A Phase 1b Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION356 in Patients With Pelizaeus Merzbacher Disease
Ionis Pharmaceuticals, Inc.
Summary
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
Description
This is a Phase 1b, open-label multiple-ascending dose (MAD) study of ION356 in approximately 24 pediatric participants with Pelizaeus-Merzbacher Disease and genetic confirmation of proteolipid protein 1 (PLP1) gene duplication. The study will have 2 parts: a 48-week multiple-ascending dose (MAD) part followed by a long-term extension (LTE) part of 109 weeks. Eligible participants will receive doses of ION356 during the MAD portion of the study and upon completion will seamlessly transition to the open-label LTE to receive doses of ION356.
Eligibility
- Age range
- 2–17 years
- Sex
- Male
- Healthy volunteers
- No
Key Inclusion Criteria 1. Participant's parent or legally accepted representative can provide informed consent, attend all scheduled study visits, provide feedback regarding the participant's symptoms, and can comply with all study requirements. 2. Diagnosis of PMD with genetic confirmation of PLP1 gene duplication. 3. Clinical phenotype and brain imaging consistent with a diagnosis of PMD. 4. Male between the ages of 2 and 17 years, inclusive, at the time of informed consent. 5. Able and willing to meet all study requirements (in the opinion of the Investigator), including travel to Study Ce…
Interventions
- DrugION356
Administered as intrathecal (IT) injection.
Locations (7)
- Ionis Investigative SiteAtlanta, Georgia
- Ionis Investigative SitePhiladelphia, Pennsylvania
- Ionis Investigative SiteSalt Lake City, Utah
- Ionis Investigative SiteLe Kremlin-Bicêtre
- Ionis Investigative SiteGöttingen
- Ionis Investigative SiteTokyo