Withdrawn study: could hydroxychloroquine ease rare muscle disease?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 However, the study was withdrawn before any participants were enrolled.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- This study was withdrawn before enrolling any participants, so no new data were collected.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.
Study facts
What this study's own registry entry says, in plain language.
- Started
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Nov 2019
- Finished
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Nov 2019
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
The population to be studied consists of 8 paediatric patients with Lipin-1 deficiency followed-up by the metabolic diseases center of Necker hospital and having been treated by Hydroxychloroquine sulfate for at least 6 months.
- Ages
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3 months to 18 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Age ≥ 3 months * Minors with Lipin-1 deficiency which were diagnosed with familiar context analysis followed by genetic diagnosis (two causal mutations on the LPIN1 gene) and treated by Hydroxychloroquine Sulfate off label use on a compassionate basis in the Metabolic Diseases Center of Necker Hospital * Patients treated by Hydroxychloroquine Sulfate for at least 6 months Exclusion Criteria: \- Opposition of parental authority holders
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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