Withdrawn study: could hydroxychloroquine ease rare muscle disease?
NCT ID NCT04007562
First seen Jun 25, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This study aimed to look back at children with Lipin-1 deficiency who were given hydroxychloroquine to prevent severe muscle breakdown and pain. The condition causes repeated, dangerous episodes of muscle damage. Researchers planned to check safety and effectiveness by measuring muscle enzymes and inflammation. However, the study was withdrawn before any participants were enrolled.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Hydroxychloroquine sulfate
- What this could lead to
- If effective, hydroxychloroquine could reduce severe muscle breakdown and pain in children with Lipin-1 deficiency, offering a treatment option where none currently exists.
- What could go wrong
- This study was withdrawn before enrolling any participants, so no new data were collected. The findings rely on past compassionate use, which is not as reliable as a controlled trial.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
-
Hôpital Necker-Enfants Malades
Paris, 75015, France