27 studies found for:    "mucopolysaccharidosis type III"
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Rank Status Study
1 Recruiting Biomarker for Sanfilippo Disease
Conditions: Mucopolysaccharidosis Type 3 A;   Mucopolysaccharidosis Type 3 B;   Mucopolysaccharidosis Type 3 C;   Mucopolysaccharidosis Type 3 D;   Heparan Sulfate Sulfatase Deficiency
Intervention:
2 Active, not recruiting Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
Conditions: Mucopolysaccharidosis Type III A;   Sanfilippo Disease Type A
Intervention: Genetic: SAF-301
3 Completed Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
Conditions: Mucopolysaccharidosis Type III A;   Sanfilippo Disease Type A
Intervention: Genetic: SAF-301
4 Recruiting Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
Conditions: Mucopolysaccharidosis Type I (MPS I);   Mucopolysaccharidosis Type II (MPS II);   Mucopolysaccharidosis Type III (MPS III);   Mucopolysaccharidosis Type VI (MPS VI);   Krabbe Disease
Intervention:
5 Active, not recruiting Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Condition: Sanfilippo Syndrome
Interventions: Biological: rhHNS-10 mg;   Biological: rhHNS-45 mg;   Biological: rhHNS-90 mg
6 Completed
Has Results
A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Condition: Sanfilippo Syndrome Type A
Intervention: Other: assessment
7 Completed Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
Condition: Sanfilippo Syndrome Type B
Intervention:
8 Completed Neurobehavioral Phenotypes in MPS III
Conditions: Sanfilippo Syndrome Type A;   Sanfilippo Syndrome Type B;   Hurler Syndrome
Intervention:
9 Completed Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
Condition: Sanfilippo Syndrome Type A (MPS IIIA)
Intervention: Biological: Recombinant human heparan N-sulfatase
10 Enrolling by invitation Safety and Efficacy Study for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093
Conditions: Sanfilippo Syndrome Type A (MPS IIIA);   Mucopolysaccharidosis III
Intervention: Drug: HGT-1410
11 Completed HSCT for High Risk Inherited Inborn Errors
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Tay Sachs Disease;   Sandhoffs Disease;   Wolman Disease;   I-Cell Disease;   Sanfilippo Syndrome;   GM1 Gangliosidosis
Interventions: Drug: Clofarabine;   Procedure: Total body Irradiation;   Drug: Melphalan;   Biological: Hematopoietic Stem Cell Transplantation;   Drug: Alemtuzumab;   Drug: mycophenylate mofetil;   Device: Cyclosporine A;   Drug: Hydroxyurea
12 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
Condition: Sanfilippo Syndrome
Intervention: Drug: Recombinant human heparan N-sulfatase [rhHNS]
13 Recruiting A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Conditions: Mucopolysaccharidosis Type IIIB;   Mucopolysaccharidosis Type 3 B;   MPS III B;   MPS 3 B
Intervention:
14 Completed A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
Condition: MPS IIIB (Sanfilippo Syndrome)
Intervention:
15 Active, not recruiting Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
Conditions: Hurler Syndrome (MPS I);   Hurler-Scheie Syndrome With Early Neurologic Involvement and/or Sensitization to Enzyme Replacement Therapy (ERT);   Hunter Syndrome (MPS II);   Sanfilippo Syndrome (MPS III);   Krabbe Disease (Globoid Leukodystrophy);   Metachromatic Leukodystrophy (MLD);   Adrenoleukodystrophy (ALD and AMN);   Sandhoff Disease;   Tay Sachs Disease;   Pelizaeus Merzbacher (PMD);   Niemann-Pick Disease;   Alpha-mannosidosis
Intervention: Biological: Enriched Hematopoetic Stem Cell Transplantation
16 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
Conditions: Mucopolysaccharidosis III, Type B (MPS IIIB);   Sanfilippo B
Intervention: Drug: SBC-103
17 Completed Natural History Studies of Mucopolysaccharidosis III
Conditions: Mucopolysaccharidosis Type IIIA;   Mucopolysaccharidosis Type IIIB
Interventions: Procedure: Lumbar puncture;   Device: Magnetic Resonance Imaging (MRI) of the brain
18 Recruiting Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
Condition: Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome
Intervention: Genetic: scAAV9.U1a.hSGSH
19 Recruiting A Treatment Study of Mucopolysaccharidosis Type IIIB
Conditions: MPS III B;   Mucopolysaccharidosis Type IIIB
Intervention: Drug: BMN 250
20 Active, not recruiting Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in MPS IIIB
Condition: Mucopolysaccharidosis IIIB
Intervention: Drug: SBC-103

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Indicates status has not been verified in more than two years