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3 recruiting studies matching your filters
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
A Global, Open-Label, Phase I/II Trial, to Evaluate Safety, Tolerability, Pharmacodynamic and Preliminary Efficacy of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), des…
Mucopolysaccharidosis IIIB
Estimated
~$10 – $8,750
JCR Pharmaceuticals Co., Ltd.NCT07640984
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