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Energy Metabolism in Branched-chain Organic Acidemias

Sponsor
German Diabetes Center (Other)
Overall Status
Completed
CT.gov ID
NCT03917212
Collaborator
University Children's Hospital, Düsseldorf (Other)
57
Enrollment
2
Locations
72
Actual Duration (Months)
28.5
Patients Per Site
0.4
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
  • Other: No intervention

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.

Study Design

Study Type:
Observational
Actual Enrollment :
57 participants
Observational Model:
Case-Control
Time Perspective:
Prospective
Official Title:
Non-invasive Measurement of Energy Metabolism in Patients With Primary and Secondary Mitochondrial Diseases
Actual Study Start Date :
Mar 1, 2012
Actual Primary Completion Date :
Nov 1, 2017
Actual Study Completion Date :
Mar 1, 2018

Arms and Interventions

Arm Intervention/Treatment
Patients

Patients with propionic acidemia, isovaleric acidemia, methylmalonic acidemia

Other: No intervention
No intervention, observational study with metabolic characterisation
Controls

Healthy humans

Other: No intervention
No intervention, observational study with metabolic characterisation

Outcome Measures

Primary Outcome Measures

  1. Hepatic and skeletal muscle energy metabolism [At baseline]

    Hepatic and skeletal muscle energy metabolism assessed by magnetic resonance spectroscopy

Secondary Outcome Measures

  1. Insulin sensitivity [At baseline]

    Insulin sensitivity measured from oral glucose tolerance test

Eligibility Criteria

Criteria

Ages Eligible for Study:
5 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
Yes
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Department of General Pediatrics, Neonatology and Pediatric Cardiology of the University Children's Hospital Düsseldorf Düsseldorf NRW Germany
2 Institute for Clinical Diabetology, German Diabetes Center Düsseldorf NRW Germany

Sponsors and Collaborators

  • German Diabetes Center
  • University Children's Hospital, Düsseldorf

Investigators

  • Principal Investigator: Michael Roden, Professeor, German Diabetes Center
  • Principal Investigator: Regina Ensenauer, Professor, Children's Hospital Düsseldorf

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
German Diabetes Center
ClinicalTrials.gov Identifier:
NCT03917212
Other Study ID Numbers:
  • MITO-MRS
First Posted:
Apr 16, 2019
Last Update Posted:
Apr 16, 2019
Last Verified:
Apr 1, 2019
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by German Diabetes Center
Organic acidemias
Mitochondria
Oxidative stress
Fatty liver
Dyslipidemia
Additional relevant MeSH terms:
Metabolic Syndrome
Insulin Resistance

Study Results

No Results Posted as of Apr 16, 2019