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

Recruiting

• Conditions: Hematopoietic Cell Transplantation; Mucopolysaccharidosis Type I
• Interventions: Drug: Laronidase therapy and a stem cell transplant

• Registration Number: NCT05634512
• Lead Sponsor: Masonic Cancer Center, University of Minnesota

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2022-11-17
• Study First Submitted: 2022-11-22
• Study First Submitted QC: 2022-11-22
• Study First Posted: 2022-12-02
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-01
• Last Update Posted: 2024-11-04
• Primary Completion: 2025-10-01
• Study Completion: 2026-10-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 24

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Collect blood from patients with MPS-IH) undergoing laronidase therapy and stem cell transplant.	Laronidase therapy and a stem cell transplant	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.

Primary Outcome Measures

Name	Time	Method
Identify covariates that impact drug exposure	2 years	Measure indicators for body size and maturation contribute to variability in laronidase exposure in patients with MPS IH.
Identify key differences pre- and post-HCT leading to variability in PK parameters	2 years	Measure endogenous source of enzyme present in relation to the transplanted cells.

Trial Locations

Locations (1)

University of Minnesota Masonic Cancer Center; 🇺🇸 Minneapolis, Minnesota, United States