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MT2021-29: Evaluation of intravenous laronidase pharmacokinetics before and after hematopoietic cell transplantation in patients with mucopolysaccharidosis type IH

Status: Recruiting

This is a prospective, observational multicenter study to collect blood from patients with mucopolysaccharidosis type IH (MPS-IH) undergoing laronidase therapy and a stem cell transplant.

I'm interested

Age: Up to 18 years old
Healthy Volunteers:
This study is NOT accepting healthy volunteers
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.
Conditions:

Rare Diseases, Cancer

Keywords:

Hematopoietic Cell Transplantation

Contact(s): Paul Orchard - orcha001@umn.edu
Principal Investigator: Silvia Illamola
IRB Number: STUDY00016560
System ID: 35782
See this study on ClinicalTrials.gov

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