Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
Conditions
Keywords
MPS I, HCT
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.
Interventions
DRUGLaronidase 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
Masonic Cancer Center, University of Minnesota
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
0 Years to 3 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Identify covariates that impact drug exposure | 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 | 2 years | Measure endogenous source of enzyme present in relation to the transplanted cells. |
Countries
United States
Outcome results
None listed