New drug hopes to boost brain function in kids with rare genetic disorder

NCT ID NCT06617429

Ongoing Disease control Sponsor: Ultragenyx Pharmaceutical Inc Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

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.

Disclaimer Read more

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.

Angelman syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ANGELMAN SYNDROME

Contacts and locations

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Locations

Boston Children's Hospital

Boston, Massachusetts, 02115, United States
British Columbia Children's Hospital

Vancouver, V6H 3V4, Canada
Carum Research Inc

Dallas, Texas, 75243, United States
Cedars Sinai

Los Angeles, California, 90048, United States
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Mercy

Kansas City, Missouri, 64108, United States
Columbia University Medical Center

New York, New York, 10032, United States
Haunersche Kinderklinik

Munich, 80336, Germany
Hokkaido University Hospital

Sapporo, 060-8648, Japan
Hospital Sant Joan de Deu

Barcelona, 08950, Spain
Hospital Universitario Parc Tauli

Barcelona, 08208, Spain
Hospital Universitario Puerta de Hierro

Madrid, 28222, Spain
Hospital Universitario Virgen del Rocio

Seville, 41013, Spain
McGill University Health Centre

Montreal, Quebec, H4A3J1, Canada
Medical University of Gdańsk

Gdansk, 80211, Poland
Nagoya City University Graduate School of Medical Sciences

Nagoya, Aichi-ken, 467-0001, Japan
Nicklaus Children's Hospital

Miami, Florida, 33155, United States
Osaka City General Hospital

Osaka, 543-0021, Japan
Polish Mothers Memorial Institute

Lodz, 93-338, Poland
Rare Disease Research

Atlanta, Georgia, 30329, United States
Rare Disease Research

Hillsborough, North Carolina, 27278, United States
Rush University

Chicago, Illinois, 60612, United States
The University of Texas

Austin, Texas, 78723, United States
UCSD, Rady Children's Hospital

San Diego, California, 92123, United States
UCSF

San Francisco, California, 94143, United States
UNC Chapel Hill Pediatrics

Chapel Hill, North Carolina, 27599, United States
Universitaetsklinikum Hamburg-Eppendorf

Hamburg, 20251, Germany
University of Leipzig

Leipzig, 04155, Germany