Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

Terminated

• Conditions: Hurler Syndrome; MPS I; Mucopolysaccharidosis Type IH
• Interventions: Drug: Laronidase

• Registration Number: NCT01572636
• Lead Sponsor: Masonic Cancer Center, University of Minnesota

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.

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.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study Start: 2012-03-28
• Study First Submitted: 2012-04-04
• Study First Submitted QC: 2012-04-04
• Study First Posted: 2012-04-06
• Primary Completion: 2018-05-01
• Study Completion: 2018-05-01
• Status Verified: 2018-09
• Last Update Submitted: 2018-09-25
• Last Update Posted: 2018-09-27

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 20

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

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Exclusion Criteria

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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Laronidase use in Hurler Syndrome	Laronidase	Laronidase receiving prior and post transplant

Primary Outcome Measures

Name	Time	Method
Overall Survival	At 1 Year	Patients alive at 1 year post transplantation.

Secondary Outcome Measures

Name	Time	Method
Incidence of Engraftment	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.
Incidence of Grade III-IV Acute Graft Versus Host Disease	Day 100	Cumulative incidence will be used to estimate grade III-IV acute GvHD, treating death as a competing risk.
Proportion of patients in need of ventilator support	1 Year	Count of patients using ventilator by 1 year.

Trial Locations

Locations (1)

Masonic Cancer Center, University of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States