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New drug aims to control angelman syndrome long-term

NCT ID NCT06415344

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 10, 2026 · Updated 4 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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 255 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2024

Expected to finish

Feb 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Signed informed consent from parent(s) or legal guardian(s). * Completed a final study visit in a prior clinical trial with GTX-102. If the subject has an ongoing adverse event, medical monitor approval is required to rollover. The Screening visit must occur within 6 months of the last visit in the prior GTX-102 trial. * From the time of informed consent through the end of the study and for at least 6 months after the final dose of GTX-102, females of childbearing potential who are sexually active must use highly effective contraception or abstinence. Males are able to participate if they agree to remain abstinent (refrain from heterosexual intercourse) or use acceptable contraceptive methods during the study and for at least 3 months after the final dose of GTX-102. Exclusion Criteria: * Discontinued from study or withdrew from study treatment in a prior GTX-102 trial. * History or evidence of any other medical, neurological, or psychological condition that would expose the subject to an undue risk of a significant AE or interfere with study assessments during the course of the trial as determined by the clinical judgment of the Investigator. * Known hypersensitivity to GTX-102 or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects. * Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study

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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

  • AP-HM - Hopital de la Timone

    Marseille, 13385, France

  • AP-HP Hopital Necker-Enfants Malades

    Paris, 75015, France

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • British Columbia Children's Hospital

    Vancouver, British Columbia, V6H 3V4, Canada

  • Childrens Hospital London Health Sciences Centre

    London, Ontario, N6A 5W9, Canada

  • Childrens Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1, Canada

  • Corporacio Sanitaria Parc Tauli - Hospital de Sabadell

    Sabadell, 08208, Spain

  • Great Ormond Street Hospital for Children

    London, WC1N 3JH, United Kingdom

  • Hospital Universitario Puerta de Hierro

    Majadahonda, 28222, Spain

  • MAGIC Clinic Ltd

    Calgary, Alberta, T2E 7Z4, Canada

  • Oxford University Hospitals NHS Foundation Trust

    Oxford, OX3 9DU, United Kingdom

  • Queensland Children's Hospital

    South Brisbane, Queensland, 4101, Australia

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30329, United States

  • Rush University Medical Center

    Chicago, Illinois, 60612, United States

  • Sheba Medical Center

    Ramat Gan, 5265601, Israel

  • The Research Institute of the McGill University Health Centre

    Montreal, Quebec, H3G 1A4, Canada

  • The Royal Children's Hospital

    Melbourne, Victoria, 3052, Australia

  • Universitaetsklinikum Hamburg-Eppendorf

    Hamburg, 20246, Germany

  • Universitaetsklinikum Leipzig

    Leipzig, 04103, Germany

  • University of California, Los Angeles (UCLA)

    Los Angeles, California, 90095, United States

  • University of California, San Diego - Rady Children's Hospital

    San Diego, California, 92123, United States

  • University of Cambridge

    Cambridge, CB2 0QQ, United Kingdom

  • Weill Cornell Medicine

    New York, New York, 10021, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.