Actively Recruiting
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
Led by Ionis Pharmaceuticals, Inc. · Updated on 2025-11-14
70
Participants Needed
11
Research Sites
375 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.
CONDITIONS
Official Title
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Documented diagnosis of Angelman syndrome with UBE3A deletion or mutation
- Male or female aged 0 to 50 years with signed informed consent from parent or legal guardian
- Receiving stable standard of care treatments (e.g., anti-epileptic, behavioral, sleep medications, gabapentin, cannabidiol, special diets, supplements) for at least 3 months before first dose
- Agree to follow study rules and not share personal or study information on social media until study completion
You will not qualify if you...
- Molecular confirmation of Angelman syndrome due to paternal uniparental disomy or imprinting defect
- Significant cardiovascular, endocrine, liver, kidney, lung, gastrointestinal, neurological, malignant, metabolic, psychiatric, or other conditions posing safety risks or interfering with study participation
- Poorly controlled seizures or status epilepticus in past 6 months posing safety risk
- Known bone, spine, bleeding, or other disorders increasing risk for lumbar puncture injury
- Previous treatment with oligonucleotides including small interfering RNA or antisense oligonucleotides
- Any prior use of gene therapy
- Any other conditions judged by the investigator to make participation unsuitable or interfere with study completion
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 11 locations
1
Rady Children's Hospital
San Diego, California, United States, 92123
Actively Recruiting
2
Colorado Children's Hospital Research Institute
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Rush University Medical Center
Chicago, Illinois, United States, 60612
Actively Recruiting
4
Boston Children's Hospital
Boston, Massachusetts, United States, 02215
Actively Recruiting
5
University of North Carolina at Chapel Hill School of Medicine
Carrboro, North Carolina, United States, 27510
Actively Recruiting
6
Texas Children's Hospital
Houston, Texas, United States, 77030
Active, Not Recruiting
7
Sydney Children's Hospital, Kids Cancer Centre
Randwick, Australia, NSW 2031
Active, Not Recruiting
8
Necker-Enfants Malades Hospital
Paris, France, 75015
Actively Recruiting
9
Sheba Medical Center
Ramat Gan, Israel, 5262100
Actively Recruiting
10
Azienda Ospedaliera Universitaria Pisana
Pisa, Italy, 56126
Actively Recruiting
11
STRONG Group University of Oxford
Oxford, Oxfordshire, United Kingdom, OX3 9DU
Active, Not Recruiting
Research Team
I
Ionis Pharmaceuticals
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
10
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