None listed
Conditions
Brief summary
This study is investigating whether a ketone product improves exercise capacity in children with IEMEM, and if the ketone product is acceptable and tolerated by participants. Secondly, to assess quality of life in children with IEMEM, and glean feedback on an IEM-specific quality of life questionnaire for future adaptation in LC-FAOD. We hypothesise that an exogenous ketone product will improve exercise capacity compared to standard therapy, in children with an IEMEM (LC-FAOD or HMG-CoA-lyase deficiency.) Also that quality of life in children with an IEMEM will be negatively impacted, compared to healthy children.
Interventions
2 study visits only - one intervention, one control
Although participants will all have the 'intervention', they will be randomised to receive either 'intervention' or 'control' at their first visit and then will receive the other at their second visit.
Ketone salt, dosed at 170mg/kg in children with inborn errors of metabolism
Ketone ingested 20mins prior to submaximal treadmill exercise test
Submaximal exercise test will be modified Bruce (no slope), 3min stages until heart rate is at 60-70%
2 study visits only - one intervention, one control Although participants will all have the 'intervention', they will be randomised to receive either 'intervention' or 'control' at their first visit and then will receive the other at their second visit. Ketone salt, dosed at 170mg/kg in children with inborn errors of metabolism Ketone ingested 20mins prior to submaximal treadmill exercise test Submaximal exercise test will be modified Bruce (no slope), 3min stages until heart rate is at 60-70% of predicted maximum, then cool down for 3 mins. Blood draws at pre-ketone, 20mins after ketone (pre exercise), and within 5 minutes of ceasing exercise. Study will be conducted by Senior metabolic dietitian (7 years in metabolics), experienced exercise physiologist, experienced metabolic staff specialists, and experienced sports physician.
Sponsors
The Sydney Children's Hospitals Network
Study design
Allocation
Randomised controlled trial
Intervention model
Crossover
Primary purpose
Treatment
Masking
Blinded (masking used) (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
All
Age
5 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
Age 5 – 18 years old Diagnosed (by biochemical data or molecular genetic diagnosis) with a long chain fatty acid oxidation disorder (LC-FAOD), or HMG-CoA-lyase deficiency. Receiving treatment from the Sydney Children’s Hospital Network (SCHN) metabolic service. Participants or parents/guardians must be able to give fully informed written consent for the investigations being performed. Participants must be willing and able to attend the exercise testing facility at the Children’s Hospital at Westmead, on two occasions in total.
Exclusion criteria
Female participants who are pregnant. Participants who do not consent to be part of the trial. Participants at moderate to high risk of adverse events during exercise testing, for example, abdominal aortic aneurysm, pulmonary hypertension, severe aortic stenosis, acute/unstable cardiac status, untreated pulmonary embolism. Participants younger than 5 years of age – to ensure participants are capable of performing the exercise test. Participants older than 18 years of age – this falls outside the study population group of interest.
Outcome results
None listed