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.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for MONOCARBOXYLATE TRANSPORTER 8 DEFICIENCY are added.
By submitting, you agree to our Terms of use
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
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
-
Contact
Email: •••••@•••••
Locations
-
Ann & Robert H. Lurie Children's Hospital of Chicago
AVAILABLEChicago, Illinois, 60611, United States
Contact
-
Baystate Medical Center
AVAILABLESpringfield, Massachusetts, 01199, United States
Contact
-
Children's Healthcare of Atlanta
AVAILABLEAtlanta, Georgia, 30329, United States
Contact
-
Children's Hospital of Philadelphia
AVAILABLEPhiladelphia, Pennsylvania, 19104, United States
Contact
-
Children's Wisconsin
AVAILABLEMilwaukee, Wisconsin, 53326, United States
Contact
-
Columbia University Irving Medical Center
AVAILABLENew York, New York, 10032, United States
Contact
-
Cook Children's Health Care System
AVAILABLEFort Worth, Texas, 76106, United States
Contact
-
Gillette's Children's Specialty Healthcare
AVAILABLESaint Paul, Minnesota, 55101, United States
Contact
-
Icahn School of Medicine at Mount Sinai
AVAILABLENew York, New York, 10029, United States
Contact
-
Le Bonheur Children's Hospital Foundation
AVAILABLEMemphis, Tennessee, 38105, United States
Contact
-
Louisiana State University Health Sciences Center and Children's Hospital
AVAILABLENew Orleans, Louisiana, 70118, United States
Contact
-
MultiCare Mary Bridge Children's Hospital
AVAILABLETacoma, Washington, 98403, United States
Contact
-
Nemour's Children Hospital
AVAILABLEJacksonville, Florida, 32207, United States
Contact
-
Oregon Health and Science University
AVAILABLEPortland, Oregon, 97239, United States
Contact
-
Rady Children's Hospital
AVAILABLESan Diego, California, 92123, United States
Contact
-
University of Alabama at Birmingham Hospital
AVAILABLEBirmingham, Alabama, 35249, United States
Contact
-
Valley Children's Hospital
AVAILABLEMadera, California, 93636-8762, United States
Contact
More trials for these conditions
Other studies related to the condition(s) this trial covers.