|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
11 November 2024 |
|
Main ID: |
NCT05634512 |
|
Date of registration:
|
22/11/2022 |
|
Prospective Registration:
|
No |
|
Primary sponsor: |
|
|
Public title:
|
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
|
|
Scientific title:
|
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH. |
|
Date of first enrolment:
|
November 17, 2022 |
|
Target sample size:
|
24 |
|
Recruitment status: |
Recruiting |
|
URL:
|
https://clinicaltrials.gov/ct2/show/NCT05634512 |
|
Study type:
|
Observational |
|
Study design:
|
|
|
Phase:
|
|
|
|
Countries of recruitment
|
|
United States
| | | | | | | |
|
Contacts
|
|
Name:
|
Paul Orchard |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
University of Minnesota Masonic Cancer Center |
|
|
Name:
|
Kim Nelson, RN |
|
Address:
|
|
|
Telephone:
|
612-273-2925 |
|
Email:
|
|
|
Affiliation:
|
|
|
|
Name:
|
Kim Nelson, RN |
|
Address:
|
|
|
Telephone:
|
612-273-2925 |
|
Email:
|
knelso62@fiarview.org |
|
Affiliation:
|
|
|
|
Key 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.
Age minimum:
0 Years
Age maximum:
3 Years
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Hematopoietic Cell Transplantation
|
|
Mucopolysaccharidosis Type I
|
|
Intervention(s)
|
|
Drug: Laronidase therapy and a stem cell transplant
|
|
Primary Outcome(s)
|
|
Identify covariates that impact drug exposure
[Time Frame: 2 years]
|
|
Identify key differences pre- and post-HCT leading to variability in PK parameters
[Time Frame: 2 years]
|
|
Secondary ID(s)
|
|
2021LS144
|
|
NCI-2022-09774
|
|
MT2021-29
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|