Actively Recruiting
HALOS: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients With Angelman Syndrome
Led by Ionis Pharmaceuticals, Inc. · Updated on 2025-11-14
70
Participants Needed
11
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerability of increasing doses of ION582, given by injection into the spinal fluid, in individuals with Angelman syndrome. This open-label Phase 1-2a study aims to understand how the body processes ION582 and monitor any potential side effects in approximately 70 participants aged from birth to 50 years. Angelman syndrome is a genetic condition characterized by developmental delays and neurological challenges. The study consists of three parts. Part 1 involves multiple ascending doses of ION582 administered over 13 weeks with at least 4 weeks between doses, followed by a minimum 12-week follow-up. Part 2 includes additional dosing over 49 weeks with a similar follow-up period. Part 3 offers extended treatment for up to three more years, followed by a 32-week safety follow-up. Participants receive ION582 as intrathecal injections during these periods. Participants will undergo regular safety assessments, laboratory tests, and monitoring of drug levels in blood and spinal fluid throughout the study. Researchers will track adverse effects, pharmacokinetics, and pharmacodynamics of ION582 up to week 81 in Parts 1 and 2. The study also includes long-term follow-up to observe ongoing effects and safety. Total participation time can extend up to several years depending on the study part.
CONDITIONS
Brief 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...
- Participant has a documented diagnosis of Angelman syndrome with UBE3A deletion or mutation
- Male or female between 0 and 50 years old with signed informed consent from parent(s) or legal guardian(s)
- Currently on stable standard treatments (anti-epileptic, behavioral, sleep medications, gabapentin, cannabidiol, special diets, supplements) for at least 3 months before first dose
- Will follow study rules and not share personal or study information on social media until study completion
You will not qualify if you...
- Confirmed Angelman syndrome due to paternal uniparental disomy or imprinting defect
- Any significant cardiovascular, endocrine, liver, kidney, lung, gastrointestinal, neurological, malignant, metabolic, psychiatric, or other condition posing safety risk or making study completion unlikely
- Poorly controlled seizures or status epilepticus in past 6 months
- Known bone, spine, bleeding, or other disorders increasing risk during lumbar puncture
- Previous treatment with oligonucleotides or gene therapy
- Any other condition making participation unsuitable or interfering with study completion
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 13 weeks
Participants receive multiple ascending doses of ION582 administered by intrathecal injection over a period of 13 weeks, with at least 4 weeks between doses.
Multiple visits for dosing approximately every 4 weeks during treatment
Duration - Minimum 12 weeks
Participants are monitored for safety and tolerability following the multiple ascending dose treatment period.
Follow-up visits during the minimum 12-week monitoring period
Duration - 49 weeks
Participants who completed Part 1 receive intrathecal bolus doses of ION582 over a 49-week period with additional dosing intervals.
Multiple dosing visits during 49-week treatment period
Duration - Minimum 12 weeks
Participants are monitored for at least 12 weeks following completion of the 49-week treatment period to assess safety and tolerability.
Follow-up visits during the minimum 12-week post-treatment monitoring period
Duration - Up to 145 weeks
Participants who completed Part 2 continue receiving intrathecal doses of ION582 for up to an additional 3 years with ongoing assessments.
Regular dosing visits during extended treatment period
Duration - 32 weeks
Participants are monitored for safety and tolerability for 32 weeks after completing the extended treatment period.
Follow-up visits during the 32-week post-treatment monitoring period
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
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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