New drug cocktail shows promise for rare muscle disease
NCT ID NCT03845712
First seen Jun 26, 2026 · Last updated Aug 14, 2026 · Updated 3 times
Summary
This phase 2 study tests a combination of two nucleoside drugs, doxecitine and doxribtimine, in 47 people with thymidine kinase 2 (TK2) deficiency, a rare genetic disorder that weakens muscles. Participants already receiving nucleoside therapy continue treatment to see if the drug is safe and helps improve their condition. The study is open-label, meaning everyone knows they are getting the active treatment.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- doxecitine and doxribtimine (nucleoside combination)
- What this could lead to
- If successful, this could provide a safe and effective treatment option to manage TK2 deficiency, a rare genetic disease that affects muscle function.
- What could go wrong
- This is a small, early-phase study with no placebo group, so results may not be conclusive. Side effects are possible, and the treatment may not work for everyone.
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
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Tk0102 1005
New York, New York, 10032, United States
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Tk0102 3031
Madrid, Spain
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Tk0102 3101
Seville, Spain
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Tk0102 3102
Barcelona, Spain
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Tk0102 3121
Esplugues de Llobregat, Spain
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Tk0102 4037
Nehariya, Israel
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Tk0102 4038
Haifa, Israel
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Tk0102 4039
Holon, Israel
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