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

NCT05634512 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 13

Last updated 2025-10-30

No results posted yet for this study

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

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.

Sponsors & Collaborators

  • Masonic Cancer Center, University of Minnesota

    lead OTHER

Principal Investigators

  • Paul Orchard · University of Minnesota Masonic Cancer Center

Eligibility

Min Age
0 Years
Max Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-11-17
Primary Completion
2025-10-28
Completion
2026-10-01

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05634512 on ClinicalTrials.gov