Ignite Creation Date:
2025-12-26 @ 10:43 PM
Ignite Modification Date:
2025-12-30 @ 5:09 PM
Study NCT ID:
NCT05634512
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-10-30
First Post:
2022-11-22
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
Sponsor:
Masonic Cancer Center, University of Minnesota
Organization:
Study Overview
Official Title:
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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.
Sixteen patients will be enrolled over a 24 month period.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: