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

NCT06150716 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2025-12-12

No results posted yet for this study

Summary

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

Conditions

  • Pelizaeus-Merzbacher Disease

Interventions

DRUG

ION356

Administered as intrathecal (IT) injection.

Sponsors & Collaborators

  • Ionis Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
2 Years
Max Age
17 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-10
Primary Completion
2028-06-30
Completion
2028-06-30
FDA Drug
Yes

Countries

  • United States
  • France
  • Germany
  • Japan
  • Netherlands

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06150716 on ClinicalTrials.gov