Sickle Cell Disease Clinical Trial
Official title:
A Phase II Study of Hematopoietic Stem Cell Therapy for Young Adults With Severe Sickle Cell Disease
This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy
and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell
disease (SCD) who have high risk features.
The primary goal of this multi-center Phase II study is to determine the safety and
feasibility of a conditioning regimen consisting of busulfan (Bu)/ fludarabine (Flu)/
anti-thymocyte globulin (ATG) in adult patients with severe SCD. A two-component design will
be used for this study. The first component will be restricted to patients who have an
HLA-identical sibling donor. Five patients will be transplanted during the first component of
the study. If no more than 2 of the first 5 patients experience unacceptable toxicity,
including death, within the first six months after transplantation, then the safety of the
regimen will be considered promising in adult SCD patients.
The second component will include patients who have a related or an unrelated human leukocyte
antigen (HLA) matched donor. Up to 15 additional patients will be transplanted in this
component of the study which will evaluate the safety and feasibility of unrelated donor
hematopoietic cell transplantation (HCT) in adults with SCD. Data related to study endpoints
for 1 year after transplantation will be collected; however, participating centers will be
encouraged to conduct long-term follow-up evaluations of patients according to standard
institutional guidelines. The purpose of this pilot safety trial is to see if this approach
is feasible and meets accrual goals lending support to the development of a subsequent full
scale investigation of HCT and comparing outcomes in a transplantation cohort to a control
cohort of adults eligible for, but unwilling or unable to receive HCT treated by supportive
therapy with a primary endpoint of five years survival for this full scale comparative trial.
This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy
and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell
disease (SCD) who have high risk features.
The primary goal of this multi-center Phase II study is to determine the safety and
feasibility of a conditioning regimen consisting of busulfan (Bu)/ fludarabine (Flu)/
anti-thymocyte globulin (ATG) in adult patients with severe SCD. A two-component design will
be used for this study. The first component will be restricted to patients who have an
HLA-identical sibling donor. Five patients will be transplanted during the first component of
the study. If no more than 2 of the first 5 patients experience unacceptable toxicity,
including death, within the first six months after transplantation, then the safety of the
regimen will be considered promising in adult SCD patients.
The second component will include patients who have a related or an unrelated human leukocyte
antigen (HLA) matched donor. Up to 15 additional patients will be transplanted in this
component of the study which will evaluate the safety and feasibility of unrelated donor
hematopoietic cell transplantation (HCT) in adults with SCD. Data related to study endpoints
for 1 year after transplantation will be collected; however, participating centers will be
encouraged to conduct long-term follow-up evaluations of patients according to standard
institutional guidelines. The purpose of this pilot safety trial is to see if this approach
is feasible and meets accrual goals lending support to the development of a subsequent full
scale investigation of HCT and comparing outcomes in a transplantation cohort to a control
cohort of adults eligible for, but unwilling or unable to receive HCT treated by supportive
therapy with a primary endpoint of five years survival for this full scale comparative trial.
The primary objective is to determine event-free survival (EFS) at 1 year after hematopoietic
cell transplantation (HCT) using bone marrow in patients with sickle cell disease. Death,
disease recurrence or graft rejection by 1 year will be considered events for this endpoint.
Secondary objectives include determining the effect of HCT on clinical and laboratory
manifestations of severe sickle cell disease and determining the incidence of other
transplant-related outcomes.
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