New drug aims to control angelman syndrome long-term
NCT ID NCT06415344
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 3 trial tests the long-term safety of GTX-102 (apazunersen) in 255 people with Angelman syndrome, a genetic disorder causing developmental delays and seizures. Participants have already completed an earlier GTX-102 study and will continue receiving the drug. The main goal is to track side effects over time.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
GTX-102 (apazunersen), an antisense oligonucleotide drug
What this could lead to
If successful, this could show that GTX-102 is safe enough to use long-term and may help manage symptoms of Angelman syndrome.
What could go wrong
This is an extension study, so it only includes people who already tolerated GTX-102. It does not prove the drug works for everyone, and long-term side effects are still unknown.
Disclaimer
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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.
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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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Locations
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AP-HM - Hopital de la Timone
Marseille, 13385, France
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AP-HP Hopital Necker-Enfants Malades
Paris, 75015, France
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4, Canada
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Childrens Hospital London Health Sciences Centre
London, Ontario, N6A 5W9, Canada
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Childrens Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
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Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
Sabadell, 08208, Spain
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Great Ormond Street Hospital for Children
London, WC1N 3JH, United Kingdom
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Hospital Universitario Puerta de Hierro
Majadahonda, 28222, Spain
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MAGIC Clinic Ltd
Calgary, Alberta, T2E 7Z4, Canada
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Oxford University Hospitals NHS Foundation Trust
Oxford, OX3 9DU, United Kingdom
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Queensland Children's Hospital
South Brisbane, Queensland, 4101, Australia
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Rare Disease Research, LLC
Atlanta, Georgia, 30329, United States
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Rush University Medical Center
Chicago, Illinois, 60612, United States
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Sheba Medical Center
Ramat Gan, 5265601, Israel
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The Research Institute of the McGill University Health Centre
Montreal, Quebec, H3G 1A4, Canada
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The Royal Children's Hospital
Melbourne, Victoria, 3052, Australia
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Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20246, Germany
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Universitaetsklinikum Leipzig
Leipzig, 04103, Germany
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University of California, Los Angeles (UCLA)
Los Angeles, California, 90095, United States
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University of California, San Diego - Rady Children's Hospital
San Diego, California, 92123, United States
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University of Cambridge
Cambridge, CB2 0QQ, United Kingdom
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Weill Cornell Medicine
New York, New York, 10021, United States