Ignite Creation Date:
2024-05-05 @ 10:52 PM
Last Modification Date:
2024-10-26 @ 10:25 AM
Study NCT ID:
NCT01206075
Status:
COMPLETED
Last Update Posted:
2014-12-30
First Post:
2010-09-20
Brief Title:
Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major
Sponsor:
University of Washington
Organization:
University of Washington
Study Overview
Official Title:
A Pilot Study to Assess the Safety and Efficacy of Mozobil G-CSF in Mobilizing Hematopoietic Stem Cells CD34 Cells in Adults With Beta-thalassemia Major
Status:
COMPLETED
Status Verified Date:
2014-12
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:
Thalassemia is considered the most common genetic disorder worldwide occurring with high frequency in Mediterranean areas the Middle East Southeast Asia and the Pacific Islands Currently the only cure for thalassemia is bone marrow transplantation from a related compatible donor Gene transfer achieved by transplantation of the patients own blood stem cells that have been genetically-modified with the corrected gene could potentially cure thalassemia
The first step in developing gene transfer for treatment of thalassemia is to develop a safe and effective method to obtain blood stem cells from thalassemia patients Eventually high numbers of genetically modified cells will need to be infused into the patient for clinical gene transfer to be effective The blood stem cells are obtained by giving a mobilization agent to the patients This causes the stem cells to leave the bone marrow and go into the blood The purpose of this study is to test the safety and effectiveness of the new mobilization agent Mozobil in causing mobilization of blood stem cells for patients with beta-thalassemia major
The first step in developing gene transfer for treatment of thalassemia is to develop a safe and effective method to obtain blood stem cells from thalassemia patients Eventually high numbers of genetically modified cells will need to be infused into the patient for clinical gene transfer to be effective The blood stem cells are obtained by giving a mobilization agent to the patients This causes the stem cells to leave the bone marrow and go into the blood The purpose of this study is to test the safety and effectiveness of the new mobilization agent Mozobil in causing mobilization of blood stem cells for patients with beta-thalassemia major
Detailed Description:
The purpose of this study is to optimize blood stem cell mobilization in adults with beta thalassemia major We seek a method of mobilization that will be safe with minimum side effects and that will yield high numbers of blood stem cells For successful gene therapy of thalassemia high numbers of genetically modified stem cells will need to be introduced into the patient Participants will include beta-thalassemia patients who failed to mobilize sufficiently with G-CSF in our previous protocol and new patients In this study we will focus on the safety and effectiveness of mobilization with Mozobil or with Mozobil plus G-CSF Following mobilization blood stem cells will be recovered using leukapheresis a procedure similar to a blood donation in which mobilized white blood cells are collected from the blood of the patient
During drug administration and leukapheresis patients will be hospitalized at George Papanicolaou Hospital in Thessaloniki Greece Patients who failed to mobilize in the previous protocol will receive Mozobil and G-CSF and will be hospitalized for 5-8 days for the duration of drug administration and leukapheresis They will receive G-CSF for several days Mozobil will be added on the last few days of G-CSF New patients will receive Mozobil only and will be hospitalized for 2-3 days for drug administration and leukapheresis Mozobil is administered at 240µgkg under the skin Participants will undergo two or three leukapheresis procedures in a row if low numbers of blood stem cells were recovered in the first and possibly second leukapheresis Participants will be discharged from the hospital the day following the last leukapheresis procedure Weekly follow-up visits will occur for the next month either at G Papanicolaou Hospital or at the participants local doctors office The total period of study participation is approximately 5 weeks In the event that Mozobil alone does not cause mobilization of high levels of blood cells patients will be invited to repeat the protocol three months later receiving Mozobil and G-CSF
During drug administration and leukapheresis patients will be hospitalized at George Papanicolaou Hospital in Thessaloniki Greece Patients who failed to mobilize in the previous protocol will receive Mozobil and G-CSF and will be hospitalized for 5-8 days for the duration of drug administration and leukapheresis They will receive G-CSF for several days Mozobil will be added on the last few days of G-CSF New patients will receive Mozobil only and will be hospitalized for 2-3 days for drug administration and leukapheresis Mozobil is administered at 240µgkg under the skin Participants will undergo two or three leukapheresis procedures in a row if low numbers of blood stem cells were recovered in the first and possibly second leukapheresis Participants will be discharged from the hospital the day following the last leukapheresis procedure Weekly follow-up visits will occur for the next month either at G Papanicolaou Hospital or at the participants local doctors office The total period of study participation is approximately 5 weeks In the event that Mozobil alone does not cause mobilization of high levels of blood cells patients will be invited to repeat the protocol three months later receiving Mozobil and G-CSF
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2P01HL053750 | NIH | None | httpsreporternihgovquickSearch2P01HL053750 |
| P01HL053750 | NIH | None | None |