ION582 for Angelman Syndrome

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

Recruiting Phase 1 Phase 2 Interventional Study
Angelman Syndrome
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
0 – 50
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
70 (estimated)
Sponsor
Ionis Pharmaceuticals, Inc. · Industry
Who this trial is looking for

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.

Are You a Good Fit for This Trial?
Must be taking Anti-epileptic medicationBehavioral management medicationsSleep medicationsGabapentinCannabidiol

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.

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About This Trial

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

Trial Locations
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Eligibility Criteria
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. …
Contacts

Ionis Pharmaceuticals

(844) 200-6263

IonisAngelmanStudy@clinicaltrialmedia.com

CONTACT