drugset / Trial / NCT02350816

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

NCT02350816 ↗

Phase 2 Terminated 17 enrolled Shire
RandomizedParallel-groupOpen-labelTreatment

Summary

This extension study will allow participants to continue receiving treatment with HGT-1410 and to initiate treatment in patients who received no-treatment in Study HGT-SAN-093, and will evaluate the long-term safety and efficacy of the study drug.

Timeline

Start
2015-04-08
Primary completion
2019-04-12
Completion
2019-04-12

Outcome

Outcome not reported

Stopped: “Completion of follow-up period”

Drugs

EvaluationDrugModalityDoseRoute
Comparator N-SULFOGLUCOSAMINE SULFOHYDROLASE RECOMBINANT Protein / enzyme biologic 45 mg Intrathecal