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Mucopolysaccharidosis III - 16 Studies Found

Status Study
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
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: Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
Condition: Sanfilippo Syndrome Type B
Date: 2012-01-10
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

Active, not recruiting Study Name: A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously
Condition:
  • Mucopolysaccharidosis III, Type B (MPS IIIB)
  • Sanfilippo B

    • Date: 2015-10-21
    Interventions: Drug: SBC-103
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]
Completed Study Name: A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
Condition: MPS IIIB (Sanfilippo Syndrome)
Date: 2014-11-03
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
  • Sanfilip
    Date: 2014-01-16
    Interventions: Genetic: SAF-301
Completed Study Name: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
Condition:
  • Mucopolysaccharidosis Type III A
  • Sanfilip
    Date: 2011-11-10
    Interventions: Genetic: SAF-301 The treatment plan consists on a direct injection of the investigational medicinal prod
Recruiting Study Name: Biomarker for Sanfilippo Disease
Condition:
  • Mucopolysaccharidosis Type 3 A
  • Mucopolysaccharidosis Type 3 B