Mucopolysaccharidoses: all 144 studies

Page 1 of 2. Open to participants first (11), then studies with results posted, then the rest; newest registration first within each.

• Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIBNot yet recruitingNCT07818759Phase 1/2
• Natural History to Assess Disease in Patients With MPS IIICNot yet recruitingNCT07712003
• Morphology in Oral Rare Syndromes & Artificial Intelligence for Clinical DiagnosisNot yet recruitingNCT07666269Not applicable
• Physical Activity Intervention for MPSRecruitingNCT07449143
• Cardiac Structure and Function in MPSRecruitingNCT07361536
• Pediatric Arthropathy Beyond Inflammation: Clinical Spectrum and Diagnostic Approach at Assiut University Children HospitalNot yet recruitingNCT07173010
• Nutritional Assessment in Patient of Mucopolysaccharide "Not yet recruitingNCT07136896Not applicable
• A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type IRecruitingNCT06519552Phase 1
• Registry of Patients Diagnosed With Lysosomal Storage DiseasesRecruitingNCT05619900
• The Myelin Disorders Biorepository ProjectRecruitingNCT03047369
• Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTRecruitingNCT01962415Phase 2
• A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in IndiaCompletedNCT05058391Phase 4Results posted
• A Study of JR-171 in Patients With Mucopolysaccharidosis ICompletedNCT04227600Phase 1/2Results posted
• Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA DiseaseTerminatedNCT04088734Phase 1/2Results posted
• Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS IITerminatedNCT03041324Phase 1/2Results posted
• Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS ITerminatedNCT02702115Phase 1/2Results posted
• LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for LeukodystrophiesCompletedNCT02699190Results 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
• Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VICompletedNCT02437253Phase 1/2Results posted
• A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)CompletedNCT02432144Phase 3Results posted
• Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of AgeCompletedNCT02418455Phase 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
• Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS ITerminatedNCT02232477Not applicableResults posted
• A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)CompletedNCT02230566Phase 3Results posted
• An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)CompletedNCT01856218Phase 1/2Results posted
• Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited AmbulationTerminatedNCT01697319Phase 2Results posted
• Safety and Exercise Study of Two Doses of BMN 110 for Morquio A SyndromeTerminatedNCT01609062Phase 2Results posted
• Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease NetworkCompletedNCT01586871Results posted
• Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)CompletedNCT01515956Phase 2Results posted
• Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)CompletedNCT01415427Phase 3Results posted
• A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)CompletedNCT01275066Phase 3Results posted
• A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)TerminatedNCT01242111Phase 1/2Results posted
• Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA PatientsCompletedNCT01155778Phase 1/2Results posted
• Allogeneic Bone Marrow Transplant for Inherited Metabolic DisordersCompletedNCT01043640Phase 2Results posted
• Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) ICompletedNCT00912925Phase 3Results posted
• A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVACompletedNCT00884949Phase 1/2Results posted
• Clinical Trial of Growth Hormone in MPS I, II, and VITerminatedNCT00748969Phase 2/3Results posted
• Immune Tolerance Study With Aldurazyme® (Laronidase)CompletedNCT00741338Phase 1/2Results posted
• Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving IdursulfaseCompletedNCT00630747Phase 2/3Results posted
• Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase TherapyCompletedNCT00607386Phase 4Results posted
• A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed InfantsTerminatedNCT00418821Phase 4Results posted
• A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VICompletedNCT00299000Phase 4Results posted
• Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) DiseaseCompletedNCT00258011Phase 3Results posted
• Stem Cell Transplantation for HurlerCompletedNCT00176917Phase 2Results posted
• Stem Cell Transplant w/Laronidase for HurlerCompletedNCT00176891Phase 2Results posted
• Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) PatientsCompletedNCT00146770Phase 3Results posted
• A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years OldCompletedNCT00146757Phase 2Results posted
• A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) DiseaseCompletedNCT00144781Phase 4Results posted
• Study of rhASB in Patients With Mucopolysaccharidosis VICompletedNCT00104234Phase 3Results posted
• Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)CompletedNCT00069641Phase 2/3Results posted
• Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VICompletedNCT00067470Phase 3Results posted
• A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy VolunteersCompletedNCT06475404Phase 1
• Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I PatientsCompletedNCT06406153Phase 3
• A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCTActive, not recruitingNCT06149403Phase 3
• Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis IICompletedNCT06022380
• 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
• A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)WithdrawnNCT05494593Phase 4
• A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS ⅡActive, not recruitingNCT05422482Phase 1
• Validation and Reliability of Iris Cameras in MucopolysaccharidosesStatus unknownNCT05354219
• Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS IIWithdrawnNCT05238324Phase 1
• A Study to Improve the Awareness of Mucopolysaccharidosis Type II in BrazilCompletedNCT05155488
• A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care SettingCompletedNCT05073783
• Cardiovascular Structure and Function in the MucopolysaccharidosesActive, not recruitingNCT05063435
• The Effect of Enzyme Replacement Therapy in MucopolysaccharidosisCompletedNCT05006222Not applicable
• MPS II ImmunophenotypingTerminatedNCT04976231
• Ophthalmic Manifestations of Patients With MucopolysaccharidosisStatus unknownNCT04770415
• A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101TerminatedNCT04655911
• Evaluation of Cardiac Affections in Patients With Mucopolysaccharidosis (MPS) in Assuit University Children Hospital (AUCH)Status unknownNCT04637646
• Mucopolysaccharidosis Type II ObservationalWithdrawnNCT04591834
• RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)CompletedNCT04571970Phase 1/2
• A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy VolunteersCompletedNCT04539340Phase 1
• Assessment of Factors That Affected Respiratory Parameters in Mucopolysaccharidoses PatientsCompletedNCT04491747
• Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIAActive, not recruitingNCT04201405Phase 1/2
• Respiratory Cathepsins, Proteases Inhibitors and Glycosaminoglycans (GAG) in MucopolysaccharidosisCompletedNCT04112602
• Open-label Study of Anakinra in MPS IIICompletedNCT04018755Phase 1/2
• A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter SyndromeCompletedNCT04007536
• A Study of GC1111 in Hunter Syndrom PatientsCompletedNCT03920540Phase 3
• Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VIStatus unknownNCT03632213Phase 2
• Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)Status unknownNCT03612869Phase 2/3
• MRS to Determine Neuroinflammation and Oxidative Stress in MPS ICompletedNCT03576729
• A Study of JR-141 in Patients With Mucopolysaccharidosis IICompletedNCT03568175Phase 2/3
• CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)Active, not recruitingNCT03566043Phase 2/3
• Mucopolysaccharidosis Type II Natural HistoryCompletedNCT03529786
• Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIBTerminatedNCT03315182Phase 1/2
• A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)Active, not recruitingNCT03227042
• Gene Therapy in Patients With Mucopolysaccharidosis DiseaseCompletedNCT03173521Phase 1/2
• Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-IIICompletedNCT03161171
• Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?CompletedNCT03150069
• A Study of JR-141 in Patients With Mucopolysaccharidosis Type IICompletedNCT03128593Phase 1/2
• Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS ICompletedNCT03071341Phase 1/2
• Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS ICompletedNCT03053089Phase 1/2
• A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative DiagnosisStatus unknownNCT03017677
• A Treatment Study of Mucopolysaccharidosis Type IIIBCompletedNCT02754076Phase 1/2
• Natural History Study of Patients With MPS IIIACompletedNCT02746341
• Study of Idursulfase-beta (GC1111) in Hunter SyndromeStatus unknownNCT02663024Phase 2
• Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181CompletedNCT02597114Phase 1
• New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in MucopolysacchardiosisStatus unknownNCT02583152
• A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)CompletedNCT02493998

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