NCT03531554

Acute Nutritional Ketosis in VLCAD Deficiency

Completed NA Last updated 21 May 2018

What this trial tests

NA trial testing ketone ester drink in VLCAD Deficiency in 5 participants. Completed in 1 April 2017.

Timeline

1 April 2016

Primary endpoint
31 March 2017

1 April 2017

Quick facts

Lead sponsor	University Medical Center Groningen
Phase	NA
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	crossover
Masking	double
Primary purpose	other
Enrollment	5
Start date	1 April 2016
Primary completion	31 March 2017
Estimated completion	1 April 2017
Sites	2 locations across Netherlands

Drugs / interventions tested

• ketone ester drink
• exercise
• muscle biopsy
• Magnetic Resonance Imaging

Conditions studied

• VLCAD Deficiency — all drugs for VLCAD Deficiency →
• Fatty Acid Oxidation Defects — all drugs for Fatty Acid Oxidation Defects →

Sponsor

University Medical Center Groningen

Who can join

Adults 16 to 65, any sex, with VLCAD Deficiency or Fatty Acid Oxidation Defects. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

To test if a ketone-ester based drink can boost muscle mitochondrial function in vivo in patients with VLCADD in order to establish a rational basis for therapeutic use in this disorder.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Ketone Administration for Seizure Disorders: History and Rationale for Ketone Esters and Metabolic Alternatives.
   Poff AM, Rho JM, D'Agostino DP. · · 2019 · cited 52× · PMID 31680801 · DOI 10.3389/fnins.2019.01041
2. Nutritional ketosis improves exercise metabolism in patients with very long-chain acyl-CoA dehydrogenase deficiency.
   Bleeker JC, Visser G, Clarke K, Ferdinandusse S, et al · · 2020 · cited 33× · PMID 31955429 · DOI 10.1002/jimd.12217