| March 11, 2008 |
| November 19, 2009 |
| December 2007 |
| December 2012 (final data collection date for primary outcome measure) |
| To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration [ Time Frame: 1 year ] [ Designated as safety issue: No ] |
| Same as current |
| Complete list of historical versions of study NCT00638547 on ClinicalTrials.gov Archive Site |
- To determine the safety and toxicity of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
- To determine brain changes with magnetic resonance imaging [ Time Frame: 1 and 2 years ] [ Designated as safety issue: No ]
- To determine neurocognitive changes present in patients with Hurler syndrome [ Time Frame: 6, 12, and 24 months ] [ Designated as safety issue: No ]
- To evaluate memory/encoding [ Time Frame: 12 and 24 months after hematopoietic stem cell transplantation ] [ Designated as safety issue: No ]
- To determine cerebral spinal fluid levels of glycosaminoglycans, cytokines and antibodies to Laronidase at baseline and at each point CSF is obtained [ Time Frame: through 1 year ] [ Designated as safety issue: Yes ]
|
| Same as current |
| |
| Intrathecal Enzyme Replacement for Hurler Syndrome |
| Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome) |
This protocol will examine whether the enzyme -L-iduronidase, delivered into the spinal fluid of patients with Hurler syndrome at intervals before and after bone marrow transplant, is a safe and effective approach to slow the neurologic degeneration seen in Hurler patients undergoing transplantation. |
Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant (performed on another study protocol). This procedure is done by lumbar puncture (also called a "spinal tap"). |
| Phase I |
| Interventional |
| Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
| MPS IH (Hurler Syndrome) |
| Drug: IRT Laronidase |
| Experimental: All patients who have received at least one dose of Laronidase. |
| |
| |
| Recruiting |
| 25 |
| December 2014 |
| December 2012 (final data collection date for primary outcome measure) |
Inclusion Criteria:
- Patients with a diagnosis of MPS IH (Hurler syndrome) are candidates for this protocol if they are being considered for hematopoietic stem cell transplantation according the University of Minnesota guidelines.
Exclusion Criteria:
- Patients are less than 8 months old, or older than 3 years of age.
- There is a history of clinically-severe hypersensitivity to Laronidase.
- There is a contraindication for repeated lumbar puncture.
- The family is not willing to undergo the necessary procedures and evaluations inherent in the study.
- Consent has not been signed for participation in the 2004-09 study of intravenous Laronidase administration.
|
| Both |
| 6 Months to 3 Years |
| No |
|
|
| United States |
| |
| NCT00638547 |
| Paul Orchard, M.D., Masonic Cancer Center, University of Minnesota |
| 0707M11762, MT2007-10 |
| Masonic Cancer Center, University of Minnesota |
|
| Principal Investigator: |
Paul Orchard, MD |
University of Minnesota Medical Center |
|
|
| Masonic Cancer Center, University of Minnesota |
| November 2009 |
