ION582 for Angelman Syndrome

Recruiting in Palo Alto (17 mi)

+10 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Ionis Pharmaceuticals, Inc.

No Placebo Group

Trial Summary

What is the purpose of this trial?

This trial is testing a drug called ION582, given directly into the spinal fluid, to see if it is safe for people with Angelman syndrome. The study will check how well patients tolerate different doses of the drug.

Eligibility Criteria

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.

Inclusion Criteria

I have been on a stable treatment plan for my condition, including medications and diets, for at least 3 months.

I am between 2 and 50 years old and have consent from my parent or guardian.

I have been diagnosed with Angelman syndrome.

Exclusion Criteria

My condition is confirmed to be due to specific genetic changes.

Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion or could interfere with the participant taking part in or completing the study

I have previously undergone gene therapy.

Participant Groups

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.

Group I: Part 3 Group 2 Experimental Treatment 1 Intervention

ION582 will be administered as IT injection of over a period of 145 weeks, with additional dosing intervals.

Group II: Part 3 Group 1 Experimental Treatment 1 Intervention

ION582 will be administered as IT injection of over a period of 145 weeks, with additional dosing intervals.

Group III: Part 2 Group 2 Experimental Treatment 1 Intervention

ION582 will be administered as IT injection of over a period of 49 weeks, with additional dosing intervals.

Group IV: Part 2 Group 1 Experimental Treatment 1 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 E Experimental Treatment 1 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 D Experimental Treatment 1 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 C Experimental Treatment 1 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 B Experimental Treatment 1 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 A Experimental Treatment 1 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 Clinic Near You

Research Locations Nearby Select from list below to view details:

Rady Children's Hospital San Diego, CA

Colorado Children's Hospital Research Institute Aurora, CO

Rush University Medical Center Chicago, IL

Boston Children's Hospital Boston, MA

More Trial Locations

Who Is Running the Clinical Trial?

Ionis Pharmaceuticals, Inc. Lead Sponsor

Biogen Industry Sponsor

References