A Phase 1b Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION356 in Patients With Pelizaeus Merzbacher Disease
Status: Recruiting
Location: See all (7) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
SUMMARY
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
Eligibility
Participation Requirements
Sex: Male
Minimum Age: 2
Maximum Age: 17
Healthy Volunteers: f
View:
• 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.
• Diagnosis of PMD with genetic confirmation of PLP1 gene duplication.
• Clinical phenotype and brain imaging consistent with a diagnosis of PMD.
• Male between the ages of 2 and 17 years, inclusive, at the time of informed consent.
• Able and willing to meet all study requirements (in the opinion of the Investigator), including travel to Study Center, procedures, measurements, and visits.
Locations
United States
Georgia
Ionis Investigative Site
RECRUITING
Atlanta
Pennsylvania
Ionis Investigative Site
RECRUITING
Philadelphia
Utah
Ionis Investigative Site
RECRUITING
Salt Lake City
Other Locations
France
Ionis Investigative Site
RECRUITING
Le Kremlin-bicêtre
Germany
Ionis Investigative Site
RECRUITING
Göttingen
Japan
Ionis Investigative Site
RECRUITING
Tokyo
Netherlands
Ionis Investigative Site
RECRUITING
Amsterdam
Contact Information
Primary
Ionis Pharmaceuticals, Inc.
IonisPelizaeusMerzbacherStudy2@clinicaltrialmedia.com
(844) 387-9520
Time Frame
Start Date: 2024-04-10
Estimated Completion Date: 2028-06
Participants
Target number of participants: 24
Treatments
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.
Related Therapeutic Areas
Sponsors
Leads: Ionis Pharmaceuticals, Inc.