Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)

Sponsor
Ionis Pharmaceuticals, Inc.
Study ID
NCT04165486
Phase
PHASE1
Status
Recruiting
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Conditions

  • Multiple System Atrophy

Eligibility Criteria

Sex
ALL
Age
40 Years - 70 Years
Healthy Volunteers
Not accepted

Interventions

  • ION464 — DRUG
    ION464 will be administered by IT injection.
  • Placebo — DRUG
    ION464-matching placebo will be administered by IT injection.

Study Details

The primary objectives are to evaluate the safety and tolerability of multiple doses of ION464 administered via intrathecal (IT) injection (Part 1) and to evaluate the long-term safety and tolerability of ION464 (Part 2) in participants with multiple system atrophy (MSA). The secondary objectives are to evaluate the pharmacodynamic (PD) effect of ION464 on the level of a potential biomarker of target engagement (Parts 1 and 2) and to evaluate the pharmacokinetic (PK) profile of ION464 in serum (Part 1).

Key Dates

First listed
Nov 18, 2019
Start date
Jul 21, 2022
Status verified
Apr 2026
Primary completion
Sep 30, 2027
Completion
Sep 30, 2027

Study Design

Enrollment
40 participants (estimated)
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Part 1: ION464
    ION464 will be administered at multiple-ascending doses by IT injection at regular intervals over 12 weeks.
  • Placebo Comparator: Part 1: Placebo
    ION464-matching placebo will be administered by IT injection at regular intervals over 12 weeks.
  • Experimental: Part 2: ION464
    ION464 will be administered at the same doses as Part 1 by IT injection, at regular intervals, for 72 weeks.
  • Placebo Comparator: Part 2: Placebo
    ION464-matching placebo will be administered by IT injection, at regular intervals, for 72 weeks.

Primary Outcome Measure

Number of Participants with Adverse Events (AEs) [ Time Frame: Baseline up to approximately 36 weeks ]

Central Contacts

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