Mucopolysaccharidosis Type IIIA - 19 Studies Found
Completed |
: A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA) : Sanfilippo Syndrome Type A : 2010-01-11 : Other: assessment Physical, developmental, neurological, behavioral, and neurocognitive assessments |
Completed |
: Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients : Sanfilippo Syndrome Type A (MPS IIIA) : 2010-06-24 :
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Completed |
: Natural History Studies of Mucopolysaccharidosis III :
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Completed |
: Neurobehavioral Phenotypes in MPS III :
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Recruiting |
: Natural History Study of Patients With MPS IIIA : Mucopolysaccharidosis IIIA : 2016-04-15 |
Completed |
: Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease : Sanfilippo Syndrome : 2014-02-10 : Drug: Recombinant human heparan N-sulfatase [rhHNS] Recombinant human heparan N-sulfatase [rhHNS] |
Recruiting |
: Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA : Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome : 2016-03-17 : Biological: scAAV9.U1a.hSGSH Self-complementary adeno-associated virus serotype 9 carrying the human SGS |
Active, not recruiting |
: Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA) : Sanfilippo Syndrome : 2011-02-16 :
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Recruiting |
: A Treatment Study of Mucopolysaccharidosis Type IIIB :
: 2016-02-27 : Drug: BMN 250 Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human ins |
Active, not recruiting |
: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy :
: 2014-01-16 : Genetic: SAF-301 |