Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

Description

This is a prospective, observational multicenter study to collect blood from patients with mucopolysaccharidosis type IH undergoing laronidase therapy and a stem cell transplant. Sixteen patients will be enrolled over a 24 month period.

Conditions

Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I

Talk to us

Study Overview

On this page

Study Details

Study overview

This is a prospective, observational multicenter study to collect blood from patients with mucopolysaccharidosis type IH undergoing laronidase therapy and a stem cell transplant. Sixteen patients will be enrolled over a 24 month period.

Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

Condition
Hematopoietic Cell Transplantation
Intervention / Treatment

-

Contacts and Locations

Minneapolis

University of Minnesota Masonic Cancer Center, Minneapolis, Minnesota, United States, 55455

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Between 0 to 3 years of age
  • * Meet protocol specific eligibility criteria for allogeneic HCT for MPS IH
  • * Planning to receive laronidase both pre and post-transplant in an inpatient setting as part of standard-of-care treatment. Virtually all patients with MPSIH being considered for transplantation at the University of Minnesota are already receiving enzyme infusions, and it is standard practice to continue to give enzyme infusions to 8 weeks post-transplant. Therefore, participation will not modify the treatment course.
  • * Patient's parent/ legal guardians are unable to provide informed consent.

Ages Eligible for Study

0 Years to 3 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Masonic Cancer Center, University of Minnesota,

Paul Orchard, PRINCIPAL_INVESTIGATOR, University of Minnesota Masonic Cancer Center

Study Record Dates

2026-10-01