|Age:||18 to 35 years old|
|Keywords:||sickle cell disease, sickle cell, sickle cell anemia, stem cells, stem cell therapy|
|Type:||Biological study, Phase [/1/,/ /2/]|
The study team will make sure you qualify to take part in the study. Qualified participants will receive detailed information about the study. This may include a list of study-related tests and procedures.
Inclusion Criteria - Ages 18 to 35 - Confirmation of sickle cell disease (SCD) diagnosis (HbSS or HbS[beta]0 genotype) - Severe SCD, defined as having 1 or more of the following manifestations: Clinically significant neurologic event (example [e.g.], stroke) or any neurological deficit lasting more than 24 hours; History of 2 or more episodes or Acute Chest Syndrome (ACS) in 2 years prior to informed consent (despite adequate supportive therapies such as asthma therapy); Three or more pain crises per year in 2 years prior to informed consent (requiring intravenous [IV] pain management in the outpatient or inpatient hospital setting); History of 2 or more cases or priapism with participant seeking medical care in the 2-years prior to informed consent; Regular RBC transfusion therapy in the year prior to informed consent (having received 8 or more transfusions to prevent vaso-occlusive clinical complications); and Echocardiographic finding of tricuspid valve regurgitant jet (TRJ) velocity of greater than or equal to 2.5 meter per second (m/s) - Clinically stable to undergo stem cell mobilization and myeloablative hematopoietic stem cell transplantation (HSCT) - Adequate physiological function, defined as the following: Karnofsky/Lansky Performance of greater than or equal to 60; Acceptable cardiac function as defined in protocol; Acceptable pulmonary function as defined in protocol; Acceptable renal function as defined in protocol; and Acceptable hepatic function as defined in protocol - Ability to understand purpose and risks of study, provide Informed Consent Form (ICF) and authorization to use protected health information - Completion of age-appropriate cancer screening - Willingness to use double-barrier method of contraception through entire study period (for participants of childbearing potential) - Willingness to receive blood transfusions - Willingness to discontinue hydroxyurea (HU) at least 30 days prior to stem cell mobilization through Day 100 post-transplantation