Exercise Intolerance Study in Long Chain Fatty Acid Oxidation Disorders
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorAcademisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
About this trial
In this study, the exercise tolerance in patients with long-chain fatty acid oxidation disorders (LcFAOD) will be compared to that of healthy volunteers who are carefully matched in terms of age, sex, BMI and physical activity score. The study aims to assess exercise capacity using cardiopulmonary exercise (CPX) tests and activity monitoring over a 7-day period. The hypothesis of the study is that LcFAOD patients will exhibit greater oxygen debt during short periods of exercise compared to healthy individuals. These findings will contribute to a better understanding of exercise intolerance in LcFAOD patients and may guide the development of targeted interventions, such as exercise training programs and enable the analysis of effectiveness of (new) treatments.
Eligibility criteria
Qualifiers
Men and women (age ≥18 years) with a confirmed diagnosis of carnitine palmitoyltransferase 2 (CPT2) deficiency, Carnitine-acylcarnitine translocase (CACT) deficiency, Mitochondrial Trifunctional protein (MTP) deficiency, Very-long-chain acyl-CoA dehydrogenase (VLCAD) deficiency or Long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency.
Disqualifiers
Pregnancy
Recent acute myocardial infarction (<6 months prior to inclusion)
Uncontrolled arrhythmia/severe conduction disorder causing hemodynamic compromise
Implantable pacemaker or other cardiac device with complete ventricular pacing
Trial design
Treatments tested in this trial
- Intermittent cardiopulmonary exercise test
- Incremental cardiopulmonary exercise test
- Echography of upper leg
- Hand grip strength with hand dynamometer
- Fill in questionnaires regarding physical activity
Treatment groups
50 Participants
are divided into 2 treatment groupsSponsors and collaborators
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Lead sponsor
Stichting Stofwisselkracht
Collaborator
Source ClinicalTrials.gov