSC Pediatric Endocrine fellowship	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Le Bonheur Children's Hospital		Memphis	Tennessee	38103	United States

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
17047217	Background	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.
17921325	Background	Neglia D, De Caterina A, Marraccini P, Natali A, Ciardetti M, Vecoli C, Gastaldelli A, Ciociaro D, Pellegrini P, Testa R, Menichetti L, L'Abbate A, Stanley WC, Recchia FA. Impaired myocardial metabolic reserve and substrate selection flexibility during stress in patients with idiopathic dilated cardiomyopathy. Am J Physiol Heart Circ Physiol. 2007 Dec;293(6):H3270-8. doi: 10.1152/ajpheart.00887.2007. Epub 2007 Oct 5.

ID	Term
D006332	Cardiomegaly
D006331	Heart Diseases
D002318	Cardiovascular Diseases
D009202	Cardiomyopathies

ID	Term
D001774	Blood Chemical Analysis
D019963	Clinical Chemistry Tests
D019411	Clinical Laboratory Techniques
D019937	Diagnostic Techniques and Procedures

ID	Term
D002311	Cardiomyopathy, Dilated
D002312	Cardiomyopathy, Hypertrophic
D007333	Insulin Resistance