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

Hematopoietic Cell Transplantation Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05634512
• Other study ID #: 2021LS144
• Secondary ID: NCI-2022-09774MT
• Status: Recruiting
• Start date: November 17, 2022
• Est. completion date: October 1, 2025

Study information

• Verified date: November 2023
• Source: Masonic Cancer Center, University of Minnesota
• Contact: Kim Nelson, RN
• Phone: 612-273-2925
• Email: knelso62[@]fiarview.org
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 24
• Est. completion date: October 1, 2025
• Est. primary completion date: October 1, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 3 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hematopoietic Cell Transplantation
• Mucopolysaccharidoses
• Mucopolysaccharidosis I
• Mucopolysaccharidosis Type I

Intervention

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.

Locations

Country	Name	City	State
United States	University of Minnesota Masonic Cancer Center	Minneapolis	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Masonic Cancer Center, University of Minnesota

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Identify covariates that impact drug exposure	Measure indicators for body size and maturation contribute to variability in laronidase exposure in patients with MPS IH.	2 years
Primary	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.	2 years