- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01572636
Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
September 25, 2018 updated by: Masonic Cancer Center, University of Minnesota
MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).
This is a standard of care treatment guideline for patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) who are being considered as candidates for first hematopoietic stem cell transplantation (HSCT) according to a University of Minnesota myeloablative HSCT protocol.
Study Overview
Status
Terminated
Conditions
Intervention / Treatment
Detailed Description
Laronidase Enzyme Replacement Therapy will be performed using laronidase once a week for 12 weeks prior to hematopoietic stem cell transplantation and for 8 weeks post-transplant to reduce pulmonary complications.
Study Type
Observational
Enrollment (Actual)
20
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
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Minnesota
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Minneapolis, Minnesota, United States, 55455
- Masonic Cancer Center, University of Minnesota
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Probability Sample
Study Population
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
Description
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)
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
Laronidase use in Hurler Syndrome
Laronidase receiving prior and post transplant
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Administered 0.58 mg/kg/dose intravenously (IV) once a week beginning 12 weeks before planned hematopoietic stem cell transplant (HSCT) and resume same dosing regimen for 8 weeks after HSCT.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Overall Survival
Time Frame: At 1 Year
|
Patients alive at 1 year post transplantation.
|
At 1 Year
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Incidence of Engraftment
Time Frame: 1 Year Post Transplant
|
The incidence of donor engraftment will be estimated by taking the simple proportion of patients achieving donor engraftment over the number of evaluable patients.
Donor engraftment will be defined as achieving an absolute neutrophil count ≥ 5x10^8/kg for three consecutive days before day 42 and maintenance of >10% donor chimerism through one year post transplant or death.
|
1 Year Post Transplant
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Incidence of Grade III-IV Acute Graft Versus Host Disease
Time Frame: Day 100
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Cumulative incidence will be used to estimate grade III-IV acute GvHD, treating death as a competing risk.
|
Day 100
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Proportion of patients in need of ventilator support
Time Frame: 1 Year
|
Count of patients using ventilator by 1 year.
|
1 Year
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Principal Investigator: Paul Orchard, M.D., Masonic Cancer Center, University of Minnesota
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
March 28, 2012
Primary Completion (Actual)
May 1, 2018
Study Completion (Actual)
May 1, 2018
Study Registration Dates
First Submitted
April 4, 2012
First Submitted That Met QC Criteria
April 4, 2012
First Posted (Estimate)
April 6, 2012
Study Record Updates
Last Update Posted (Actual)
September 27, 2018
Last Update Submitted That Met QC Criteria
September 25, 2018
Last Verified
September 1, 2018
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 2011OC140
- MT2011-21C (Other Identifier: Blood and Marrow Transplant Program)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Hurler Syndrome
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Masonic Cancer Center, University of MinnesotaWithdrawnMucopolysaccharidosis Type IH | Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome) | MPS IH, Hurler Syndrome
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Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedMucopolysaccharidosis I | Hurler's Syndrome | Hurler-Scheie Syndrome | Scheie's SyndromeUnited States
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Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedMucopolysaccharidosis I | Scheie Syndrome | Hurler Syndrome | Hurler-Scheie SyndromeNetherlands, France, Germany, United Kingdom
-
Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedMucopolysaccharidosis I | Hurler-Scheie Syndrome | Hurlers SyndromeUnited States, Canada, Germany
-
Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedMucopolysaccharidosis I | Scheie Syndrome | Hurler Syndrome | Hurler-Scheie SyndromeJapan
-
Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCTerminatedMucopolysaccharidosis I | Hurler's Syndrome | Hurler-Scheie Syndrome | ScheieItaly
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Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedMucopolysaccharidosis I | Scheie Syndrome | Hurler's Syndrome | Hurler-Scheie SyndromeUnited States, Canada, United Kingdom, Brazil, Germany, Italy, Netherlands
-
Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedMucopolysaccharidosis I | Scheie Syndrome | Hurler's Syndrome | Hurler-Scheie SyndromeBrazil, Canada
-
Orchard TherapeuticsRecruitingMPS-IH (Hurler Syndrome)Netherlands, United States, Italy, United Kingdom
-
REGENXBIO Inc.Active, not recruitingHurler Syndrome | Hurler-Scheie Syndrome | Mucopolysaccharidosis Type I (MPS I)United States, Brazil, Israel
Clinical Trials on Laronidase
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Masonic Cancer Center, University of MinnesotaCompleted
-
Masonic Cancer Center, University of MinnesotaCompleted
-
Masonic Cancer Center, University of MinnesotaRecruitingMucopolysaccharidosis Type I | Hematopoietic Cell TransplantationUnited States
-
Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCTerminatedMucopolysaccharidosis I | Hurler's Syndrome | Hurler-Scheie Syndrome | ScheieItaly
-
Patricia I. Dickson, M.D.The Ryan FoundationTerminatedMucopolysaccharidosis I | Scheie Syndrome | Lysosomal Storage Disease | Spinal Cord Compression | Hurler-Scheie SyndromeFinland, United States
-
Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis I | Hurler SyndromeUnited States
-
Genzyme, a Sanofi CompanyCompleted
-
Patricia I. Dickson, M.D.University of California, Los Angeles; FDA Office of Orphan Products Development and other collaboratorsTerminatedMucopolysaccharidosis I | Lysosomal Storage Diseases | Spinal Cord CompressionUnited States, Finland
-
Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedMucopolysaccharidosis I | Hurler's Syndrome | Hurler-Scheie Syndrome | Scheie's SyndromeUnited States
-
Patricia I. Dickson, M.D.National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University... and other collaboratorsCompletedMucopolysaccharidosis I | Scheie Syndrome | Lysosomal Storage Disease | Cognitive Decline | Hurler-Scheie SyndromeUnited States