Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

Part of paid clinical trials in Atlanta, Georgia.

Sponsor
Ionis Pharmaceuticals, Inc.
Study ID
NCT06150716
Phase
PHASE1
Status
Recruiting
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Conditions

  • Pelizaeus-Merzbacher Disease

Eligibility Criteria

Sex
MALE
Age
2 Years - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • ION356 — DRUG
    Administered as intrathecal (IT) injection.

Study Details

The primary purpose of this study is to evaluate the safety and tolerability of ION356.

Key Dates

Start date
Apr 10, 2024
Status verified
Dec 2025
Primary completion
Jun 30, 2028
Completion
Jun 30, 2028

Study Design

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

Arms

  • Experimental: Cohort A: ION356 Dose A
    Participants will receive ION356 intrathecally at Dose A in the MAD Period, followed by ION356 Dose A in the LTE Period.
  • Experimental: Cohort B: ION356 Dose B
    Participants will receive ION356 intrathecally at Dose B in the MAD Period, followed by ION356 Dose B in the LTE Period.

Primary Outcome Measure

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs [ Time Frame: Up to Week 145 ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Ionis Investigative SiteAtlantaGeorgia30342-
Ionis Investigative SitePhiladelphiaPennsylvania19104-
Ionis Investigative SiteSalt Lake CityUtah84113-

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By research site
Ionis Investigative Site· Atlanta, GAIonis Investigative Site· Philadelphia, PAIonis Investigative Site· Salt Lake City, UT

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