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Mucopolysaccharidosis Type IIIA - 18 Studies Found

Status Study
Completed Study Name: A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Condition: Sanfilippo Syndrome Type A
Date: 2010-01-11
Interventions: Other: assessment Physical, developmental, neurological, behavioral, and neurocognitive assessments
Completed Study Name: Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
Condition: Sanfilippo Syndrome Type A (MPS IIIA)
Date: 2010-06-24
Interventions:
  • Biological: Recombinant human heparan N-sulfatase

Completed Study Name: Natural History Studies of Mucopolysaccharidosis III
Condition:
  • Mucopolysaccharidosis Type IIIA
  • Mucopolysaccharidosis Type IIIB
    Date: 2013-11-26
    Interventions:
    • Procedure: Lumbar puncture
    • Device: Magneti
Completed Study Name: Neurobehavioral Phenotypes in MPS III
Condition:
  • Sanfilippo Syndrome Type A
  • Sanfilippo Syndrome Type B
  • Date: 2013-05-28
Recruiting Study Name: Natural History Study of Patients With MPS IIIA
Condition: Mucopolysaccharidosis IIIA
Date: 2016-04-15
Completed Study Name: Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
Condition: Sanfilippo Syndrome
Date: 2014-02-10
Interventions: Drug: Recombinant human heparan N-sulfatase [rhHNS] Recombinant human heparan N-sulfatase [rhHNS]
Recruiting Study Name: Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
Condition: Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome
Date: 2016-03-17
Interventions: Biological: scAAV9.U1a.hSGSH Self-complementary adeno-associated virus serotype 9 carrying the human SGS
Active, not recruiting Study Name: Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Condition: Sanfilippo Syndrome
Date: 2011-02-16
Interventions:
  • Biological: rhHNS-10 mg Once p
Recruiting Study Name: A Treatment Study of Mucopolysaccharidosis Type IIIB
Condition:
  • MPS III B
  • Mucopolysaccharidosis Type IIIB

Date: 2016-02-27
Interventions: Drug: BMN 250 Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human ins
Active, not recruiting Study Name: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
Condition:
  • Mucopolysaccharidosis Type III A
  • Sanfilippo Disease Type A

    • Date: 2014-01-16
    Interventions: Genetic: SAF-301
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