NCT01572636

Brief Summary

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.

Trial Health

57
Monitor

Trial Health Score

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

Enrollment
20

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Mar 2012

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
terminated

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

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 28, 2012

Completed
7 days until next milestone

First Submitted

Initial submission to the registry

April 4, 2012

Completed
2 days until next milestone

First Posted

Study publicly available on registry

April 6, 2012

Completed
6.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2018

Completed
Last Updated

September 27, 2018

Status Verified

September 1, 2018

Enrollment Period

6.1 years

First QC Date

April 4, 2012

Last Update Submit

September 25, 2018

Conditions

Mucopolysaccharidosis Type IHMPS IHurler Syndrome

Keywords

Hurler Syndrome

Outcome Measures

Primary Outcomes (1)

  • Overall Survival

    Patients alive at 1 year post transplantation.

    At 1 Year

Secondary Outcomes (3)

  • Incidence of Engraftment

    1 Year Post Transplant

  • Incidence of Grade III-IV Acute Graft Versus Host Disease

    Day 100

  • Proportion of patients in need of ventilator support

    1 Year

Study Arms (1)

Laronidase use in Hurler Syndrome

Laronidase receiving prior and post transplant

Drug: Laronidase

Interventions

LaronidaseDRUG

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.

Also known as: Aldurazyme
Laronidase use in Hurler Syndrome

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability 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

You may qualify if:

  • 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

You may not qualify if:

  • No prior therapy with laronidase enzyme replacement therapy (ERT)

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 I

Interventions

Iduronidase

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, 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)

Glycoside HydrolasesHydrolasesEnzymesEnzymes and Coenzymes

Study Officials

  • Paul Orchard, M.D.

    Masonic Cancer Center, University of Minnesota

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 4, 2012

First Posted

April 6, 2012

Study Start

March 28, 2012

Primary Completion

May 1, 2018

Study Completion

May 1, 2018

Last Updated

September 27, 2018

Record last verified: 2018-09

Locations

US(1)