Hurler-Scheie Syndrome
Information
- Disease name
- Hurler-Scheie Syndrome
- Disease ID
- Description
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
| Genes | Mutation | Description | Source | Links |
|---|
| NCT ID | Status | Phase | Summary | Start date | Completion date |
|---|---|---|---|---|---|
| NCT03580083 | Active, not recruiting | Phase 1/Phase 2 | RGX-111 Gene Therapy in Patients With MPS I | April 3, 2019 | October 2024 |
| NCT00144781 | Completed | Phase 4 | A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease | December 2004 | January 2006 |
| NCT00146757 | Completed | Phase 2 | A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old | October 2002 | May 2005 |
| NCT00146770 | Completed | Phase 3 | Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients | May 2001 | March 2005 |
| NCT00258011 | Completed | Phase 3 | Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease | December 2005 | October 2006 |
| NCT00144768 | Completed | Phase 4 | A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients | July 2004 | May 2007 |
| NCT00852358 | Completed | N/A | A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I | June 2009 | April 2015 |
| NCT00912925 | Completed | Phase 3 | Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I | December 2000 | September 2001 |
| NCT00418821 | Terminated | Phase 4 | A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants | October 22, 2010 | December 21, 2022 |
| NCT00786968 | Terminated | Phase 1 | Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I | January 2008 | October 2011 |
| NCT01372228 | Terminated | Phase 1/Phase 2 | Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders | April 2011 | April 2016 |
- OrphaNumber from OrphaNet (Orphanet)
- 93476