RecruitingPhase 2Phase 3NCT02716246

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

Sponsor

Ultragenyx Pharmaceutical Inc

Enrollment

36 participants

Start Date

Apr 25, 2016

Study Type

INTERVENTIONAL

Conditions

Mucopolysaccharidosis III MPS IIIA Sanfilippo Syndrome Sanfilippo A

Summary

The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.

Eligibility

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Adjuvant IM Therapy, a drug called Optimized Prophylactic IM Therapy, and others for people with mps iiia, mucopolysaccharidosis iii, and other related conditions. The study is currently recruiting participants at 5 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

BIOLOGICALUX111

Self-complementary adeno-associated virus serotype 9 carrying the human SGSH gene under the control of a U1a promoter (scAAV9.U1a.hSGSH) will be delivered one time through a venous catheter inserted into a peripheral limb vein.

DRUGProphylactic Immunomodulatory (IM) Therapy

DRUGOptimized Prophylactic IM Therapy

DRUGAdjuvant IM Therapy

The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive adjuvant IM therapy.