Bortezomib to Treat Significant Complication of HSCT (Bortezomib)
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|ClinicalTrials.gov Identifier: NCT01929980|
Recruitment Status : Completed
First Posted : August 28, 2013
Results First Posted : October 24, 2016
Last Update Posted : October 24, 2016
|Condition or disease||Intervention/treatment||Phase|
|Allogeneic Stem Cell Transplantation Refractory Autoimmune Cytopenia(s)||Drug: Bortezomib||Phase 2|
The purpose of this research study is to study the safety and effectiveness of a drug called bortezomib for the treatment of autoimmune cytopenia(s) (low blood cell counts) after bone marrow transplant that are not responding to standard treatments. Autoimmune cytopenias are low blood counts due to antibodies or proteins produced against an individual's own blood cells. Having a low red blood cell count (anemia) can make a person feel tired and require blood transfusions frequently. A low platelet count (blood cells that help blood to clot) can make a person bleed or bruise easily. A low neutrophil (white blood cell) count can make a person have infections.
All of these things can be a serious complication after bone marrow transplant and can cause prolonged hospital stay. Bortezomib is being used in children with certain types of blood cancer, however, bortezomib has not been used in children with autoimmune cytopenia(s) and its use in this study is investigational.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Use of Bortezomib to Treat Refractory Autoimmune Cytopenia(s) in Allogeneic Stem Cell Transplantation|
|Study Start Date :||July 2012|
|Actual Primary Completion Date :||May 2014|
|Actual Study Completion Date :||May 2014|
Four doses of bortezomib, 1.3mg/m2, will be given intravenously (through a needle in a vein) or subcutaneously (under the skin) on Days 1, 4, 8, 11.
The format of receiving medications is- Therapy Dose and Route Frequency Rituximab 375 mg/m2 intravenously Once on day 1. Plasmapheresis 2 hours prior to Bortezomib Day 1,4, 8 and 11 Bortezomib 1.3 mg/m2 intravenously Day 1,4,8 and 11
- Number of Participants With Response [ Time Frame: 6 weeks ]
For Autoimmune Hemolytic Anemia- At least 3 of 5 criteria should be met.
- Stabilization of hemoglobin without transfusions by 2 weeks
- Conversion of DAT from + to - by 6 weeks
- Normalization of serum haptoglobin levels by 6 weeks
- Normalization of indirect bilirubin levels by 6 weeks
- Reduction in the frequency of transfusions by 50% by 4 weeks
For Autoimmune Neutropenia- At least 2 of 3 criteria should be met.
- Stabilization of absolute neutrophil count by 2 weeks
- Undetectable antineutrophil antibodies by 6 weeks
- Reduction in GCSF dose by 50% by 6 weeks
For Autoimmune Thrombocytopenia- At least 2 of 3 criteria should be met.
- Stabilization of platelet count without platelet transfusions by 2 weeks
- Undetectable antiplatelet antibodies by 6 weeks
- Reduction in the frequency of platelet transfusions by 50% from pre-bortezomib values by 6 weeks
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01929980
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|Principal Investigator:||Lisa Filipovich, MD||Children's Hospital Medical Center, Cincinnati|