Mucopolysaccharidosis I: all 74 studies
Page 1 of 1. Open to participants first (7), then studies with results posted, then the rest; newest registration first within each.
- A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type IRecruitingNCT06519552Phase 1
- Long Term Follow-Up for RGX-111Enrolling by invitationNCT06103487
- MPS (RaDiCo Cohort) (RaDiCo-MPS)RecruitingNCT06036693
- ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS IRecruitingNCT05682144Phase 1
- Registry of Patients Diagnosed With Lysosomal Storage DiseasesRecruitingNCT05619900
- Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICRecruitingNCT04528355
- Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTRecruitingNCT01962415Phase 2
- A Study of JR-171 in Patients With Mucopolysaccharidosis ICompletedNCT04227600Phase 1/2Results posted
- Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationCompletedNCT03513328Phase 1/2Results posted
- MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)CompletedNCT03406962Phase 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
- Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VICompletedNCT02437253Phase 1/2Results posted
- Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS ITerminatedNCT02232477Not applicableResults posted
- Administration of IV Laronidase Post Bone Marrow Transplant in HurlerCompletedNCT01173016Phase 1Results posted
- Allogeneic Bone Marrow Transplant for Inherited Metabolic DisordersCompletedNCT01043640Phase 2Results posted
- Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) ICompletedNCT00912925Phase 3Results posted
- Clinical Trial of Growth Hormone in MPS I, II, and VITerminatedNCT00748969Phase 2/3Results posted
- Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant DisordersCompletedNCT00744692Phase 1Results posted
- Immune Tolerance Study With Aldurazyme® (Laronidase)CompletedNCT00741338Phase 1/2Results posted
- Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of MetabolismTerminatedNCT00668564Phase 2Results 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
- 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
- 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
- Baby Detect : Genomic Newborn ScreeningCompletedNCT05687474
- Safety and Efficacy of Encapsulated Allogeneic MPS-1 TherapyWithdrawnNCT05665036Phase 1/2
- Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.Active, not recruitingNCT05634512
- China Post-marketing Surveillance (PMS) Study of Aldurazyme®CompletedNCT05134571Phase 4
- 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
- Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIXTerminatedNCT04628871
- An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)CompletedNCT04453085Phase 1/2
- A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456TerminatedNCT04008849
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT03655223
- BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 StudyNo longer availableNCT03639844
- RGX-111 Gene Therapy in Patients With MPS ISuspendedNCT03580083Phase 1/2
- MRS to Determine Neuroinflammation and Oxidative Stress in MPS ICompletedNCT03576729
- Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler VariantActive, not recruitingNCT03488394Phase 1/2
- S 81694 Plus Paclitaxel in Metastatic Breast CancerCompletedNCT03411161Phase 1/2
- 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
- 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
- Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)WithdrawnNCT02715505Phase 1/2
- Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181CompletedNCT02597114Phase 1
- Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS ICompletedNCT02371226Phase 1
- Biomarker for Hurler Disease (BioHurler)WithdrawnNCT02298712
- Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)WithdrawnNCT02298647
- MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisActive, not recruitingNCT02171104Phase 2
- Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)TerminatedNCT02095015
- Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool ChildrenCompletedNCT01938014
- Bone Marrow Transplant With Abatacept for Non-Malignant DiseasesCompletedNCT01917708Phase 1
- Neurobehavioral Phenotypes in MPS IIICompletedNCT01873911
- Longitudinal Studies of Brain Structure and Function in MPS DisordersCompletedNCT01870375
- Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia ClinicsTerminatedNCT01675674
- Human Placental-Derived Stem Cell TransplantationCompletedNCT01586455Phase 1
- Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler SyndromeTerminatedNCT01572636
- Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersTerminatedNCT01372228Phase 1/2
- A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS ICompletedNCT00852358Not applicable
- Extension Study of Intrathecal Enzyme Replacement Therapy for MPS ITerminatedNCT00786968Phase 1
- Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood CellsCompletedNCT00730314Phase 1/2
- Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked ProductsCompletedNCT00695279
- Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood CellsCompletedNCT00692926Phase 1
- ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesTerminatedNCT00654433Phase 3
- Intrathecal Enzyme Replacement for Hurler SyndromeCompletedNCT00638547Phase 1
- Effects of Growth Hormone in Chronically Ill ChildrenWithdrawnNCT00286689Not applicable
- Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) ITerminatedNCT00215527Phase 1
- Mucopolysaccharidosis I (MPS I) RegistryCompletedNCT00144794
- A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated PatientsCompletedNCT00144768Phase 4
- Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell TransplantationStatus unknownNCT00005900