Mucopolysaccharidosis III: all 58 studies

Page 1 of 1. Open to participants first (16), then studies with results posted, then the rest; newest registration first within each.

• MPS IIIA Disease Monitoring ProgramNot yet recruitingNCT07855562
• Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIBNot yet recruitingNCT07818759Phase 1/2
• Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)AvailableNCT07733856
• Natural History to Assess Disease in Patients With MPS IIICNot yet recruitingNCT07712003
• Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)Not yet recruitingNCT07579910Phase 3
• An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS IIIRecruitingNCT06614894Phase 2/3
• Study of Cannabidiol in Sanfilippo SyndromeRecruitingNCT06333041Phase 2/3
• Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)RecruitingNCT06181136Phase 1/2
• MPS (RaDiCo Cohort) (RaDiCo-MPS)RecruitingNCT06036693
• Natural History Study of Participants With Sanfilippo Syndrome Type IIICRecruitingNCT05825131
• The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)RecruitingNCT05705674
• Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLEEnrolling by invitationNCT05492799Phase 4
• ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramEnrolling by invitationNCT05368038
• Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIAEnrolling by invitationNCT04360265Phase 3
• Longitudinal Study of Neurodegenerative DisordersRecruitingNCT03333200
• Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHRecruitingNCT02716246Phase 2/3
• Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA DiseaseTerminatedNCT04088734Phase 1/2Results posted
• A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA PatientsCompletedNCT03811028Phase 1/2Results posted
• A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA PatientsCompletedNCT03423186Phase 1/2Results posted
• 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 IntravenouslyTerminatedNCT02618512Phase 1/2Results posted
• An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.TerminatedNCT02350816Phase 2Results posted
• Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)CompletedNCT02324049Phase 1/2Results posted
• Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA DiseaseCompletedNCT02060526Phase 2Results posted
• Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal DisordersCompletedNCT01626092Not applicableResults posted
• Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease NetworkCompletedNCT01586871Results posted
• Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)TerminatedNCT01299727Phase 1/2Results posted
• Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA PatientsCompletedNCT01155778Phase 1/2Results posted
• A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)CompletedNCT01047306Results posted
• HSCT for High Risk Inherited Inborn ErrorsCompletedNCT00383448Phase 2Results posted
• Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)Active, not recruitingNCT06567769Phase 1
• An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIBActive, not recruitingNCT06488924Phase 1/2
• Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIAActive, not recruitingNCT06095388Phase 1/2
• A Natural History Study of Sanfilippo Syndrome Type DCompletedNCT05648851
• A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)CompletedNCT05523206
• Natural History Observational Study of MPS IIIa in SMCStatus unknownNCT04918641
• A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101TerminatedNCT04655911
• Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIAActive, not recruitingNCT04201405Phase 1/2
• Open-label Study of Anakinra in MPS IIICompletedNCT04018755Phase 1/2
• A Treatment Extension Study of Mucopolysaccharidosis Type IIIBStatus unknownNCT03784287Phase 2
• Early Check: Expanded Screening in NewbornsActive, not recruitingNCT03655223
• Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)Status unknownNCT03612869Phase 2/3
• Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIBTerminatedNCT03315182Phase 1/2
• Intracerebral Gene Therapy in Children With Sanfilippo Type B SyndromeCompletedNCT03300453Phase 1/2
• A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)Active, not recruitingNCT03227042
• Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-IIICompletedNCT03161171
• A Treatment Study of Mucopolysaccharidosis Type IIIBCompletedNCT02754076Phase 1/2
• A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)CompletedNCT02493998
• Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIBTerminatedNCT02293408
• A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIBCompletedNCT02293382
• Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance ImagingCompletedNCT02090179
• Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene TherapyCompletedNCT02053064Phase 1/2
• Natural History Studies of Mucopolysaccharidosis IIICompletedNCT02037880
• Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool ChildrenCompletedNCT01938014
• Neurobehavioral Phenotypes in MPS IIICompletedNCT01873911
• Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)CompletedNCT01509768
• Intracerebral Gene Therapy for Sanfilippo Type A SyndromeCompletedNCT01474343Phase 1/2
• Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersTerminatedNCT01372228Phase 1/2
• ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesTerminatedNCT00654433Phase 3

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