NCT05634512

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

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 13 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 0 Years – 3 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hematopoietic Cell Transplantation
• Mucopolysaccharidosis Type I

Interventions

Type	Name	Description
DRUG	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.

Timeline

• Start date: 2022-11-17
• Primary completion: 2025-10-28
• Completion: 2026-10-01
• First posted: 2022-12-02
• Last updated: 2025-10-30

Locations

1 site across 1 country: United States