NCT06150716View on ClinicalTrials.gov

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

PHASE1RecruitingINTERVENTIONAL
Enrollment

24

Participants

Timeline

Start Date

April 10, 2024

Primary Completion Date

June 30, 2028

Study Completion Date

June 30, 2028

Conditions
Pelizaeus-Merzbacher Disease
Interventions
DRUG

ION356

Administered as intrathecal (IT) injection.

Trial Locations (7)

19104

RECRUITING

Ionis Investigative Site, Philadelphia

30342

RECRUITING

Ionis Investigative Site, Atlanta

37075

RECRUITING

Ionis Investigative Site, Göttingen

84113

RECRUITING

Ionis Investigative Site, Salt Lake City

94270

RECRUITING

Ionis Investigative Site, Le Kremlin-Bicêtre

187-8551

RECRUITING

Ionis Investigative Site, Tokyo

1105AZ

RECRUITING

Ionis Investigative Site, Amsterdam

Sponsors
All Listed Sponsors
lead

Ionis Pharmaceuticals, Inc.

INDUSTRY