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

Part of paid clinical trials in San Diego, California.

Sponsor
Ionis Pharmaceuticals, Inc.
Study ID
NCT05127226
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Angelman Syndrome

Eligibility Criteria

Sex
ALL
Age
0 Years - 50 Years
Healthy Volunteers
Not accepted

Interventions

  • ION582 — DRUG
    ION582 will be administered by IT injection.

Study Details

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

Key Dates

First listed
Nov 19, 2021
Start date
Dec 22, 2021
Status verified
Nov 2025
Primary completion
Mar 31, 2029
Completion
Mar 31, 2029

Study Design

Enrollment
70 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Part 1 MAD: Cohort A
    ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration.
  • Experimental: Part 1 MAD: Cohort B
    ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration.
  • Experimental: Part 1 MAD: Cohort C
    ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration.
  • Experimental: Part 1 MAD: Cohort D
    ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration.
  • Experimental: Part 1 MAD: Cohort E
    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.
  • Experimental: Part 1 MAD: Cohort F
    ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 12 weeks between each dose administration.
  • Experimental: Part 2 Group 1
    ION582 will be administered as IT injection of over a period of 49 weeks, with additional dosing intervals.
  • Experimental: Part 2 Group 2
    ION582 will be administered as IT injection of over a period of 49 weeks, with additional dosing intervals.
  • Experimental: Part 3 Group 1
    ION582 will be administered as IT injection of over a period of 145 weeks, with additional dosing intervals.
  • Experimental: Part 3 Group 2
    ION582 will be administered as IT injection of over a period of 145 weeks, with additional dosing intervals.

Primary Outcome Measure

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). [ Time Frame: Part 1: Up to Week 45; Part 2: Up to Week 81 ]

Central Contacts

Locations (6)

FacilityCityStateZIPSite coordinators
Rady Children's HospitalSan DiegoCalifornia92123-
Colorado Children's Hospital Research InstituteAuroraColorado80045-
Rush University Medical CenterChicagoIllinois60612-
Boston Children's HospitalBostonMassachusetts02215-
University of North Carolina at Chapel Hill School of MedicineCarrboroNorth Carolina27510-
Texas Children's HospitalHoustonTexas77030-

Find similar trials in San Diego, CA

By research site
Rady Children's Hospital· San Diego, CAColorado Children's Hospital Research Institute· Aurora, CORush University Medical Center· Chicago, ILBoston Children's Hospital· Boston, MAUniversity of North Carolina at Chapel Hill School of Medicine· Carrboro, NCTexas Children's Hospital· Houston, TX

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