Mucopolysaccharidosis II: all 73 studies

Page 1 of 1. Open to participants first (8), then studies with results posted, then the rest; newest registration first within each.

• An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007Enrolling by invitationNCT06075537Phase 2/3
• MPS (RaDiCo Cohort) (RaDiCo-MPS)RecruitingNCT06036693
• Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter SyndromeAvailableNCT05795361
• Registry of Patients Diagnosed With Lysosomal Storage DiseasesRecruitingNCT05619900
• An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) SubjectsEnrolling by invitationNCT05594992Phase 3
• ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramEnrolling by invitationNCT05368038
• UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like CellsRecruitingNCT02254863Phase 1
• 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
• Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS IITerminatedNCT03041324Phase 1/2Results posted
• Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of AgeCompletedNCT02455622Phase 4Results posted
• Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VICompletedNCT02437253Phase 1/2Results posted
• Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094CompletedNCT02412787Phase 2/3Results posted
• Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive ImpairmentCompletedNCT02055118Phase 2/3Results posted
• An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive ImpairmentCompletedNCT01506141Phase 1/2Results posted
• Collection and Study of Cerebrospinal Fluid in Patients With Hunter SyndromeCompletedNCT01449240Results posted
• Allogeneic Bone Marrow Transplant for Inherited Metabolic DisordersCompletedNCT01043640Phase 2Results posted
• Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®CompletedNCT00937794Results posted
• A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®CompletedNCT00920647Phase 1/2Results posted
• An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome PatientsCompletedNCT00882921Results posted
• Clinical Trial of Growth Hormone in MPS I, II, and VITerminatedNCT00748969Phase 2/3Results 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
• Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)CompletedNCT00069641Phase 2/3Results posted
• An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta)CompletedNCT07344376Phase 3
• RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)Active, not recruitingNCT07236606Phase 3
• A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy VolunteersCompletedNCT06475404Phase 1
• Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive ImpairmentActive, not recruitingNCT06031259Phase 2/3
• Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis IICompletedNCT06022380
• Baby Detect : Genomic Newborn ScreeningCompletedNCT05687474
• Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIActive, not recruitingNCT05665166Phase 1/2
• 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
• A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)Active, not recruitingNCT05371613Phase 2/3
• Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS IIWithdrawnNCT05238324Phase 1
• A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type IIActive, not recruitingNCT05208281Phase 2/3
• A Study to Improve the Awareness of Mucopolysaccharidosis Type II in BrazilCompletedNCT05155488
• MPS II ImmunophenotypingTerminatedNCT04976231
• Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIXTerminatedNCT04628871
• Long-term Follow-Up for RGX-121Active, not recruitingNCT04597385
• Mucopolysaccharidosis Type II ObservationalWithdrawnNCT04591834
• A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)Active, not recruitingNCT04573023Phase 3
• 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
• An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type IIActive, not recruitingNCT04348136Phase 2/3
• A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter SyndromeActive, not recruitingNCT04251026Phase 1/2
• A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter SyndromeCompletedNCT04007536
• A Study of GC1111 in Hunter Syndrom PatientsCompletedNCT03920540Phase 3
• An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis IIActive, not recruitingNCT03708965Phase 2
• Early Check: Expanded Screening in NewbornsActive, not recruitingNCT03655223
• Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)CompletedNCT03582449
• 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
• A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21)CompletedNCT03359213Phase 2
• Hunter Outcome Survey (HOS)CompletedNCT03292887
• Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-IIICompletedNCT03161171
• Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VIActive, not recruitingNCT03153319Phase 1/2
• A Study of JR-141 in Patients With Mucopolysaccharidosis Type IICompletedNCT03128593Phase 1/2
• Study of Idursulfase-beta (GC1111) in Hunter SyndromeStatus unknownNCT02663024Phase 2
• Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter SyndromeCompletedNCT02262338Phase 1
• MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisActive, not recruitingNCT02171104Phase 2
• The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) PatientsStatus unknownNCT02044692
• Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool ChildrenCompletedNCT01938014
• Longitudinal Studies of Brain Structure and Function in MPS DisordersCompletedNCT01870375
• Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)CompletedNCT01822184
• Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia ClinicsTerminatedNCT01675674
• Safety and Efficacy of HunteraseCompletedNCT01645189Phase 3
• A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723CompletedNCT01602601
• Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersTerminatedNCT01372228Phase 1/2
• Biomarkers for Hunter SyndromeTerminatedNCT01330277
• To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome PatientsCompletedNCT01301898Phase 1/2
• Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)CompletedNCT00004454Phase 1/2

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