Back to resultsA Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease
Primary Purpose
Sickle Cell Disease
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Plerixafor
Busulfan
BIVV003
Sponsored by
About this trial
This is an interventional treatment trial for Sickle Cell Disease focused on measuring Intervention: Genetic: BIVV003, Drug: Busulfan, Biological: Plerixafor, Phase: Phase 1/2
Eligibility Criteria
Inclusion Criteria
- Ages 18 to 40
- 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); Six 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
Sites / Locations
- UCSF Benioff Children's HospitalRecruiting
- University of California Davis Comprehensive Cancer CenterRecruiting
- Children's Healthcare of AtlantaRecruiting
- Investigational Site Number 101Recruiting
- Karmanos Cancer InstituteRecruiting
- Children's Hospital of Philadelphia_Investigational site number 108
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
BIVV003
Arm Description
Participants will receive plerixafor as subcutaneous (SQ) administration followed by myeloablative conditioning therapy with intravenous (IV) busulfan. BIVV003 will then be administered as a 1-time IV infusion of autologous Cluster of Differentiation 34 + Hematopoietic Stem/Progenitor Cell (CD34+HSPC) transfected ex vivo with zinc finger nuclease (ZFN) messenger ribonucleic acid (mRNAs) targeting the B-cell lymphoma/leukemia 11A (BCL11A) locus.
Outcomes
Primary Outcome Measures
Percentage of Participants who are Alive at Post-transplantation Day 100
The percentage of participants who are alive at post-transplantation Day 100 will be calculated using the Kaplan-Meier estimate.
Percentage of Participants who are Alive at Post-transplantation Week 52
The percentage of participants who are alive at post-transplantation Week 52 will be calculated using the Kaplan-Meier estimate.
Percentage of Participants who are Alive at Post-transplantation Week 104
The percentage of participants who are alive at post-transplantation Week 104 will be calculated using the Kaplan-Meier estimate.
Percentage of Participants With Successful Engraftment
Successful engraftment is defined by absolute neutrophil count (ANC) greater than or equal to >=500 cells/microliter (mL) for 3 consecutive days.
Number of Participants With Adverse Events (AEs)
An AE is any untoward medical occurrence in a participant administered a pharmaceutical product that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
Number of Participants With Serious Adverse Events (SAEs)
An SAE is any untoward medical occurrence that at any dose: Results in death, in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); however, this does not include an event that, had it occurred in a more severe form, might have caused death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect or is a medically important event.
Secondary Outcome Measures
CD34 + HSPC Yield from Plerixafor Stem Cell Mobilization
Proportion of Participants with Sufficient Stem Cell Mobilization for Rescue Aliquot and BIVV003 Production
Yield of Zinc Finger Nuclease (ZFN)-edited Investigational Product
Time to Initial Neutrophil Recovery Following BIVV003 Infusion
Time to Platelet Recovery Following BIVV003 Infusion
Percentage of Participants With Maintenance of Absolute Neutrophil Count (ANC) of >=500/mcL to last Participant Visit
Percentage of participants maintaining ANC of >=500/mcL to last Participant Visit (Week 104) will be calculated.
Percentage of Participants With Maintenance of Platelet count of >=50,000/mcL to last Participant Visit
The percentage of participants attaining a post-transplant platelet count of >=50,000/mcL and maintaining this level through last Participant Visit (Week 104) will be calculated.
Change From Baseline in Peripheral Blood Fetal Hemoglobin (HbF) Levels
Change from baseline in HbF up to Week 104 will be assessed.
Change From Baseline in Peripheral Blood Percent (%)F cells
Change from baseline in %F cells up to Week 104 will be assessed.
Change From Baseline in Peripheral Blood Sickle Hemoglobin (HbS) Levels
Change from baseline in peripheral blood HbS levels up to Week 104 will be assessed.
Change From Baseline in Peripheral blood total hemoglobin (Hb) concentration
Change From baseline in peripheral blood total hemoglobin (Hb) concentration up to week 104 will be assessed.
Change From Baseline in Reticulocyte Count
Change from baseline in reticulocyte count up to Week 104 will be assessed.
Change From Baseline in Lactate Dehydrogenase (LDH) Levels
Change from baseline in LDH levels up to Week 104 will be assessed.
Change From Baseline in Haptoglobin Levels
Change from baseline in haptoglobin levels up to Week 104 will be assessed.
Change From Baseline in Serum Bilirubin Levels
Change from baseline in serum bilirubin levels up to Week 104 will be assessed.
Change From Baseline in Patient-Reported Outcomes Measurement Information System 57 (PROMIS-57) Scale Score
Quality of life (QoL) measures including fatigue will be assessed using PROMIS-57 scale. This is a 57-item questionnaire with 8 questions per domain for assessing physical and mental well-being in participants with SCD. 57 questions are summed into a total score, which is transformed into an age specific normalized t-score with 50 representing normal, and lower scores representing increasing disability.
Number of Participants With Sickle Cell Disease (SCD)-related Clinical Events
Number of participants with SCD-related clinical events (including vaso-occlusive crisis [VOC], pain episodes etc.) will be reported.
Number of SCD Related Clinical Events by Severity
Severity will be categorized by toxicity grade according to CTCAE Version 5.0. AEs not listed in the CTCAE Version 5.0 will be evaluated by: Grade 1=Mild, Grade 2=Moderate, Grade 3=Severe or medically significant but not immediately life threatening, Grade 4=Life-threatening consequences; Grade 5=Death.
Participants lymphocyte Counts
Lymphocyte counts will be measured to assess reconstitution of immune function post-BIVV003 transplantation.
Participants Immunoglobulin levels
Immunoglobulin levels will be measured to assess reconstitution of immune function post-BIVV003 transplantation.
Number of Red Blood Cell (RBC) Transfusions Received During the Post-transplantation Study Period
The number of RBC transfusions received during the Post-Transplantation study period will be reported.
Total Volume of RBC Transfused
Total volume of RBC transfused during the Post-Transplantation study period will be reported.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03653247
Brief Title
A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease
Official Title
A Phase 1/2, Open-Label, Multicenter, Single-Arm Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 6, 2019 (Actual)
Primary Completion Date
August 9, 2024 (Anticipated)
Study Completion Date
August 9, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sangamo Therapeutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This is an open label, multicenter, Phase 1/2 study in approximately eight adults with severe Sickle Cell Disease (SCD). The study will evaluate the safety, tolerability, and efficacy of autologous hematopoietic stem cell transplantation using BIVV003.
Detailed Description
Subject participation in this study will be approximately 136 weeks. Enrolled subjects will be asked to participate in a separate long-term follow-up study to monitor the safety and efficacy of BIVV003 treatment for a total of 15 years post-transplant.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease
Keywords
Intervention: Genetic: BIVV003, Drug: Busulfan, Biological: Plerixafor, Phase: Phase 1/2
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
BIVV003
Arm Type
Experimental
Arm Description
Participants will receive plerixafor as subcutaneous (SQ) administration followed by myeloablative conditioning therapy with intravenous (IV) busulfan. BIVV003 will then be administered as a 1-time IV infusion of autologous Cluster of Differentiation 34 + Hematopoietic Stem/Progenitor Cell (CD34+HSPC) transfected ex vivo with zinc finger nuclease (ZFN) messenger ribonucleic acid (mRNAs) targeting the B-cell lymphoma/leukemia 11A (BCL11A) locus.
Intervention Type
Biological
Intervention Name(s)
Plerixafor
Intervention Description
Plerixafor subcutaneous injection will be administered prior to apheresis.
Intervention Type
Drug
Intervention Name(s)
Busulfan
Intervention Description
Busulfan IV infusion will be administered as myeloablative conditioning therapy.
Intervention Type
Genetic
Intervention Name(s)
BIVV003
Other Intervention Name(s)
Autologous CD34 + hematopoietic stem cells
Intervention Description
BIVV003 will be administered as an IV infusion following myeloablative conditioning with busulfan.
Primary Outcome Measure Information:
Title
Percentage of Participants who are Alive at Post-transplantation Day 100
Description
The percentage of participants who are alive at post-transplantation Day 100 will be calculated using the Kaplan-Meier estimate.
Time Frame
Day 100
Title
Percentage of Participants who are Alive at Post-transplantation Week 52
Description
The percentage of participants who are alive at post-transplantation Week 52 will be calculated using the Kaplan-Meier estimate.
Time Frame
Week 52
Title
Percentage of Participants who are Alive at Post-transplantation Week 104
Description
The percentage of participants who are alive at post-transplantation Week 104 will be calculated using the Kaplan-Meier estimate.
Time Frame
Week 104
Title
Percentage of Participants With Successful Engraftment
Description
Successful engraftment is defined by absolute neutrophil count (ANC) greater than or equal to >=500 cells/microliter (mL) for 3 consecutive days.
Time Frame
Up to Day 42
Title
Number of Participants With Adverse Events (AEs)
Description
An AE is any untoward medical occurrence in a participant administered a pharmaceutical product that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
Time Frame
Up to Week 104
Title
Number of Participants With Serious Adverse Events (SAEs)
Description
An SAE is any untoward medical occurrence that at any dose: Results in death, in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); however, this does not include an event that, had it occurred in a more severe form, might have caused death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect or is a medically important event.
Time Frame
Up to Week 104
Secondary Outcome Measure Information:
Title
CD34 + HSPC Yield from Plerixafor Stem Cell Mobilization
Time Frame
Approximately 12 weeks
Title
Proportion of Participants with Sufficient Stem Cell Mobilization for Rescue Aliquot and BIVV003 Production
Time Frame
Approximately 12 weeks
Title
Yield of Zinc Finger Nuclease (ZFN)-edited Investigational Product
Time Frame
Approximately 12 weeks
Title
Time to Initial Neutrophil Recovery Following BIVV003 Infusion
Time Frame
Up to Week 104
Title
Time to Platelet Recovery Following BIVV003 Infusion
Time Frame
Up to Week 104
Title
Percentage of Participants With Maintenance of Absolute Neutrophil Count (ANC) of >=500/mcL to last Participant Visit
Description
Percentage of participants maintaining ANC of >=500/mcL to last Participant Visit (Week 104) will be calculated.
Time Frame
Up to Week 104
Title
Percentage of Participants With Maintenance of Platelet count of >=50,000/mcL to last Participant Visit
Description
The percentage of participants attaining a post-transplant platelet count of >=50,000/mcL and maintaining this level through last Participant Visit (Week 104) will be calculated.
Time Frame
Up to Week 104
Title
Change From Baseline in Peripheral Blood Fetal Hemoglobin (HbF) Levels
Description
Change from baseline in HbF up to Week 104 will be assessed.
Time Frame
Baseline up to Week 104
Title
Change From Baseline in Peripheral Blood Percent (%)F cells
Description
Change from baseline in %F cells up to Week 104 will be assessed.
Time Frame
Baseline up to Week 104
Title
Change From Baseline in Peripheral Blood Sickle Hemoglobin (HbS) Levels
Description
Change from baseline in peripheral blood HbS levels up to Week 104 will be assessed.
Time Frame
Baseline up to Week 104
Title
Change From Baseline in Peripheral blood total hemoglobin (Hb) concentration
Description
Change From baseline in peripheral blood total hemoglobin (Hb) concentration up to week 104 will be assessed.
Time Frame
Baseline up to Week 104
Title
Change From Baseline in Reticulocyte Count
Description
Change from baseline in reticulocyte count up to Week 104 will be assessed.
Time Frame
Baseline up to Week 104
Title
Change From Baseline in Lactate Dehydrogenase (LDH) Levels
Description
Change from baseline in LDH levels up to Week 104 will be assessed.
Time Frame
Baseline up to Week 104
Title
Change From Baseline in Haptoglobin Levels
Description
Change from baseline in haptoglobin levels up to Week 104 will be assessed.
Time Frame
Baseline up to Week 104
Title
Change From Baseline in Serum Bilirubin Levels
Description
Change from baseline in serum bilirubin levels up to Week 104 will be assessed.
Time Frame
Baseline up to Week 104
Title
Change From Baseline in Patient-Reported Outcomes Measurement Information System 57 (PROMIS-57) Scale Score
Description
Quality of life (QoL) measures including fatigue will be assessed using PROMIS-57 scale. This is a 57-item questionnaire with 8 questions per domain for assessing physical and mental well-being in participants with SCD. 57 questions are summed into a total score, which is transformed into an age specific normalized t-score with 50 representing normal, and lower scores representing increasing disability.
Time Frame
Baseline up to Week 104
Title
Number of Participants With Sickle Cell Disease (SCD)-related Clinical Events
Description
Number of participants with SCD-related clinical events (including vaso-occlusive crisis [VOC], pain episodes etc.) will be reported.
Time Frame
Baseline up to Week 104
Title
Number of SCD Related Clinical Events by Severity
Description
Severity will be categorized by toxicity grade according to CTCAE Version 5.0. AEs not listed in the CTCAE Version 5.0 will be evaluated by: Grade 1=Mild, Grade 2=Moderate, Grade 3=Severe or medically significant but not immediately life threatening, Grade 4=Life-threatening consequences; Grade 5=Death.
Time Frame
Baseline up to Week 104
Title
Participants lymphocyte Counts
Description
Lymphocyte counts will be measured to assess reconstitution of immune function post-BIVV003 transplantation.
Time Frame
At Weeks 13 and 52
Title
Participants Immunoglobulin levels
Description
Immunoglobulin levels will be measured to assess reconstitution of immune function post-BIVV003 transplantation.
Time Frame
At Weeks 13 and 52
Title
Number of Red Blood Cell (RBC) Transfusions Received During the Post-transplantation Study Period
Description
The number of RBC transfusions received during the Post-Transplantation study period will be reported.
Time Frame
Up to Week 104
Title
Total Volume of RBC Transfused
Description
Total volume of RBC transfused during the Post-Transplantation study period will be reported.
Time Frame
Up to Week 104
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
40 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria
Ages 18 to 40
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); Six 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Medical Monitor
Phone
510-307-7266
Email
clinicaltrials@sangamo.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Sangamo Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
UCSF Benioff Children's Hospital
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Marci Moriarty, R.N.
Email
marci.moriarty@ucsf.edu
Facility Name
University of California Davis Comprehensive Cancer Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kristina Curl Johnston
Phone
916-501-9393
Email
kkcurljohnston@ucdavis.edu
Facility Name
Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Judson Russell
Email
bmt@choa.org
Facility Name
Investigational Site Number 101
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rick Gustafson
Email
Richard.gustafson@nih.gov
Facility Name
Karmanos Cancer Institute
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Marie Ventimiglia
Email
ventimim@karmanos.org
Facility Name
Children's Hospital of Philadelphia_Investigational site number 108
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Withdrawn
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
34175041
Citation
Brusson M, Miccio A. Genome editing approaches to beta-hemoglobinopathies. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1.
Results Reference
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A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease
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