Mucopolysaccharidosis III - 17 Studies Found

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
:
  • Biological: rhHNS-10 mg Once p
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 : Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
: Sanfilippo Syndrome Type B
: 2012-01-10
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
:
  • Biological: Recombinant human heparan N-sulfatase

Active, not recruiting : 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
:
  • Mucopolysaccharidosis III, Type B (MPS IIIB)
  • Sanfilippo B

    • : 2015-10-21
    : Drug: SBC-103
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]
Completed : A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
: MPS IIIB (Sanfilippo Syndrome)
: 2014-11-03
Active, not recruiting : Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
:
  • Mucopolysaccharidosis Type III A
  • Sanfilip
    : 2014-01-16
    : Genetic: SAF-301
Completed : Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
:
  • Mucopolysaccharidosis Type III A
  • Sanfilip
    : 2011-11-10
    : Genetic: SAF-301 The treatment plan consists on a direct injection of the investigational medicinal prod
Completed : Biomarker for Sanfilippo Disease
:
  • Mucopolysaccharidosis Type 3 A
  • Mucopolysaccharidosis Type 3 B