NCT00176891

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Mar 2004

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2004

Completed
1.5 years until next milestone

First Submitted

Initial submission to the registry

September 12, 2005

Completed
3 days until next milestone

First Posted

Study publicly available on registry

September 15, 2005

Completed
7.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2012

Completed
6.7 years until next milestone

Results Posted

Study results publicly available

July 11, 2019

Completed
Last Updated

July 11, 2019

Status Verified

July 1, 2019

Enrollment Period

8.7 years

First QC Date

September 12, 2005

Results QC Date

April 6, 2017

Last Update Submit

July 10, 2019

Conditions

Mucopolysaccharidosis IHurler Syndrome

Keywords

Laronidase ERTStem cell transplantstorage diseaseinborn errors of metabolismhurler syndromeglycosaminoglycansenzyme replacement

Outcome Measures

Primary Outcomes (2)

  • Number of Patients Alive at One Year Post Transplant

    one year

  • Number of Patients Requiring Ventilator Support at One Year Post Transplant

    one year

Secondary Outcomes (6)

  • Donor Engraftment

    Day 100 post transplant

  • Patients With Grade III-IV Acute GVHD

    Day 100 post transplant

  • Reduction in Glycosaminoglycans (GAG)

    Prior to, During and After ERT

  • Toxicity (Adverse Events) Associated With Infusions of Laronidase

    1 year post transplant

  • Development of Anti-iduronidase Antibodies in Serum

    1 Year

  • +1 more secondary outcomes

Study Arms (1)

Laronidase ERT Treatment

EXPERIMENTAL

Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.

Procedure: Stem Cell TransplantDrug: Laronidase ERT

Interventions

Stem Cell TransplantPROCEDURE

enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following

Also known as: ERT
Laronidase ERT Treatment
Laronidase ERTDRUG

Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.

Also known as: Aldurazyme®
Laronidase ERT Treatment

Eligibility Criteria

AgeUp to 7 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify if:

  • Not being considered for University of Minnesota myeloablative HSCT protocol.
  • Previous administration of laronidase enzyme
  • Second or subsequent HSCT.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Mucopolysaccharidosis IMetabolism, Inborn Errors

Interventions

Stem Cell TransplantationIduronidase

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Cell TransplantationCell- and Tissue-Based TherapyBiological TherapyTherapeuticsTransplantationSurgical Procedures, OperativeGlycoside HydrolasesHydrolasesEnzymesEnzymes and Coenzymes

Limitations and Caveats

All of the patients on this trial also underwent allogeneic blood/marrow transplantation. All the serious and non-serious adverse events listed were related to the process of transplantation and not to the study medication, Laronidase.

Results Point of Contact

Title
Dr. Paul Orchard
Organization
Masonic Cancer Center, University of Minnesota
orcha001@umn.edu
(612) 626-2313

Study Officials

  • Paul Orchard, MD

    Masonic Cancer Center, University of Minnesota

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 12, 2005

First Posted

September 15, 2005

Study Start

March 1, 2004

Primary Completion

November 1, 2012

Study Completion

November 1, 2012

Last Updated

July 11, 2019

Results First Posted

July 11, 2019

Record last verified: 2019-07

Locations

US(1)