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

University of Minnesota (UMN)

Status: Active, not recruiting

Conditions: Hematopoietic Cell Transplantation; Mucopolysaccharidosis Type I

Treatments: Drug: Laronidase therapy and a stem cell transplant

Study type: Observational

Funder types: Other

Identifiers

NCT05634512

2021LS144

NCI-2022-09774 (Registry Identifier)

MT2021-29 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 13 patients

Sex: All

Ages: Under 3 years old

Volunteers: No Healthy Volunteers

Inclusion 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.

Exclusion criteria

• Patient's parent/ legal guardians are unable to provide informed consent.

Trial design

13 participants in 1 patient group

Collect blood from patients with MPS-IH) undergoing laronidase therapy and stem cell transplant.

Description:

The primary aim is to characterize the PK of IV laronidase in individuals with MPS IH and identify patient specific covariates that impact drug exposure. The secondary aim is to identify key differences pre-and post-HCT leading to variability in PK parameters for patients receiving IV laronidase therapy for the treatment of MPS-IH.

Treatment:

Drug: Laronidase therapy and a stem cell transplant