New drug hopes to boost brain function in kids with rare genetic disorder
NCT ID NCT06617429
First seen Jun 25, 2026 · Last updated Aug 07, 2026 · Updated 3 times
Summary
This Phase 3 trial tests a drug called GTX-102 (apazunersen) in 129 children with Angelman syndrome, a genetic condition causing severe developmental delays. The study compares the drug to a sham procedure to see if it improves cognitive function and other symptoms over about 11 months. Participants receive multiple injections into the spine.
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
- What this could lead to
- If successful, this could lead to a treatment that improves cognitive function and reduces symptoms in children with Angelman syndrome.
- What could go wrong
- This is an early Phase 3 trial with a small number of participants. The treatment may not work better than a sham procedure, and there could be side effects from the lumbar puncture injections.
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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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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British Columbia Children's Hospital
Vancouver, V6H 3V4, Canada
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Carum Research Inc
Dallas, Texas, 75243, United States
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Cedars Sinai
Los Angeles, California, 90048, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Mercy
Kansas City, Missouri, 64108, United States
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Columbia University Medical Center
New York, New York, 10032, United States
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Haunersche Kinderklinik
Munich, 80336, Germany
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Hokkaido University Hospital
Sapporo, 060-8648, Japan
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Hospital Sant Joan de Deu
Barcelona, 08950, Spain
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Hospital Universitario Parc Tauli
Barcelona, 08208, Spain
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Hospital Universitario Puerta de Hierro
Madrid, 28222, Spain
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Hospital Universitario Virgen del Rocio
Seville, 41013, Spain
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McGill University Health Centre
Montreal, Quebec, H4A3J1, Canada
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Medical University of Gdańsk
Gdansk, 80211, Poland
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Nagoya City University Graduate School of Medical Sciences
Nagoya, Aichi-ken, 467-0001, Japan
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Nicklaus Children's Hospital
Miami, Florida, 33155, United States
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Osaka City General Hospital
Osaka, 543-0021, Japan
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Polish Mothers Memorial Institute
Lodz, 93-338, Poland
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Rare Disease Research
Atlanta, Georgia, 30329, United States
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Rare Disease Research
Hillsborough, North Carolina, 27278, United States
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Rush University
Chicago, Illinois, 60612, United States
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The University of Texas
Austin, Texas, 78723, United States
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UCSD, Rady Children's Hospital
San Diego, California, 92123, United States
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UCSF
San Francisco, California, 94143, United States
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UNC Chapel Hill Pediatrics
Chapel Hill, North Carolina, 27599, United States
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Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20251, Germany
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University of Leipzig
Leipzig, 04155, Germany
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can early parent coaching help infants with rare genetic disorders thrive?
- Newborn screening study aims to catch rare diseases at birth
- New hope for angelman syndrome: drug trial targets brain function
- New registry aims to shed light on angelman syndrome
- New study tracks angelman syndrome progression in kids and adults
- 2000-Patient study aims to uncover hidden metabolic risks in rare genetic disorders