New drug aims to control angelman syndrome long-term
NCT ID NCT06415344
First seen Jun 27, 2026 · Last updated Jul 28, 2026 · Updated 2 times
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.
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
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
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