Not applicableStudy completedNCT05155488What this trial is testingImprove the Awareness of Mucopolysaccharidosis Type II in BrazilWho this might be right forMucopolysaccharidosis (MPS) Takeda 250
Not applicableStudy completedNCT06022380What this trial is testingCore Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis IIWho this might be right forMucopolysaccharidosis Type II Manchester University NHS Foundation Trust 46
Early research (Phase 1)Study completedNCT06475404What this trial is testingThe Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy VolunteersWho this might be right forMucopolysaccharidosis Type IIMetabolic Diseases AO GENERIUM 18
Early research (Phase 1)Looking for participantsNCT04532047What this trial is testingPEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)Who this might be right forMPS IMPS IIMPS IVA+6 more University of California, San Francisco 10
Large-scale testing (Phase 3)Ended earlyNCT00748969What this trial is testingClinical Trial of Growth Hormone in MPS I, II, and VIWho this might be right forMucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis VI Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 2
Not applicableStudy completedNCT04007536What this trial is testingPotential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter SyndromeWho this might be right forMucopolysaccharidosis II Denali Therapeutics Inc. 18
Not applicableActive Not RecruitingNCT04597385What this trial is testingLong-term Follow-Up for RGX-121Who this might be right forMucopolysaccharidosis II REGENXBIO Inc. 27
Large-scale testing (Phase 3)Active Not RecruitingNCT07236606What this trial is testingRGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)Who this might be right forMPS IIHunter Syndrome (MPS II) REGENXBIO Inc. 2
Not applicableStudy completedNCT01822184What this trial is testingObservational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)Who this might be right forMucopolysaccharidosis (MPS)Hunter Syndrome Shire 100
Testing effectiveness (Phase 2)Study completedNCT01506141What this trial is testingAn 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 ImpairmentWho this might be right forHunter Syndrome Takeda 15
Post-approval studies (Phase 4)Study completedNCT00607386What this trial is testingSafety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase TherapyWho this might be right forHunter SyndromeMucopolysaccharidosis IIMPS II Shire 28
Not applicableStudy completedNCT01449240What this trial is testingCollection and Study of Cerebrospinal Fluid in Patients With Hunter SyndromeWho this might be right forHunter Syndrome Shire 10
Not applicableStudy completedNCT01602601What this trial is testingTest the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723Who this might be right forMucopolysaccharidosis II GlaxoSmithKline 10
Large-scale testing (Phase 3)Ended earlyNCT00654433What this trial is testingALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesWho this might be right forInherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more Aldagen 40
Testing effectiveness (Phase 2)Ended earlyNCT03041324What this trial is testingAscending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS IIWho this might be right forMucopolysaccharidosis IIMPS II Sangamo Therapeutics 9
Testing effectiveness (Phase 2)Study completedNCT00920647What this trial is testingA 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®Who this might be right forHunter Syndrome Shire 16
Post-approval studies (Phase 4)WithdrawnNCT05494593What this trial is testingELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)Who this might be right forMucopolysaccharidosis (MPS)Hunter Syndrome Takeda
Not applicableEnded earlyNCT01675674What this trial is testingStudy to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia ClinicsWho this might be right forMucopolysaccharidosesMucopolysaccharidosis IMucopolysaccharidosis II+2 more National MPS Society 3,000
Testing effectiveness (Phase 2)UnknownNCT02663024What this trial is testingStudy of Idursulfase-beta (GC1111) in Hunter SyndromeWho this might be right forMucopolysaccharidosis II Green Cross Corporation 20
Not applicableStudy completedNCT01938014What this trial is testingLysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool ChildrenWho this might be right forMucopolysaccharidosis Type I (MPS I)Mucopolysaccharidosis Type II (MPS II)Mucopolysaccharidosis Type III (MPS III)+2 more University of Chicago 19