Experimental drug offers hope for babies with rare genetic brain condition
NCT ID NCT04143295
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This trial tests a drug called DITPA in male infants with MCT8 deficiency, a rare genetic disorder that causes severe intellectual disability and movement problems. The drug aims to improve thyroid hormone function in the brain. Only infants whose families have a history of the condition and choose not to terminate the pregnancy are eligible.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Diiodothyropropionic acid (DITPA)
What this could lead to
If it works, this could offer a treatment to improve brain development and movement in infants with MCT8 deficiency, potentially reducing severe disability.
What could go wrong
This is an early-stage trial with very few participants, so results may not apply broadly. The drug may not fully correct the underlying genetic problem, and side effects are unknown.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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.
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Other studies related to the condition(s) this trial covers.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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University of Miami, Miller School of Medicine
AVAILABLEMiami, Florida, 33136, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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