CompletedPHASE1, PHASE2NCT03811028

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

Studying Sanfilippo syndrome type A

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Key facts

Sponsor
Swedish Orphan Biovitrum
Principal Investigator
Paul Harmatz, MD, M.D., Ph.D
Children´s Hospital and Research Center, Oakland
Intervention
SOBI003(drug)
Enrollment
6 enrolled
Eligibility
All sexes
Timeline
20192021

Study locations (3)

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 NCT03811028 on ClinicalTrials.gov

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