New drug trial hopes to ease angelman syndrome symptoms in kids

NCT ID NCT05630066

Completed Disease control Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a drug called alogabat in 48 children aged 5-17 with Angelman syndrome (a specific genetic type). The goal was to see how the drug moves through the body and if it is safe. Researchers also looked at brain activity changes to see if the drug works as expected. This is not a cure, but aims to control symptoms.

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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

Locations

Boston Children's Hospital

Boston, Massachusetts, 02115, United States
CHRU de Brest

Brest, 29609, France
CHU Dijon Bourgogne Hôpital François Mitterand

Dijon, 21000, France
Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine

Carrboro, North Carolina, 27510, United States
Columbia University Medical Center

New York, New York, 10032, United States
Corporacio Sanitaria Parc Tauli

Sabadell, Barcelona, 08208, Spain
Dr. Von Haunersches Kinderspital

München, 80337, Germany
Groupe Hospitalier Necker Enfants Malades

Paris, 75015, France
Hopital la Timone Enfants

Marseille, 13005, France
Hospital Sant Joan de Deu

Esplugues de Llobregat · Barcelona, Barcelona, 08950, Spain
Hospital Universitario Puerta De Hierro Majadahonda

Madrid, 28222, Spain
Hospital Universitario de Navarra;Unidad de Neuropediatría

Pamploa, Navarre, 31008, Spain
IRCCS Eugenio Medea

Conegliano Veneto (TV), Veneto, 31015, Italy
IRCCS Istituto G. Gaslini

Genoa, Liguria, 16147, Italy
Multicare Institute for Research and Innovation

Tacoma, Washington, 98405, United States
Ospedale Pediatrico Bambino Gesù

Rome, Lazio, 00165, Italy
Queensland Children?s Hospital

South Brisbane, Queensland, 4101, Australia
Rush Medical Center

Chicago, Illinois, 60612, United States
Vanderbilt Children's Hospital

Nashville, Tennessee, 37232-9119, United States

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