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

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Hematopoietic Cell Transplantation Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis I Mucopolysaccharidosis Type I Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Age range

0 year–3 year

Sex eligibility

All sexes

Study type

Observational

Primary location

University of Minnesota Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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.

Primary outcomes

Identify covariates that impact drug exposure

Time frame: 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

Time frame: 2 years

Measure endogenous source of enzyme present in relation to the transplanted cells.

Sponsors and collaborators

Lead sponsor

Masonic Cancer Center, University of Minnesota

Other

Registry information

Important dates

Study start: 2022
Primary completion: 2025
Study completion: 2026
First posted: Dec 2, 2022
Registry last updated: Oct 30, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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