Ignite Creation Date:
2024-05-05 @ 11:07 AM
Last Modification Date:
2024-10-26 @ 9:04 AM
Study NCT ID:
NCT00004454
Status:
COMPLETED
Last Update Posted:
2005-06-24
First Post:
1999-10-18
Brief Title:
Phase III Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II Mild Hunter Syndrome
Sponsor:
Eunice Kennedy Shriver National Institute of Child Health and Human Development NICHD
Organization:
Office of Rare Diseases ORD
Study Overview
Official Title:
None
Status:
COMPLETED
Status Verified Date:
2003-10
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:
OBJECTIVES I Evaluate the safety and feasibility of treating mucopolysaccharidosis II mild Hunter syndrome by lymphocyte gene therapy
II Determine the levels of iduronate-2-sulfatase enzyme in these patients attained by infusing increasing doses of lymphocytes transduced with a retroviral vector designed for insertion and expression of this iduronate-2-sulfatase gene L2SN
III Determine the duration of survival of these transduced cells in these patients
IV Determine whether monthly infusion of L2SN-transduced lymphocytes accomplishes metabolic correction as measured by glycosaminoglycan excretion decrease in liver or spleen volume any therapeutic effect upon cardiac and pulmonary dysfunction or any other effects from treatment
II Determine the levels of iduronate-2-sulfatase enzyme in these patients attained by infusing increasing doses of lymphocytes transduced with a retroviral vector designed for insertion and expression of this iduronate-2-sulfatase gene L2SN
III Determine the duration of survival of these transduced cells in these patients
IV Determine whether monthly infusion of L2SN-transduced lymphocytes accomplishes metabolic correction as measured by glycosaminoglycan excretion decrease in liver or spleen volume any therapeutic effect upon cardiac and pulmonary dysfunction or any other effects from treatment
Detailed Description:
PROTOCOL OUTLINE Peripheral blood lymphocytes are harvested from patient by apheresis stimulated to initiate the growth of T-lymphocytes transduced with retrovirus L2SN containing iduronate-2-sulfatase and reinfused into the patient
Patients receive 12 monthly infusions of these retroviral-mediated gene transduced lymphocytes with the first three infusions in a dose escalation format
Patients are monitored for at least 2 hours after completion of each infusion Patients are followed at 1 year after treatment and then until death
Patients receive 12 monthly infusions of these retroviral-mediated gene transduced lymphocytes with the first three infusions in a dose escalation format
Patients are monitored for at least 2 hours after completion of each infusion Patients are followed at 1 year after treatment and then until death
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?:
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| UMN-5P01HD32652 | None | None | None |
| UMN-HUNTER | None | None | None |