RecruitingPhase 1NCT06150716

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

Studying Pelizaeus-Merzbacher disease

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Key facts

Sponsor
Ionis Pharmaceuticals, Inc.
Intervention
ION356(drug)
Enrollment
24 enrolled
Eligibility
2-17 years · MALE
Timeline
20242028

Study locations (7)

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Open NCT06150716 on ClinicalTrials.gov

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