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First received: April 14, 2023

Clinical Trial: Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

Study Status: Completed

Recruit Status: RECRUITING

Condition: MPS IVA

Study Type: INTERVENTIONAL


Official Title: A Proof of Concept Study to Evaluate the Safety and Efficacy of Voxzogo (Vosoritide) for the Treatment of Growth Deficits in MPS IVA and VI

Brief Summary: This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 48 weeks to 6 patients with MPS IVA or VI.Prior to enrollment in the interventional arm of study, subjects will be followed for a minimum of 24 weeks to gather information on safety profiles and determine annualized growth velocity.The primary study endpoint is the determination of safety and tolerability of daily vosoritide treatment in MPS.Exploratory endpoints include changes in…

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