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Stem Cell Transplant w/Laronidase for Hurler

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00176891
Recruitment Status : Completed
First Posted : September 15, 2005
Results First Posted : July 11, 2019
Last Update Posted : July 11, 2019
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Tracking Information
First Submitted Date  ICMJE September 12, 2005
First Posted Date  ICMJE September 15, 2005
Results First Submitted Date  ICMJE April 6, 2017
Results First Posted Date  ICMJE July 11, 2019
Last Update Posted Date July 11, 2019
Study Start Date  ICMJE March 2004
Actual Primary Completion Date November 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 10, 2019)
  • Number of Patients Alive at One Year Post Transplant [ Time Frame: one year ]
  • Number of Patients Requiring Ventilator Support at One Year Post Transplant [ Time Frame: one year ]
Original Primary Outcome Measures  ICMJE
 (submitted: September 12, 2005)
Survival at one year and the proportion of patients in need of ventilator support by one year
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 10, 2019)
  • Donor Engraftment [ Time Frame: Day 100 post transplant ]
  • Patients With Grade III-IV Acute GVHD [ Time Frame: Day 100 post transplant ]
  • Reduction in Glycosaminoglycans (GAG) [ Time Frame: Prior to, During and After ERT ]
    Data was not collected on this outcome measure and is not available for reporting.
  • Toxicity (Adverse Events) Associated With Infusions of Laronidase [ Time Frame: 1 year post transplant ]
    Data was not collected on this outcome measure and is not available for reporting.
  • Development of Anti-iduronidase Antibodies in Serum [ Time Frame: 1 Year ]
  • Patients With Improvement in Obstructive Apnea (Breathing) by Polysomnography [ Time Frame: Baseline, 12 weeks after laronidase, after transplant ]
Original Secondary Outcome Measures  ICMJE
 (submitted: September 12, 2005)
  • Donor engraftment over one year post transplant
  • Grade III-IV acute GvHD
  • Disease specific measurements:
  • (reduction in GAG, improvement in polysomnography)
  • Toxicity associated with infusions of enzyme
  • Development of Anti-iduronidase Antibodies in Serum
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Stem Cell Transplant w/Laronidase for Hurler
Official Title  ICMJE Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)
Brief Summary The investigators hypothesize that weekly infusions of Laronidase ERT for 10-12 weeks prior to transplant and 8 weeks following transplant will result in a reduction of glycosaminoglycans (GAG) burden that is associated with decreased complications following transplant.
Detailed Description

Subjects will receive laronidase once a week intravenously for 10-12 weeks prior to transplant and for approximately 8 weeks after transplant. Laronidase will be given by intravenous infusion (IV) through a catheter and from there to your child's body's cells and organs to break down the glycosaminoglycans (GAG) buildup.

Prior to starting ERT, subjects will have a complete physical examination, which includes a complete assessment of your child's airway and lungs. In addition to standard treatment evaluations and tests, which are done prior to hematopoietic stem cell transplant (HSCT), subjects will have the following tests: an additional teaspoon of blood for a baseline test for serum antibodies against laronidase, before and after the fourth dose of laronidase, the investigators will collect 2 teaspoons of blood for an alpha-L-iduronidase enzyme level; to watch for side effects to laronidase and the development of antibodies to laronidase, approximately 2 teaspoons of blood will be collected every 3 weeks while the subject is receiving laronidase ERT.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Mucopolysaccharidosis I
  • Hurler Syndrome
Intervention  ICMJE
  • Procedure: Stem Cell Transplant
    enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following
    Other Name: ERT
  • Drug: Laronidase ERT
    Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.
    Other Name: Aldurazyme®
Study Arms  ICMJE Experimental: Laronidase ERT Treatment
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.
Interventions:
  • Procedure: Stem Cell Transplant
  • Drug: Laronidase ERT
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 12, 2005)
25
Original Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE November 2012
Actual Primary Completion Date November 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 7 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00176891
Other Study ID Numbers  ICMJE MT2004-09
0403M57728 ( Other Identifier: IRB, University of Minnesota )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Masonic Cancer Center, University of Minnesota
Study Sponsor  ICMJE Masonic Cancer Center, University of Minnesota
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Paul Orchard, MD Masonic Cancer Center, University of Minnesota
PRS Account Masonic Cancer Center, University of Minnesota
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP