Ignite Creation Date:
2024-05-05 @ 12:08 PM
Last Modification Date:
2024-10-26 @ 9:21 AM
Study NCT ID:
NCT00258011
Status:
COMPLETED
Last Update Posted:
2014-03-07
First Post:
2005-11-22
Brief Title:
Study of Aldurazyme Replacement Therapy in Patients With Mucopolysaccharidosis I MPS I Disease
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
A Safety Confirmatory Study of JC0498 Laronidase in Mucopolysaccharidosis I MPS I Patients
Status:
COMPLETED
Status Verified Date:
2014-02
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 multi-center open label study conducted to evaluate the safety of laronidase administered by intravenous drip infusion in Japanese patients with MPS I disease
Following baseline evaluation patients will receive weekly infusions of JC0498 at an intravenous dose of 100 unitskg Patient safety will be monitored continuously throughout the trial In addition the effects of JC0498 treatment in this patient population will be assessed by periodically evaluating aspects of MPS I disease in patients at scheduled intervals over the duration of the trial
Since patients may be eligible for the trial if they have received JC0498 a portion of the data may be captured retrospectively and recorded onto the case report forms CRFs
This study represents the first good clinical practice GCP effort to characterize MPS I in the Japanese population and evaluate the effects of JC0498 on disease manifestations
Following baseline evaluation patients will receive weekly infusions of JC0498 at an intravenous dose of 100 unitskg Patient safety will be monitored continuously throughout the trial In addition the effects of JC0498 treatment in this patient population will be assessed by periodically evaluating aspects of MPS I disease in patients at scheduled intervals over the duration of the trial
Since patients may be eligible for the trial if they have received JC0498 a portion of the data may be captured retrospectively and recorded onto the case report forms CRFs
This study represents the first good clinical practice GCP effort to characterize MPS I in the Japanese population and evaluate the effects of JC0498 on disease manifestations
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None