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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
Sponsor:
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati

Brief Summary:
The purpose of this trial is to study the safety and effectiveness of a drug called Bortezomib for the treatment of low blood cell counts after bone marrow transplant.

Condition or disease Intervention/treatment Phase
Allogeneic Stem Cell Transplantation Refractory Autoimmune Cytopenia(s) Drug: Bortezomib Phase 2

Detailed Description:

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.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

Drug Information available for: Bortezomib

Arm Intervention/treatment
Experimental: Bortezomib

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

Drug: Bortezomib
Other Names:
  • PS-341
  • Velcade




Primary Outcome Measures :
  1. Number of Participants With Response [ Time Frame: 6 weeks ]

    For Autoimmune Hemolytic Anemia- At least 3 of 5 criteria should be met.

    1. Stabilization of hemoglobin without transfusions by 2 weeks
    2. Conversion of DAT from + to - by 6 weeks
    3. Normalization of serum haptoglobin levels by 6 weeks
    4. Normalization of indirect bilirubin levels by 6 weeks
    5. Reduction in the frequency of transfusions by 50% by 4 weeks

    For Autoimmune Neutropenia- At least 2 of 3 criteria should be met.

    1. Stabilization of absolute neutrophil count by 2 weeks
    2. Undetectable antineutrophil antibodies by 6 weeks
    3. Reduction in GCSF dose by 50% by 6 weeks

    For Autoimmune Thrombocytopenia- At least 2 of 3 criteria should be met.

    1. Stabilization of platelet count without platelet transfusions by 2 weeks
    2. Undetectable antiplatelet antibodies by 6 weeks
    3. Reduction in the frequency of platelet transfusions by 50% from pre-bortezomib values by 6 weeks



Information from the National Library of Medicine

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Ages Eligible for Study:   4 Months to 29 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • All patients, having undergone allogeneic stem cell transplantation at our center.
  • Should have failed at least 2 standard treatments for autoimmune cytopenias. Standard treatments include corticosteroids, rituximab, IVIG, plasmapheresis, withdrawal of cyclosporine, cyclophosphamide and MMF. Definition of "failed" treatment will be no response of cytopenia after 2 weeks of continued treatment OR requirement of daily GCSF at 10 mcgs/kg/day for autoimmune neutropenia despite 2 weeks of treatment, transfusions of packed red blood cells or platelets 3 times weekly for 2weeks despite continued treatment OR 5days/week plasmapheresis for 2 weeks and inability to wean the duration.
  • Definition of autoimmune hemolytic anemia- development of anemia, where there is a hemoglobin drop of >2 g/dL/48 hours or an absolute value of hemoglobin < 8 g/dL, and evidence of hemolysis by positive direct Coombs test with compatible peripheral blood cell morphology, reticulocyte count and bilirubin level.
  • Definition of autoimmune neutropenia - absolute neutrophil counts < 500 for 2 weeks and presence of anti-neutrophil antibodies.
  • Definition of autoimmune thrombocytopenia- Platelet counts < 20,000 cells/uL for 2 weeks and presence of anti-platelet antibodies.

Exclusion Criteria:

  • Ongoing life threatening infections
  • Documented anaphylaxis to bortezomib
  • Failed engraftment
  • Relapse of primary malignancy
  • ≥6/8 matched or haploidentical transplants

Information from the National Library of Medicine

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


Locations
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United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Investigators
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Principal Investigator: Lisa Filipovich, MD Children's Hospital Medical Center, Cincinnati

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Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT01929980     History of Changes
Other Study ID Numbers: 2012:1089
First Posted: August 28, 2013    Key Record Dates
Results First Posted: October 24, 2016
Last Update Posted: October 24, 2016
Last Verified: October 2016
Keywords provided by Children's Hospital Medical Center, Cincinnati:
HSCT
cytopenia
immune suppression
proteasome inhibitor
Additional relevant MeSH terms:
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Anemia
Leukopenia
Thrombocytopenia
Hematologic Diseases
Leukocyte Disorders
Blood Platelet Disorders
Bortezomib
Antineoplastic Agents