Energy Metabolism in Branched-chain Organic Acidemias | NCT03917212

Studies
Energy Metabolism in Branched-chain Organic Acidemias

NCT03917212

Sponsor

German Diabetes Center

Status

Completed

Last Update Posted

Apr 16, 2019Actual

Enrollment

57Actual

Phase

Not provided

Conditions

Energy Metabolism
Metabolic Syndrome
Insulin Resistance

Interventions

No intervention

Countries

Germany

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

Apr 2019

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Mar 2012Actual

Primary Completion Date

Nov 2017Actual

Completion Date

Mar 2018Actual

First Submitted Date

Apr 9, 2019

First Submission Date that Met QC Criteria

Apr 15, 2019

First Posted Date

Apr 16, 2019Actual

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

Apr 15, 2019

Last Update Posted Date

Apr 16, 2019Actual

Name	Class
University Children's Hospital, Düsseldorf	UNKNOWN

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

Energy metabolism and insulin sensitivity were assessed in a case-control study in patients with branched-chain organic acidemias.

Detailed Description

Children and adults with branched-chain organic acidemias and matched healthy humans underwent deep metabolic phenotyping using non-invasive magnetic resonance spectroscopy, oral glucose tolerance tests and measurements of leucocyte respirometry in order to assess changes in energy metabolism and insulin sensitivity.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

Retention

Enrollment

57Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Patients		Patients with propionic acidemia, isovaleric acidemia, methylmalonic acidemia	Other: No intervention
Controls		Healthy humans	Other: No intervention

Interventions

Name	Type	Description	Arm Group Labels	Other Names
No intervention	Other	No intervention, observational study with metabolic characterisation	Controls Patients

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Hepatic and skeletal muscle energy metabolism	Hepatic and skeletal muscle energy metabolism assessed by magnetic resonance spectroscopy	At baseline

Secondary Outcomes

Measure	Description	Time Frame
Insulin sensitivity	Insulin sensitivity measured from oral glucose tolerance test	At baseline

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

known branched-chain organic acidemia diagnosed by enzymatic or genetic test
age>5 years

Exclusion Criteria:

acute inflammatory conditions with fever, cold symptoms, gastro-intestinal infection within the last 24 hours
contraindications for an oral glucose tolerance test such as fasting blood glucose >126 mg/dl, severe chronic gastrointestinal conditions, allergy against black currant
contraindications for magnetic resonance tomography (MRT) such as claustrophobia, metallic implants which are not MRT compatible

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

5 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Patients with known propionic acidemia, methylmalonic acidemia or isovaleric acidemia were enrolled as well as healthy humans of comparable age, sex and BMI

Sampling Method

Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Michael Roden, Professeor	German Diabetes Center	Principal Investigator
Regina Ensenauer, Professor	Children's Hospital Düsseldorf	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Department of General Pediatrics, Neonatology and Pediatric Cardiology of the University Children's Hospital Düsseldorf		Düsseldorf	North Rhine-Westphalia		Germany

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D024821	Metabolic Syndrome
D007333	Insulin Resistance
D005234	Fatty Liver
D050171	Dyslipidemias

Ancestor Terms

ID	Term
D006946	Hyperinsulinism
D044882	Glucose Metabolism Disorders
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases

Browse Leaves

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Browse Branches

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