Hurler syndrome
Information
- Disease name
- Hurler 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 |
| NCT00176891 | Completed | Phase 2 | Stem Cell Transplant w/Laronidase for Hurler | March 2004 | November 2012 |
| NCT00258011 | Completed | Phase 3 | Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease | December 2005 | October 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 |
| NCT00638547 | Completed | Phase 1 | Intrathecal Enzyme Replacement for Hurler Syndrome | January 2, 2008 | November 18, 2018 |
| NCT01043640 | Completed | Phase 2 | Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders | December 2009 | June 2017 |
| NCT01173016 | Completed | Phase 1 | Administration of IV Laronidase Post Bone Marrow Transplant in Hurler | May 29, 2012 | March 4, 2016 |
| NCT01873911 | Completed | Neurobehavioral Phenotypes in MPS III | December 2010 | August 2014 | |
| NCT03513328 | Completed | Phase 1/Phase 2 | Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation | June 15, 2018 | February 19, 2023 |
| NCT01917708 | Completed | Phase 1 | Bone Marrow Transplant With Abatacept for Non-Malignant Diseases | January 2014 | September 19, 2019 |
| NCT03639844 | No longer available | BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study | |||
| NCT02171104 | Recruiting | Phase 2 | MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | July 10, 2014 | July 14, 2028 |
| NCT01572636 | Terminated | Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome | March 28, 2012 | May 1, 2018 | |
| NCT00286689 | Withdrawn | N/A | Effects of Growth Hormone in Chronically Ill Children | February 3, 2006 | February 3, 2006 |
- OrphaNumber from OrphaNet (Orphanet)
- 93473