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18 studies found for:    MUCOPOLYSACCHARIDOSIS TYPE IIIA
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Rank Status Study
1 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
2 Recruiting Natural History Study of Patients With MPS IIIA
Condition: Mucopolysaccharidosis IIIA
Intervention:
3 Completed Neurobehavioral Phenotypes in MPS III
Conditions: Sanfilippo Syndrome Type A;   Sanfilippo Syndrome Type B;   Hurler Syndrome
Intervention:
4 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
5 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
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 Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
Conditions: Mucopolysaccharidosis Type III A;   Sanfilippo Disease Type A
Intervention: Genetic: SAF-301
8 Completed 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
9 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]
10 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:
11 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
12 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:
13 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
14 Recruiting A Treatment Study of Mucopolysaccharidosis Type IIIB
Conditions: MPS III B;   Mucopolysaccharidosis Type IIIB
Intervention: Drug: BMN 250
15 Recruiting A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Conditions: Mucopolysaccharidosis Type IIIB;   Mucopolysaccharidosis Type 3 B;   MPS III B;   MPS 3 B
Intervention:
16 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
17 Terminated ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Conditions: Inherited Metabolic Diseases;   Lysosomal Storage Disorders;   Peroxisomal Storage Diseases;   Inborn Errors of Metabolism;   Mucopolysaccharidosis
Intervention: Biological: ALD-101
18 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

Indicates status has not been verified in more than two years