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

MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).

Status

Terminated

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT01572636

Enrollment

Registered

2012-04-06

Start date

2012-03-28

Completion date

2018-05-01

Last updated

2018-09-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Mucopolysaccharidosis Type IH, MPS I, Hurler Syndrome

Keywords

Hurler Syndrome

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.

Interventions

DRUGLaronidase

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.

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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)

Design outcomes

Primary

Measure	Time frame	Description
Overall Survival	At 1 Year	Patients alive at 1 year post transplantation.

Secondary

Measure	Time frame	Description
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.

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026