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

N/AActive Not RecruitingNCT05634512

Masonic Cancer Center, University of Minnesota13 enrolled

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Hematopoietic Cell Transplantation Mucopolysaccharidosis Type I

Interventions

Laronidase therapy and a stem cell transplantDRUG

To identify key differences leading to variability in PK parameters for patients receiving IV laronidase therapy for the treatment of MPS-IH. There will be 12 samples per patient (6 pre-transplant and 6-post-transplant). Laronidase will be administered IV per protocol using standard dosing (0.58 mg/kg intravenously on a weekly basis), and six (n=6) blood samples will be collected over 24 hours for the determination of mononuclear cell lysates and plasma laronidase concentrations for a total of 18mL. The second PK monitoring will be obtained using the same PK design but following complete or near-completedonor derived myeloid engraftment which is evaluated at different time pointspost-HCT (day 30, day 42, day 60). The standard is to continue ERT through 8 weeks post-transplant.

Eligibility

Sex: ALLMin age: 0 YearsMax age: 3 Years

Medical Language ↔ Plain English

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.

Locations (1)

University of Minnesota Masonic Cancer Center

Minneapolis, Minnesota, United States

Outcomes

Primary Outcomes

Identify covariates that impact drug exposure

Measure indicators for body size and maturation contribute to variability in laronidase exposure in patients with MPS IH.

Time frame: 2 years

Identify key differences pre- and post-HCT leading to variability in PK parameters

Measure endogenous source of enzyme present in relation to the transplanted cells.

Time frame: 2 years

Data from ClinicalTrials.gov

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