Exercise test could revolutionize monitoring of rare metabolic muscle disease
NCT ID NCT03802279
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This study looked at whether an exercise test can be used to monitor patients with a rare hereditary disease that causes severe muscle breakdown (rhabdomyolysis). Researchers measured heart function and oxygen use during exercise in 27 patients. The goal was to find a reliable way to track disease severity and treatment effects in future clinical trials.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- What this could lead to
- If successful, this could provide a simple, non-invasive way to monitor disease severity and treatment response in patients with rhabdomyolysis due to hereditary metabolic disorders.
- What could go wrong
- This is a small, completed observational study with only 27 participants. It does not test a treatment, so it may not directly lead to new therapies. The effort test may not be feasible for all patients.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Hôpital Necker-Enfants Malades
Paris, 75015, France