Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)
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|ClinicalTrials.gov Identifier: NCT01666080|
Recruitment Status : Recruiting
First Posted : August 16, 2012
Last Update Posted : November 3, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Hematologic Disorders Hemoglobinopathies Immunodeficiencies||Drug: Busulfan Drug: Fludarabine Radiation: Total body irradiation Biological: Stem cell transplant Drug: Keppra||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)|
|Actual Study Start Date :||August 2012|
|Estimated Primary Completion Date :||June 2023|
|Estimated Study Completion Date :||June 2023|
Reduced Intensity Conditioning
Includes patients receiving a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using reduced intensity conditioning (RIC). Patients will receive busulfan, fludarabine, total body irradiation and stem cell transplant. Keppra will be given for seizure prophylaxis.
0.4 mg/kg (0.5 mg/kg if <4 years of age) intravenously (IV) every 6 hours on Days -8 and -7.
Other Name: Busulfex
40 mg/m^2 intravenously (IV) over 1 hour on days -6 through -2.
Other Name: Fludara
Radiation: Total body irradiation
200 cGy on Day -1
Biological: Stem cell transplant
stem cell infusion on day 0
Keppra will be given for seizure prophylaxis during busulfan administration as per the standard institutional protocol.
Other Name: Levetiracetam
- Time to Engraftment [ Time Frame: Day 42 ]Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm3 (0.5 x 109/L) or greater.
- Incidence of Graft Failure [ Time Frame: Day 42 ]Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets.
- Status of Donor Chimerism [ Time Frame: Day 100, 6 Months, 1 Year ]A state in bone marrow transplantation in which donor hematopoietic cells and host cells exist compatibly without signs of rejection.
- Incidence of Acute Graft-Versus-Host Disease (GVHD) [ Time Frame: Day 100 ]Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
- Change in Incidence of Chronic Graft-Versus-Host Disease (GVHD) [ Time Frame: 6 Months, 1 Year ]Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host.
- Incidence of Transplant Related Mortality [ Time Frame: 6 Months ]In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation.
- Incidence of Overall Survival [ Time Frame: 6 Months ]
The percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease. Also called survival rate.
Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||up to 55 Years (Child, Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Diagnosis of any disease for which a second or greater hematopoietic stem cell transplant is needed due to insufficient donor chimerism following hematopoietic recovery after previous HSCT. Determination of "insufficiency of donor chimerism" will be made by the treating transplant physician. Occasionally donor derived engraftment may be present, but sustained aplasia or failed recovery of sufficient hematopoiesis requires administration of a second graft. This intervention may be used for both situations.
Donor Availability: Patients considered for transplantation must have a sufficient graft as based on current criteria of the University of Minnesota Blood and Marrow Transplantation Program
- Transplantation using sufficiently matched related donors (such as matched siblings) or unrelated donors will be considered. Both granulocyte-colony stimulating factor (GCSF) stimulated peripheral blood grafts and bone marrow grafts will be considered, although bone marrow will be the priority.
- Cord blood grafts, both related and unrelated, are also eligible. As this protocol will use a reduced intensity regimen, this protocol will use the current recommendations of the University of Minnesota for choosing cord blood grafts. If a single cord blood unit cell dose is insufficient, double cord transplantation should be considered if sufficiently matched cord blood units are available. The priority of choosing cord blood donors is based on the current institutional recommendations.
- Exclusion of Metabolic Disorder or other Inherited Disorder Carrier Status from related donor and unrelated cord blood grafts as appropriate for primary disease.
At the discretion of the treating transplant physician, an allograft from the previous donor may be used, if available.
Age, Performance Status, Consent
- Age: 0 to 55 years
- Consent: voluntary written consent (adult or parental/guardian)
- Previous irradiation that precludes the safe administration of an additional dose of 200 cGy of total body irradiation (TBI). Radiation Oncology will evaluate all patients who have had previous radiation therapy or TBI for approval to receive an additional 200 cGy of TBI
- Pregnant or breastfeeding
- Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted
- HIV positive
- While it would be advantageous to begin therapy on this second transplant regimen > 6 months following a prior myeloablative regimen or >2 months after a reduced intensity regimen, it is recognized that there are circumstances where this may not be practical.
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): NCT01666080
|Contact: Troy Lund, M.D., Ph.D.||email@example.com|
|Contact: Paul Orchard, M.D.||firstname.lastname@example.org|
|United States, Minnesota|
|Masonic Cancer Center, University of Minnesota||Recruiting|
|Minneapolis, Minnesota, United States, 55455|
|Contact: Weston P. Miller, M.D. 612-626-2778 email@example.com|
|Principal Investigator: Weston P. Miller, M.D.|
|Principal Investigator:||Troy Lund, M.D., Ph.D.||Masonic Cancer Center, University of Minnesota|
|Responsible Party:||Masonic Cancer Center, University of Minnesota|
|Other Study ID Numbers:||
MT2012-11C ( Other Identifier: Blood and Marrow Transplantation Program )
|First Posted:||August 16, 2012 Key Record Dates|
|Last Update Posted:||November 3, 2022|
|Last Verified:||November 2022|
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