ACTIVE_NOT_RECRUITING

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

Conditions

Hematopoietic Cell Transplantation Mucopolysaccharidosis Type I MPS I HCT

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2022-11-17

Study Completion:2026-10-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05634512

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:0 Years to 3 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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.	* Patient's parent/ legal guardians are unable to provide informed consent.

Inclusion Criteria

Exclusion 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.

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

Contacts and Locations

Principal Investigator

Paul Orchard

PRINCIPAL_INVESTIGATOR

University of Minnesota Masonic Cancer Center

Study Locations (Sites)

University of Minnesota Masonic Cancer Center

Minneapolis, Minnesota, 55455

United States

Collaborators and Investigators

Sponsor: Masonic Cancer Center, University of Minnesota

Paul Orchard, PRINCIPAL_INVESTIGATOR, University of Minnesota Masonic Cancer Center

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-11-17

Study Completion Date2026-10-01

Study Record Updates

Study Start Date2022-11-17

Study Completion Date2026-10-01

Terms related to this study

Keywords Provided by Researchers

MPS I
HCT

Additional Relevant MeSH Terms

Hematopoietic Cell Transplantation
Mucopolysaccharidosis Type I