Experimental drug tiratricol now available for rare genetic disorder before official approval
NCT ID NCT05911399
First seen Nov 01, 2025 · Last updated Jun 23, 2026 · Updated 37 times
Summary
This program provides early access to tiratricol, a thyroid hormone-like drug, for people with Allan-Herndon-Dudley syndrome (AHDS), a rare genetic condition that affects movement and development. Eligible patients must have a confirmed genetic diagnosis and a doctor who believes the drug may help. The goal is to offer treatment before the drug is fully approved.
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This is a summary of
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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Contacts and locations
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
AVAILABLEChicago, Illinois, 60611, United States
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Baystate Medical Center
AVAILABLESpringfield, Massachusetts, 01199, United States
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Children's Healthcare of Atlanta
AVAILABLEAtlanta, Georgia, 30329, United States
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Children's Hospital of Philadelphia
AVAILABLEPhiladelphia, Pennsylvania, 19104, United States
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Children's Wisconsin
AVAILABLEMilwaukee, Wisconsin, 53326, United States
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Columbia University Irving Medical Center
AVAILABLENew York, New York, 10032, United States
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Cook Children's Health Care System
AVAILABLEFort Worth, Texas, 76106, United States
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Gillette's Children's Specialty Healthcare
AVAILABLESaint Paul, Minnesota, 55101, United States
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Icahn School of Medicine at Mount Sinai
AVAILABLENew York, New York, 10029, United States
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Le Bonheur Children's Hospital Foundation
AVAILABLEMemphis, Tennessee, 38105, United States
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Louisiana State University Health Sciences Center and Children's Hospital
AVAILABLENew Orleans, Louisiana, 70118, United States
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MultiCare Mary Bridge Children's Hospital
AVAILABLETacoma, Washington, 98403, United States
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Nemour's Children Hospital
AVAILABLEJacksonville, Florida, 32207, United States
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Oregon Health and Science University
AVAILABLEPortland, Oregon, 97239, United States
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Rady Children's Hospital
AVAILABLESan Diego, California, 92123, United States
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University of Alabama at Birmingham Hospital
AVAILABLEBirmingham, Alabama, 35249, United States
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Valley Children's Hospital
AVAILABLEMadera, California, 93636-8762, United States
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What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Tiratricol (a thyroid hormone-like drug taken by mouth or feeding tube)
What this could lead to
If it works, tiratricol could help manage symptoms of Allan-Herndon-Dudley syndrome, improving quality of life for patients with this rare condition.
What could go wrong
This is an expanded access program, not a formal trial, so evidence is limited. Benefits are uncertain and risks may include side effects from the drug.
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.