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Tests treatment safety and results for Angelman Syndrome
Official title HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
ClinicalTrials.gov ID: NCT05127226
What this study is testing
What is ION582?
ION582 is an investigational medicine, given as a treatment applied to the skin, being studied as a potential treatment for angelman syndrome.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 50
You may be able to join if
- Participant has a documented and certified diagnosis of Angelman syndrome (AS) (ubiquitin-protein ligase E3A [UBE3A] deletion or UBE3A mutation)
- Male or female between the ages of 0-50 years of age, with signed informed consent from parent(s) or legal guardian(s)
- Currently receiving stable standard of care treatments such as, stable doses of anti-epileptic medication, behavioral management medications, sleep...
- Follow good study practice and not participate in the sharing of personal or study information on social media platforms, such as any website or...
You likely can't join if
- Has documented molecular AS confirmation of paternal uniparental disomy (UPD) or imprinting defect (ID).
- Any clinically significant (CS) cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic...
- Known bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture. Previous treatment with an...
- Any prior use of gene therapy. Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for...
The study team makes the final eligibility decision.
Where it's taking place
- San Diego, California, United States
- Aurora, Colorado, United States
- Chicago, Illinois, United States
- Boston, Massachusetts, United States
- Carrboro, North Carolina, United States
- Houston, Texas, United States
- Randwick, Australia
- Paris, France
- Ramat Gan, Israel
- Pisa, Italy
- Oxford, Oxfordshire, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0 years to 50 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Diego, California, United States; Aurora, Colorado, United States; Chicago, Illinois, United States; Boston, Massachusetts, United States; Carrboro, North Carolina, United States; Houston, Texas, United States and 5 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.