Ignite Creation Date:
2024-05-05 @ 9:57 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001630
Status:
COMPLETED
Last Update Posted:
2017-07-02
First Post:
1999-11-03
Brief Title:
Treatment of Autoimmune Thrombocytopenia AITP
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
High-Dose Cyclophosphamide With CD34 Selected Autologous Hematopoietic Cell Support for Treatment of Refractory Chronic Autoimmune Thrombocytopenia
Status:
COMPLETED
Status Verified Date:
2011-03-21
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:
Platelets are particles found along with red and white blood cells in the blood that play a role in the process of blood clotting Disorders affecting the platelets can lower the amount of platelets in the blood and put patients at risk of bleeding The condition of low platelets is referred to as thrombocytopenia
Thrombocytopenia can be associated with a variety of diseases including cancer leukemia tuberculosis or as a result of an autoimmune reaction Autoimmune reactions are disorders in which the normal immune system begins attacking itself Autoimmune thrombocytopenia AITP is a disorder of low blood platelet counts in which platelets are destroyed by antibodies produced by the immune system
Unfortunately many patients with AITP do not respond to standard treatments for thrombocytopenia Cyclophosphamide is a drug that works to suppress the activity of the immune system Researchers believe that combining this drug with transplanted rescued blood stem cells may provide effective treatment for AITP
The purpose of this study is to explore the affordability and safety of this therapy for the treatment of AITP The effectiveness of the therapy will be measured by the number of patients whose platelet levels rise greater than 100000m3
If this treatment approach appears affordable this study will form the basis for a larger study to compare alternate treatment approaches
Thrombocytopenia can be associated with a variety of diseases including cancer leukemia tuberculosis or as a result of an autoimmune reaction Autoimmune reactions are disorders in which the normal immune system begins attacking itself Autoimmune thrombocytopenia AITP is a disorder of low blood platelet counts in which platelets are destroyed by antibodies produced by the immune system
Unfortunately many patients with AITP do not respond to standard treatments for thrombocytopenia Cyclophosphamide is a drug that works to suppress the activity of the immune system Researchers believe that combining this drug with transplanted rescued blood stem cells may provide effective treatment for AITP
The purpose of this study is to explore the affordability and safety of this therapy for the treatment of AITP The effectiveness of the therapy will be measured by the number of patients whose platelet levels rise greater than 100000m3
If this treatment approach appears affordable this study will form the basis for a larger study to compare alternate treatment approaches
Detailed Description:
Autoimmune Thrombocytopenia AITP is a disorder of low blood platelet counts in which platelet destruction is caused by antiplatelet autoantibodies A large proportion of patients with chronic AITP are refractory to standard therapies including corticosteroids immune globulin and splenectomy Cyclophosphamide is a cytotoxic immunosuppressive agent which may induce durable remissions of refractory autoimmune diseases High-dose cyclophosphamide with peripheral blood stem cell PBPC rescue has been proposed as a potential definitive therapy for AITP however the infusion of autoreactive lymphocytes could result in relapse The use of PBPC depleted of T-lymphocytes could circumvent this limitation
The purpose of this phase III study is to explore the feasibility and safety of this approach and to seek preliminary evidence of effectiveness of using high-dose cyclophosphamide 50 mgkgday x 4 followed by infusion of autologous PBPC enriched for CD34 cells concomitantly depleted of CD3 cells for the treatment of patients with refractory AITP Safetyfeasibility parameters to be examined will include the ability to mobilize harvest and purify sufficient PBPC to yield greater than 2 x 106 CD34 cellskg symptomatic acceptability and hematologic toxicities of the mobilization regimen filgrastim 10 microgramskgday IV tolerability of the leukapheresis procedure including the central line placement and maintenance depth and duration of blood cell nadirs following chemotherapy peritransplant bleeding episodes and transfusion requirements episodes of febrile neutropenia culture-proven infections and antibiotic usage Effectiveness will be gauged by the rapidity and number of patients to achieve complete remission platelet count greater than 100000mm3 and partial remission platelet count greater than 50000mm3 or doubling of the platelet count with resolution of bleeding episodes Ancillary evidence of therapeutic effect will be sought by examining changes in titers of platelet surface glycoprotein antibodies In addition alterations in T-lymphocyte subsets will be examined by flow cytometry If this treatment approach appears feasible this study will form the basis for a larger trial to compare alternate treatment approaches
The purpose of this phase III study is to explore the feasibility and safety of this approach and to seek preliminary evidence of effectiveness of using high-dose cyclophosphamide 50 mgkgday x 4 followed by infusion of autologous PBPC enriched for CD34 cells concomitantly depleted of CD3 cells for the treatment of patients with refractory AITP Safetyfeasibility parameters to be examined will include the ability to mobilize harvest and purify sufficient PBPC to yield greater than 2 x 106 CD34 cellskg symptomatic acceptability and hematologic toxicities of the mobilization regimen filgrastim 10 microgramskgday IV tolerability of the leukapheresis procedure including the central line placement and maintenance depth and duration of blood cell nadirs following chemotherapy peritransplant bleeding episodes and transfusion requirements episodes of febrile neutropenia culture-proven infections and antibiotic usage Effectiveness will be gauged by the rapidity and number of patients to achieve complete remission platelet count greater than 100000mm3 and partial remission platelet count greater than 50000mm3 or doubling of the platelet count with resolution of bleeding episodes Ancillary evidence of therapeutic effect will be sought by examining changes in titers of platelet surface glycoprotein antibodies In addition alterations in T-lymphocyte subsets will be examined by flow cytometry If this treatment approach appears feasible this study will form the basis for a larger trial to compare alternate treatment approaches
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 |
|---|---|---|---|
| 97-H-0154 | None | None | None |