Sickle Cell DiseasePhase 1/2NCT03653247
A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease
A Phase 1/2 study testing Plerixafor, Busulfan and BIVV003 in people with Sickle Cell Disease, run by Sangamo Therapeutics at 5 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 40 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Plerixafor, Busulfan and BIVV003
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 5 sitesUnited States
- Study dates
- 6 Mar 2019to 17 Jul 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 40 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
Show all 22Show fewer
- 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
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Investigational Site Number 101
Bethesda, Maryland, 20892
Karmanos Cancer Institute
Detroit, Michigan, 48201
UCSF Benioff Children's Hospital
Oakland, California, 94609
University of California Davis Comprehensive Cancer Center
Sacramento, California, 95817
About this study
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
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.
Read the 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.
- Conditions
- Sickle Cell Disease
- Being tested
- Plerixafor, Busulfan, BIVV003
- Keywords
- Intervention: Genetic: BIVV003, Drug: Busulfan, Biological: Plerixafor, Phase: Phase 1/2
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlerixaforBiological
Plerixafor subcutaneous injection will be administered prior to apheresis.
BusulfanDrug
Busulfan IV infusion will be administered as myeloablative conditioning therapy.
BIVV003Genetic
Also called Autologous CD34 + hematopoietic stem cells
BIVV003 will be administered as an IV infusion following myeloablative conditioning with busulfan.
| Study group | Role | What they receive |
|---|---|---|
| BIVV003 | Experimental | 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. Receives: Biological: Plerixafor; Drug: Busulfan; Genetic: BIVV003 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Day 100
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.
Measured: Week 52
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.
Measured: Week 104
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.
Measured: Up to Day 42
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.
Measured: Up to Week 104
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.
Measured: Up to Week 104
Secondary outcomes (22)
CD34 + HSPC Yield from Plerixafor Stem Cell Mobilization
Measured: Approximately 12 weeks
Proportion of Participants with Sufficient Stem Cell Mobilization for Rescue Aliquot and BIVV003 Production
Measured: Approximately 12 weeks
Yield of Zinc Finger Nuclease (ZFN)-edited Investigational Product
Measured: Approximately 12 weeks
Time to Initial Neutrophil Recovery Following BIVV003 Infusion
Measured: Up to Week 104
Time to Platelet Recovery Following BIVV003 Infusion
Measured: Up to Week 104
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.
Measured: Up to Week 104
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.
Measured: Up to Week 104
Change From Baseline in Peripheral Blood Fetal Hemoglobin (HbF) Levels
Change from baseline in HbF up to Week 104 will be assessed.
Measured: Baseline up to Week 104
Change From Baseline in Peripheral Blood Percent (%)F cells
Change from baseline in %F cells up to Week 104 will be assessed.
Measured: Baseline up to Week 104
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.
Measured: Baseline up to Week 104
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.
Measured: Baseline up to Week 104
Change From Baseline in Reticulocyte Count
Change from baseline in reticulocyte count up to Week 104 will be assessed.
Measured: Baseline up to Week 104
Change From Baseline in Lactate Dehydrogenase (LDH) Levels
Change from baseline in LDH levels up to Week 104 will be assessed.
Measured: Baseline up to Week 104
Change From Baseline in Haptoglobin Levels
Change from baseline in haptoglobin levels up to Week 104 will be assessed.
Measured: Baseline up to Week 104
Change From Baseline in Serum Bilirubin Levels
Change from baseline in serum bilirubin levels up to Week 104 will be assessed.
Measured: Baseline up to Week 104
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.
Measured: Baseline up to Week 104
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.
Measured: Baseline up to Week 104
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.
Measured: Baseline up to Week 104
Participants lymphocyte Counts
Lymphocyte counts will be measured to assess reconstitution of immune function post-BIVV003 transplantation.
Measured: At Weeks 13 and 52
Participants Immunoglobulin levels
Immunoglobulin levels will be measured to assess reconstitution of immune function post-BIVV003 transplantation.
Measured: At Weeks 13 and 52
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.
Measured: Up to Week 104
Total Volume of RBC Transfused
Total volume of RBC transfused during the Post-Transplantation study period will be reported.
Measured: Up to Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2018
- Study start6 Mar 2019
- Primary completion17 Jul 2025
- Study completion17 Jul 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sangamo Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Sangamo Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03653247
- Sponsor's protocol ID
- 003SCD101
- Status last verified
- Sep 2025
- First submitted
- 10 Aug 2018
- Last updated
- 12 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 34175041
Study at a glance
From the registry record, last updated by the sponsor on 12 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03653247
- Acronym / sponsor ID
- 003SCD101
- Conditions
- Sickle Cell Disease
- Keywords
- Intervention: Genetic: BIVV003, Drug: Busulfan, Biological: Plerixafor, Phase: Phase 1/2
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Sangamo TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 6 Mar 2019 (actual)
- Primary completion
- 17 Jul 2025 (actual)
- Study completion
- 17 Jul 2025 (actual)
- First posted
- 31 Aug 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BIVV003 | Experimental | Biological: Plerixafor, Drug: Busulfan, Genetic: BIVV003Participants 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. |
Who may qualify
- Ages
- 18 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- 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
Key exclusion criteria (22 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PlerixaforPlerixafor subcutaneous injection will be administered prior to apheresis. | Biological | BIVV003 |
| BusulfanBusulfan IV infusion will be administered as myeloablative conditioning therapy. | Drug | BIVV003 |
| BIVV003Also: Autologous CD34 + hematopoietic stem cellsBIVV003 will be administered as an IV infusion following myeloablative conditioning with busulfan. | Genetic | BIVV003 |
Primary outcome
Percentage of Participants who are Alive at Post-transplantation Day 100
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Up to Day 42
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)
Time frame: Up to Week 104
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)
Time frame: Up to Week 104
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.
22 secondary outcomes
CD34 + HSPC Yield from Plerixafor Stem Cell Mobilization
Time frame: Approximately 12 weeks
Proportion of Participants with Sufficient Stem Cell Mobilization for Rescue Aliquot and BIVV003 Production
Time frame: Approximately 12 weeks
Yield of Zinc Finger Nuclease (ZFN)-edited Investigational Product
Time frame: Approximately 12 weeks
Time to Initial Neutrophil Recovery Following BIVV003 Infusion
Time frame: Up to Week 104
Time to Platelet Recovery Following BIVV003 Infusion
Time frame: Up to Week 104
Percentage of Participants With Maintenance of Absolute Neutrophil Count (ANC) of >=500/mcL to last Participant Visit
Time frame: Up to Week 104
Percentage of Participants With Maintenance of Platelet count of >=50,000/mcL to last Participant Visit
Time frame: Up to Week 104
Change From Baseline in Peripheral Blood Fetal Hemoglobin (HbF) Levels
Time frame: Baseline up to Week 104
Change From Baseline in Peripheral Blood Percent (%)F cells
Time frame: Baseline up to Week 104
Change From Baseline in Peripheral Blood Sickle Hemoglobin (HbS) Levels
Time frame: Baseline up to Week 104
Change From Baseline in Peripheral blood total hemoglobin (Hb) concentration
Time frame: Baseline up to Week 104
Change From Baseline in Reticulocyte Count
Time frame: Baseline up to Week 104
Change From Baseline in Lactate Dehydrogenase (LDH) Levels
Time frame: Baseline up to Week 104
Change From Baseline in Haptoglobin Levels
Time frame: Baseline up to Week 104
Change From Baseline in Serum Bilirubin Levels
Time frame: Baseline up to Week 104
Change From Baseline in Patient-Reported Outcomes Measurement Information System 57 (PROMIS-57) Scale Score
Time frame: Baseline up to Week 104
Number of Participants With Sickle Cell Disease (SCD)-related Clinical Events
Time frame: Baseline up to Week 104
Number of SCD Related Clinical Events by Severity
Time frame: Baseline up to Week 104
Participants lymphocyte Counts
Time frame: At Weeks 13 and 52
Participants Immunoglobulin levels
Time frame: At Weeks 13 and 52
Number of Red Blood Cell (RBC) Transfusions Received During the Post-transplantation Study Period
Time frame: Up to Week 104
Total Volume of RBC Transfused
Time frame: Up to Week 104
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Sep 2025.
- Derived from this study: PubMed 34175041Brusson 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Sangamo Therapeutics
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Plerixafor, Busulfan and BIVV003 in people with Sickle Cell Disease, run by Sangamo Therapeutics at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants who are Alive at Post-transplantation Day 100 (time frame: Day 100). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03653247, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Sep 2025.