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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01572636 |
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Date of registration:
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04/04/2012 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
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Scientific title:
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MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH). |
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Date of first enrolment:
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March 28, 2012 |
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Target sample size:
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20 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT01572636 |
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Study type:
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Observational |
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Study design:
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Phase:
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Countries of recruitment
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United States
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Contacts
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Name:
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Paul Orchard, M.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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Masonic Cancer Center, University of Minnesota |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) and being
considered as a candidate for first transplant according to a University of Minnesota
myeloablative hematopoietic stem cell transplant (HSCT) protocol
Exclusion Criteria:
- No prior therapy with laronidase enzyme replacement therapy (ERT)
Age minimum:
N/A
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Hurler Syndrome
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MPS I
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Mucopolysaccharidosis Type IH
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Intervention(s)
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Drug: Laronidase
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Primary Outcome(s)
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Overall Survival
[Time Frame: At 1 Year]
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Secondary Outcome(s)
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Incidence of Grade III-IV Acute Graft Versus Host Disease
[Time Frame: Day 100]
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Incidence of Engraftment
[Time Frame: 1 Year Post Transplant]
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Proportion of patients in need of ventilator support
[Time Frame: 1 Year]
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Secondary ID(s)
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MT2011-21C
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2011OC140
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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