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Antisense Oligonucleotide
ION582 for Angelman Syndrome
Phase 1 & 2
Waitlist Available
Research Sponsored by Ionis Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Male or female between the ages of 2-50 years of age, with signed informed consent from parent(s) or legal guardian(s)
Participant has a documented and certified diagnosis of Angelman syndrome (AS) (ubiquitin-protein ligase E3A [UBE3A] deletion or UBE3A mutation)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up part 1: up to week 45; part 2: up to week 81
Awards & highlights
Study Summary
This trial will test if a new drug is safe and tolerable for people with Angelman syndrome.
Who is the study for?
This trial is for individuals aged 2-50 with a certified diagnosis of Angelman syndrome, who have been on stable standard care treatments and medications for at least 3 months. They must not share study info on social media until the study ends. Excluded are those with certain genetic profiles, risks from lumbar puncture, previous oligonucleotide treatment or gene therapy, uncontrolled seizures, or other significant health issues.Check my eligibility
What is being tested?
The HALOS trial is testing ION582 given intrathecally (directly into the spinal canal) to see how safe it is and how well tolerated in different doses among participants with Angelman syndrome. The focus is on understanding the drug's effects and how it moves through and out of the body.See study design
What are the potential side effects?
While specific side effects aren't listed here, common ones related to intrathecal administration may include headache, back pain, nausea or vomiting. There could also be potential reactions at the injection site or systemic effects depending on how ION582 works in the body.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am between 2 and 50 years old and have consent from my parent or guardian.
Select...
I have been diagnosed with Angelman syndrome.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ part 1: up to week 45; part 2: up to week 81
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~part 1: up to week 45; part 2: up to week 81
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
To evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters).
Secondary outcome measures
Concentration ION582 in CSF
Maximum Observed Plasma Concentration (Cmax) of ION582
Plasma Elimination Half-Life (t1/2λz) of ION582
+1 moreTrial Design
9Treatment groups
Experimental Treatment
Group I: Part 3 Group 2Experimental Treatment1 Intervention
ION582 will be administered as IT injection of over a period of 145 weeks, with additional dosing intervals.
Group II: Part 3 Group 1Experimental Treatment1 Intervention
ION582 will be administered as IT injection of over a period of 145 weeks, with additional dosing intervals.
Group III: Part 2 Group 2Experimental Treatment1 Intervention
ION582 will be administered as IT injection of over a period of 49 weeks, with additional dosing intervals.
Group IV: Part 2 Group 1Experimental Treatment1 Intervention
ION582 will be administered as IT injection of over a period of 49 weeks, with additional dosing intervals.
Group V: Part 1 MAD: Cohort EExperimental Treatment1 Intervention
ION582 will be administered as IT injection of over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration.
Group VI: Part 1 MAD: Cohort DExperimental Treatment1 Intervention
ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration.
Group VII: Part 1 MAD: Cohort CExperimental Treatment1 Intervention
ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration.
Group VIII: Part 1 MAD: Cohort BExperimental Treatment1 Intervention
ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration.
Group IX: Part 1 MAD: Cohort AExperimental Treatment1 Intervention
ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration.
Find a Location
Who is running the clinical trial?
Ionis Pharmaceuticals, Inc.Lead Sponsor
146 Previous Clinical Trials
15,231 Total Patients Enrolled
1 Trials studying Angelman Syndrome
73 Patients Enrolled for Angelman Syndrome
BiogenIndustry Sponsor
639 Previous Clinical Trials
467,385 Total Patients Enrolled
1 Trials studying Angelman Syndrome
20 Patients Enrolled for Angelman Syndrome
Media Library
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Does the age requirement for this trial preclude anyone over eighteen?
"This medical trial can only be joined by patients aged 2 to 50. Out of the total 1358 participants, 375 are minors and 983 have surpassed retirement age."
Answered by AI
Who may participate in this clinical exploration?
"This clinical trial is seeking 44 participants aged between 2 and 50 that have been diagnosed with Angelman syndrome. In order to participate, potential volunteers must be receiving a stable regimen of anti-epileptic medications as well as any behavioral management drugs, sleep aids, gabapentin, cannabidiol or nutrition supplements for at least three months prior to enrolment. Additionally, all recruited individuals are obligated to follow best practice procedures and abstain from sharing information about the study on social media websites until completion has been announced."
Answered by AI
What is the current size of the cohort participating in this clinical experiment?
"Affirmative. The information found on clinicaltrials.gov illustrates that this research is currently enrolling individuals for participation. This trial was initiated on December 22, 2021 and recently modified October 13th 2022; it requires 44 participants to be recruited from 6 different sites."
Answered by AI
Are participants being accepted for this investigation presently?
"Correct. Records on clinicaltrials.gov show that this medical trial, which was initiated December 22nd 2021 is currently recruiting volunteers. A total of 44 participants need to be enrolled from 6 different clinics."
Answered by AI
At what sites is this investigation taking place?
"Patients for this trial are being enrolled at Texas Children's Hospital in Houston, Rush University Medical Center in Chicago and Boston Children's Hospital in Massachusetts as well as 6 other sites."
Answered by AI
Who else is applying?
What state do they live in?
California
Tennessee
How old are they?
18 - 65
What site did they apply to?
Rush University Medical Center
Rady Children's Hospital
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?
0
Why did patients apply to this trial?
I found out Angelman Syndrome is no cure. However, I found hope in your study and able to cure my child.
PatientReceived no prior treatments
How responsive is this trial?
Typically responds via
Phone Call
Most responsive sites:
- Rady Children's Hospital: < 24 hours
Average response time
- < 1 Day
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