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Acute Nutritional Ketosis in VLCAD Deficiency: testing the metabolic basis for therapeutic use

Phase 2
Completed
Conditions
VLCADD
10000546
metabolic disease
Registration Number
NL-OMON44949
Lead Sponsor
niversitair Medisch Centrum Groningen
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Completed
Sex
Not specified
Target Recruitment
6
Inclusion Criteria

* Confirmed VLCADD by genetic profiling
* age: 16-65

Exclusion Criteria

* contra indications for MRI studies (assessed by standardised questionnaire as previously used in METc 08-267/K. Such as metal implants, vascular clips, eye-implants, metal particles in the eye, tattoos containing iron oxide, suspected pregnancy, refusal to be informed of strucural brain abnormalities that could be detected)
* inability to perform bicycle exercise.
* recent episode of rhabdomyolysis, or treatment for acute renal failure in the past 2
months.
* intercurrent illness which may influence exercise tolerance (anaemia, musculoskeletal
injury, or other undiagnosed illness under investigation).
* known coronary artery disease, positive history for angina, or changes on ECG
suggestive of previous ischaemia without a negative stress test.
* insulin-dependent diabetes mellitus.
* loss of, or an inability to give informed consent.
* pregnancy or current breastfeeding, or females not taking the oral contraceptive pill
* any other cause which in the opinion of the investigators, may affect the volunteers
ability to participate in the study

Study & Design

Study Type
Observational invasive
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
<p>To investigate:<br /><br><br /><br>1. the potential of a novel dietary substrate preparation to enhance muscle<br /><br>mitochondrial function in VLCADD via acute nutritional ketosis.<br /><br><br /><br>2. if tri-citric acid cycle substrate supply in muscle of patients with<br /><br>VLCADDis boosted by acute nutritional ketosis.</p><br>
Secondary Outcome Measures
NameTimeMethod
<p>nvt</p><br>
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