New hope for rare brain disorder: early drug access program opens
NCT ID NCT05911399
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This program offers early access to tiratricol, a thyroid hormone-like drug, for people with Allan-Herndon-Dudley syndrome (AHDS), a rare genetic condition that affects brain development and movement. Patients must have a confirmed genetic diagnosis and be considered likely to benefit by their doctor. The goal is to provide treatment before the drug is fully approved.
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, this could provide a treatment option for a rare, severe neurological condition with no approved therapies.
- What could go wrong
- This is an expanded access program, not a formal trial, so data on effectiveness and safety is still limited. The drug may not work for all patients and could have side effects.
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
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Study contacts
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Contact
Email: •••••@•••••
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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