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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: |
NCT00176891 |
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Date of registration:
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12/09/2005 |
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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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Stem Cell Transplant w/Laronidase for Hurler
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Scientific title:
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Phase II Study of Combined Laronidase (AldurazymeTM) 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 2004 |
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Target sample size:
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25 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00176891 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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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, MD |
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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:
- Patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome)
who are candidates for first hematopoietic stem cell transplant (HSCT) according to a
University of Minnesota myeloablative HSCT protocol.
Exclusion Criteria:
- Not being considered for University of Minnesota myeloablative HSCT protocol.
- Previous administration of laronidase enzyme
- Second or subsequent HSCT.
Age minimum:
N/A
Age maximum:
7 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Hurler Syndrome
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Mucopolysaccharidosis I
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Intervention(s)
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Procedure: Stem Cell Transplant
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Drug: Laronidase ERT
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Primary Outcome(s)
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Number of Patients Alive at One Year Post Transplant
[Time Frame: one year]
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Number of Patients Requiring Ventilator Support at One Year Post Transplant
[Time Frame: one year]
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Secondary Outcome(s)
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Patients With Grade III-IV Acute GVHD
[Time Frame: Day 100 post transplant]
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Donor Engraftment
[Time Frame: Day 100 post transplant]
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Patients With Improvement in Obstructive Apnea (Breathing) by Polysomnography
[Time Frame: Baseline, 12 weeks after laronidase, after transplant]
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Development of Anti-iduronidase Antibodies in Serum
[Time Frame: 1 Year]
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Reduction in Glycosaminoglycans (GAG)
[Time Frame: Prior to, During and After ERT]
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Toxicity (Adverse Events) Associated With Infusions of Laronidase
[Time Frame: 1 year post transplant]
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Secondary ID(s)
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MT2004-09
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0403M57728
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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