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

NCT05634512 | Active Not Recruiting DMC

• 3 other identifiers: 2021LS144NCI-2022-09774MT2021-29
• Study Type: observational
• Target: 13
• Locations: 1 country
• Sites: 1

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.

Conditions

Hematopoietic Cell Transplantation; Mucopolysaccharidosis Type I

Keywords

MPS I; HCT

Outcome Measures

Primary Outcomes (2)

• 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

• 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

Study Arms (1)

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

Drug: Laronidase therapy and a stem cell transplant

Interventions

Laronidase therapy and a stem cell transplant DRUG

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.

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

Eligibility Criteria

• Age: 0 Years - 3 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Study entry is open to pediatric patients regardless of gender or ethnic background. While there will be every effort to seek out and include females and minority patients, the patient population is dependent upon the MPS-IH populations at the University of Minnesota.

You may qualify if:

• 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.

You may not qualify if:

• Patient's parent/ legal guardians are unable to provide informed consent.

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota: lead

Study Sites (1)

University of Minnesota Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Mucopolysaccharidosis I

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Paul Orchard

  University of Minnesota Masonic Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 22, 2022
• First Posted: December 2, 2022
• Study Start: November 17, 2022
• Primary Completion: October 28, 2025
• Study Completion (Estimated): October 1, 2026
• Last Updated: October 30, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)