Evaluating Laronidase Treatment in Young Patients Undergoing Stem Cell Transplant
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
Masonic Cancer Center, University of Minnesota · NCT05634512
This study is testing how well the drug laronidase works in young patients with a specific genetic disorder who are getting a stem cell transplant.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 24 (estimated) |
| Ages | 0 Years to 3 Years |
| Sex | All |
| Sponsor | Masonic Cancer Center, University of Minnesota (other) |
| Locations | 1 site (Minneapolis, Minnesota) |
| Trial ID | NCT05634512 on ClinicalTrials.gov |
What this trial studies
This observational study aims to collect blood samples from patients with mucopolysaccharidosis type IH who are receiving laronidase therapy before and after undergoing hematopoietic cell transplantation. The study will enroll sixteen patients over a 24-month period, focusing on the pharmacokinetics of laronidase in this specific patient population. By monitoring the effects of laronidase in conjunction with stem cell transplant, the study seeks to gather important data that could inform future treatment protocols.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 0 to 3 years with mucopolysaccharidosis type IH who are planning to receive both laronidase and a stem cell transplant.
Not a fit: Patients whose parents or legal guardians cannot provide informed consent will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance understanding of laronidase therapy's effectiveness in young patients undergoing stem cell transplantation.
How similar studies have performed: While this study focuses on a specific patient population and treatment combination, similar approaches in other studies have shown promise in understanding enzyme therapies in conjunction with transplantation.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Minneapolis, Minnesota
- University of Minnesota Masonic Cancer Center — Minneapolis, Minnesota, United States (RECRUITING)
Study contacts
- Principal investigator: Paul Orchard — University of Minnesota Masonic Cancer Center
- Study coordinator: Kim Nelson, RN
- Email: knelso62@fiarview.org
- Phone: 612-273-2925
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I, MPS I, HCT