A Study of the Safety and Effectiveness of Experimental BIVV003 for Stem Cell Transplant for Severe Sickle Cell Disease

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Age: 18 to 35 years old
Healthy Volunteers: No
Keywords: sickle cell disease, sickle cell, sickle cell anemia, stem cells, stem cell therapy
Type: Biological study, Phase [/1/,/ /2/]
2 Participants
This research study is being done to see if the investigational product being studied is well-tolerated. We hope to understand the safety of the product, and to see if it improves the health of patients with sickle cell disease.

Sickle cell disease is an inherited disorder. Red blood cells become sickle shaped (shaped like a crescent) and rigid. This leads to a variety of serious complications throughout the body. It is possible to replace the abnormal red blood cells in sickle cell patients with normal red blood cells. This is done by a bone marrow transplant from a matched donor.

The investigational product to be tested in the present study is known by its code name BIVV003. BIVV003 is made from stem cells that will be collected from your own blood. Stem cells are the primitive cells that make all your blood cells. The stem cells contain the genetic code for making hemoglobin. A portion of that code decides which kind of hemoglobin is made: adult, which is the sickle hemoglobin in your case, or fetal (baby hemoglobin). Bioverativ Therapeutics Inc. has found a way to change this code to make more of the fetal hemoglobin and less of the sickle hemoglobin.
This study requires

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.

Who can participate

Inclusion criteria:

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

Exclusion criteria:

  • Previous receipt of an autologous or allogeneic HSCT or solid organ transplantation
  • Previous treatment with gene therapy
  • Current enrollment in an interventional study or having received an investigational drug within 30 days of study enrollment
  • Pregnant or breastfeeding female
  • Female or male who plans to become pregnant or impregnate a partner, respectively, during the anticipated study period
  • Contraindication to plerixafor, apheresis, or busulfan
  • Treatment with prohibited medication in previous 30 days
  • Known allergy or hypersensitivity to plerixafor, busulfan, or investigational product excipients
  • History of active malignancy within past 5 years, any history of hematologic malignancy, or a family history of a cancer predisposition syndrome (without negative result of candidate)
  • Current diagnosis of uncontrolled seizures
  • History of significant bleeding disorder
  • Clinically significant infection
  • Any major organ dysfunction involving brain, kidney, liver, lung, or heart (e.g., congestive heart failure, pulmonary hypertension)
  • Corrected QT interval of more than 500 millisecond (ms) based on screening electrocardiogram (ECG)
  • Positive for human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV)
  • Known to have a gamma-globin variant associated with altered oxygen affinity
  • Hereditary persistence of fetal hemoglobin (HPFH) or HbF concentration of more than or equal to 20 percent (%) at screening
  • Absolute Neutrophil Count (ANC) of less than or equal to 1,000 per microliter
  • Platelet count of less than 100,000 per microliter
  • History of platelet alloimmunization (precluding ability to provide transfusion support)
  • Extensive Red Blood Cell (RBC) alloimmunization (precluding ability to provide transfusion support)
  • Judged unsuitable for participation by investigator and/or sponsor
Benefits and risks of participating

We cannot promise any benefits to you as a result of taking part in this research study.


The study doctor will discuss the risks associated with taking part in this research study.
You will not be paid to take part in this research study.
Study duration and period
We expect that you will be in this research study for up to 2.5 years (up to approximately 120 weeks).
Recruitment period
From May 7, 2019
UC Davis Comprehensive Cancer Center
4501 X Street
Sacramento, CA 95817
Trisha Yassear
Research Topic
  • Sickle Cell Disease

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