Clinical trial

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

Name

2021LS144

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.

Trial arms

Trial start

2022-11-17

Estimated PCD

2024-10-01

Trial end

2025-10-01

Status

Recruiting

Treatment

Laronidase therapy and a stem cell transplant

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.

Arms:

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

Size

Primary endpoint

Identify covariates that impact drug exposure

2 years

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

2 years

Eligibility criteria

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.

Protocol

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'CASE_ONLY', 'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'count': 24, 'type': 'ESTIMATED'}}

Updated at

2023-11-30

1 organization

1 product

2 indications

Organization

Masonic Cancer Center, University of Minnesota

Product

Laronidase

Indication

Hematopoietic Cell Transplantation

Indication

Mucopolysaccharidosis Type I