ION582 for Angelman Syndrome
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
- Age
- 0 – 50
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 70 (estimated)
- Sponsor
- Ionis Pharmaceuticals, Inc. · Industry
This trial is looking for adults and children diagnosed with Angelman syndrome to find out if increasing doses of a new treatment, ION582, are safe to use. Participants will receive the treatment through a procedure to inject it into their spine.
You may be able to join if
- I have a diagnosis of Angelman syndrome.
- I am between 0 and 50 years old.
- I have my parent’s or legal guardian’s permission.
- I have been taking stable standard treatments for at least 3 months.
- I will not share personal or study information on social media.
You may not be able to join if
- I have a paternal uniparental disomy or imprinting defect.
- I have serious health conditions that could make participation unsafe.
- I have poorly controlled seizures or had Status Epilepticus in the past 6 months.
- I have a condition that could make a lumbar puncture risky.
- I have had previous treatment with oligonucleotides.
- I have used gene therapy before.
- I have any other health issues that would make me unsuitable for the study.
Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your EligibilityThe purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.
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