638547
Last Update Posted: 2018-12-03
Recruiting has ended
All Genders accepted | 6 months-3 Years |
26 Estimated Participants | No Expanded Access |
Interventional Study | Does not accept healthy volunteers |
Intrathecal Enzyme Replacement for Hurler Syndrome
This protocol will examine whether the enzyme alpha-L-iduronidase (Laronidase), 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. This procedure is done by lumbar puncture (also called a "spinal tap").
Eligibility
Relevant conditions:
Hurler Syndrome
If you aren't sure if you meet the criteria above speak to your healthcare professional. Criteria may be updated but not reflected here, do not hesitate to contact the trial if you think are close to fitting criteria.
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Contact Information
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Data sourced from ClinicalTrials.gov