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Clinical Trials in the USA / NCT05127226
Active, not recruiting Phase 1/2

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

NCT05127226 · tracked via the Priya Life Science USA tracker
Sponsor
Ionis Pharmaceuticals, Inc.
Phase
Phase 1/2
Started
2021-12-22
Last updated
2026-08-20

Condition(s) studied

Angelman Syndrome

Investigational drug(s) / intervention(s)

ION582

ION582: ION582 will be administered by IT injection.

Study summary

The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.

Eligibility

Sex
ALL
Min age
0 Years
Max age
50 Years
Healthy volunteers
No
Key Inclusion Criteria: 1. Participant has a documented and certified diagnosis of Angelman syndrome (AS) (ubiquitin-protein ligase E3A \[UBE3A\] deletion or UBE3A mutation) 2. Male or female between the ages of 0-50 years of age, with signed informed consent from parent(s) or legal guardian(s) 3. Currently receiving stable standard of care treatments such as, stable doses of anti-epileptic medication, behavioral management medications, sleep medications, gabapentin, cannabidiol, and including special diets, supplements or nutritional support for at least 3 months prior to first dose. 4. Follow good study practice and not participate in the sharing of personal or study information on social media platforms, such as any website or social media site (e.g., Facebook, Instagram, Twitter, YouTube, etc.) until notified that the study is completed. Key Exclusion Criteria: 1. Has documented molecular AS confirmation of paternal uniparental disomy (UPD) or imprinting defect (ID). 2. Any clinically significant (CS) cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in the judgment of the Investigator, will pose a safety risk, will make the patient unsuitable for participation in, and/or unable to complete the study procedures. Has poorly controlled seizures as determined by the Investigator or has documented Status Epilepticus in the past 6 months that could pose a safety risk while on study. 3. Known bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture. Previous treatment with an oligonucleotide (including small interfering ribonucleic acid, antisense oligonucleotide \[ASOs\]). COVID-19 vaccinations are allowed. 4. Any prior use of gene therapy. Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion or could interfere with the participant taking part in or completing the study.

Primary outcome measure(s)

Trial sites (11)

FacilityCityRegionStatus
Rady Children's Hospital San Diego California
Colorado Children's Hospital Research Institute Aurora Colorado
Rush University Medical Center Chicago Illinois
Boston Children's Hospital Boston Massachusetts
University of North Carolina at Chapel Hill School of Medicine Carrboro North Carolina
Texas Children's Hospital Houston Texas
Sydney Children's Hospital, Kids Cancer Centre Randwick Australia
Necker-Enfants Malades Hospital Paris France
Sheba Medical Center Ramat Gan Israel
Azienda Ospedaliera Universitaria Pisana Pisa Italy
STRONG Group University of Oxford Oxford Oxfordshire

More Ionis Pharmaceuticals, Inc. trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05127226 on ClinicalTrials.gov ↗ ← All trials in the USA