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Phase 2

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

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

MPS IIIASanfilippo SyndromeSanfilippo A+1 more

Estimated

~$10 – $8,750

Ultragenyx Pharmaceutical IncNCT02716246

Phase 2

Randomized, Double-Blind, Placebo-Controlled Cross-Over Study to Evaluate the Effects of Cannabidiol on Neurobehavioral and Function Outcomes in Sanfilippo Syndrome

The goal of this clinical trial is to test cannabidiol in Sanfilippo syndrome. The main questions it aims to answer are: 1) determine the safety of cannabidiol…

Sanfilippo SyndromeMucopolysaccharidosis III

Estimated

~$10 – $6,250

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterNCT06333041

Phase 1

An Open-Label, Single Injection, Ascending Dose Phase I/II Clinical Study of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB

The goal of this clinical trial is to learn if one infusion of rAAV9-CMV-hNAGLUop can treat patients with Sanfilippo syndrome (MPS III) 6 months or older. The …

Sanfilippo Syndrome (MPS III)

Estimated

~$10 – $7,500

NeuroGTNCT07818759

Observational Study to Assess Disease Symptoms and Progression in Participants With Sanfilippo Syndrome Type C (Mucopolysaccharidosis Type III [MPS III] C)

This is a REMOTE prospective observational study of participants with MPS IIIC. No investigational medicinal product will be administered as part of this stud…

Sanfilippo Syndrome Type CMucopolysaccharidosis (MPS) IIIC

Estimated

~$10 – $1,125

Phoenix NestNCT07712003

A Combination Retrospective and Prospective Natural History Study of Participants With Sanfilippo Syndrome Mucopolysaccharidosis Type IIIC (MPS IIIC)

This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.

Sanfilippo Syndrome Type C

Estimated

~$10 – $1,125

Phoenix NestNCT05825131

Phase 2

An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III

A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).

Sanfilippo SyndromeMPS3

Estimated

~$10 – $7,500

Ozlem Goker-AlpanNCT06614894