Sickle Cell DiseasePhase 1NCT03282656
Gene Transfer for Sickle Cell Disease
A Phase 1 study testing single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a in people with Sickle Cell Disease, run by David Williams at 2 sites in United States.
Trial summary
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. Read more about this study
- Who is it for?
- Ages 3 to 40 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesUnited States
- Study dates
- 13 Feb 2018to 13 Nov 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 3 to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of SCD with genotype HbSS, HbS/0 thalassemia, HbSD, or HbSO
- Severe symptomatic SCD, defined by the presence of one or more of the following clinical complications:
- Minimum of two episodes of acute chest syndrome (ACS) in the 2 years before study entry.
- History of three or more episodes of severe pain events requiring a visit to a medical facility and treatment with parenteral opioids in the 2 years before study entry.
- Recurrent priapism (> 2 episodes) in the 2 years before study entry.
- Red-cell alloimmunization (>2 antibodies) during long-term transfusion therapy.
Show all 19Show fewer
- Receiving, or indicated to receive (based on prior stroke or elevated transcranial Doppler (TCD) results), chronic transfusions for primary or secondary stroke prophylaxis.
- Age 3 years to 40 years .
- Failure of hydroxyurea therapy due to lack of clinical improvement or inability to tolerate due to side effects (e.g., myelosuppression, gastrointestinal symptoms, or hepatic enzyme elevations). Clinical criteria must be met despite taking hydroxyurea for greater than or equal to 6 months, unless contraindicated or not tolerated. Patients taking hydroxyurea who still meet all inclusion criteria are eligible for the trial.
- No HLA-genotypically identical related bone marrow donor available
- Parental/guardian/patient signed informed consent
- Willingness to return for follow-up for 15 years
- White blood cell (WBC) count within the range of 3.0 - 20.0 x 109 /L Hemoglobin within the range of 5 - 11 g/dL Platelet count within the range of 100 - 600 x 109 /L PT and PTT within normal limits, unless prolonged due to anticoagulation requirement.
- Adequate organ function and performance status:
- Performance status ≥70% (Lansky play for age <16 years, Karnofsky for age ≥16 years)
- Left ventricular ejection fraction >40% or shortening fraction >25%
- Direct bilirubin ≤ 2.0 mg/dL
- Serum creatinine </= 1.5 times the upper limit of normal for age, and creatinine clearance or GFR >/= 70 mL/min/1.73 m2.
- For ages > 7 years, DLCO (corrected for hemoglobin), FEV1, FVC >50% of predicted; if age < 7 years, then oxygen saturation >92% on room air.
Who cannot join
- Contraindication to bone marrow harvest, or to administration of conditioning medication (busulfan).
- Subjects who have undergone allogeneic transplant previously.
- Known positive HIV serology or HIV nucleic acid testing, or positive serology for HCV, HBV, or HTLV.
- Uncontrolled infection.
- Active malignancy.
- Known myelodysplasia of the bone marrow or abnormal bone marrow cytogenetics.
Show all 10Show fewer
- Receipt of an investigational study drug or procedure within 90 days of study enrollment.
- Pregnancy, or breastfeeding in a postpartum female, or absence of adequate contraception for fertile subjects. Females of child-bearing potential must agree to use a medically acceptable method of birth control such as oral contraceptive, intrauterine device, barrier and spermicide, or contraceptive implant/injection from Screening through at least 6 months after drug product infusion. Male subjects must agree to use effective contraception (including condoms) from Screening through at least 6 months after drug product infusion.
- Acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on prior biopsy.
- An assessment by the Investigators that the subject will not comply with the study procedures outlined in the study protocol
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Boston Children's Hospital
Boston, Massachusetts, 02115
UCLA - Mattel Children's Hospital
Los Angeles, California, 90095
About this study
Official title: Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Sickle Cell Disease
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace the patient's disease gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of GVHD, reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to introduce the gene into the patient's own blood stem cells is to engineer and use a modified version of a virus (called a 'vector') that efficiently inserts the "correcting" genetic material into the cells. The vector is a specialized biological medicine that has been formulated for use in human beings.
The investigators have recently discovered a gene that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition. In summary, the advantages of a gene therapy approach include: 1) it can be used even if the patient does not have a matched donor available; 2) it may allow a reduction in the amount of chemotherapy required to prepare the patient for the transplant; and 3) it will avoid the strong medicines often required to prevent and treat GVHD and rejection. The goal is to test whether this approach is safe, and whether using gene therapy to change the expression of this particular gene will lead to increased fetal hemoglobin production in people with sickle cell disease.
Read the detailed description
This is an open-label, non-randomized, single center, pilot and feasibility, single arm cohort study involving a single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a. Accrual will be a maximum of 7 evaluable patients with SCD. The study will have three strata:
- Stratum 1: ages ≥18-40
- Stratum 2: ages ≥12-<18
- Stratum 3: ages ≥3-<12
To determine the feasibility and safety of administering a lentiviral gene transfer vector encoding a small hairpin (sh) RNA targeting the γ-globin gene repressor, BCL11A, in patients with severe SCD.
Patients will undergo standard work-up for autologous bone marrow transplantation according to institutional guidelines and then undergo two bone marrow harvests at a minimum of 4 weeks apart that will be used for a back-up marrow (minimum of 2 x 106 CD34+ cells/kg) and for a harvest of autologous bone marrow for gene transfer.
Patients will receive blood transfusions for a period of 3 months prior to the planned date of product infusion, with a goal of achieving a HbS level ≤ 30% by the time of gene transfer. If the subject is already on a chronic transfusion regimen as part of baseline disease management, the regimen may be continued. The timing of transfusions will be coordinated to occur within 7 days prior to any procedures requiring anesthesia such as bone marrow harvest.
Hematopoietic cells will be collected from the patient in advance of the treatment, to serve as a salvage procedure ("back-up graft"), should there be no hematopoietic recovery observed following the injection of genetically-manipulated cells, or should manipulated cells fail to meet release criteria. Bone marrow (up to 20 mL/kg) will be harvested from the patient under general anesthesia from the posterior iliac crests on both sides by multiple punctures at a minimum of 4 weeks prior to gene therapy. A portion of the bone marrow containing at least 2 x 106 CD34+ cells/kg will be frozen and stored unmanipulated according to standard clinical procedures for autologous bone marrow collection to constitute the back-up graft. If the number of CD34+ cells that exceeds 2 x 106 cells/kg is greater than or equal to 1 x 106 cells/kg, these excess cells would be processed for transduction, transduced, and then frozen. Transduction will be carried out on the selected CD34+ cells and transduced cells will be cryopreserved. If cells from the first harvest were transduced and frozen, these gene-modified cells will be thawed and prepared for infusion in parallel with the product from the subsequent harvest(s). In this case two separate products would be infused. All cell manipulation procedures and release testing will be performed in the transduction facility in accordance with Good Manufacturing Practice (GMP) following process specific standard operating procedures. Final Drug Product will be accompanied by a Certificate of Analysis, documenting that all release testing is complete and within specification. Subjects will receive myeloablative conditioning with Busulfan administered on days -5 to -2, prior to infusion of transduced cells. Cells will be infused intravenously over 30-45 minutes after standard prehydration and premedication according to Boston Children's Hospital Hematopoietic Stem Cell Transplantation Unit standard guidelines. This standard requires that the patient be on continuous cardiac, respiratory and oxygen saturation monitor throughout the infusion and for 30 minutes afterwards. Vital signs will be measured and recorded pre-transfusion, 15 minutes into transfusion, every hour for duration of infusion, and end of transfusion. The RN will stay with the patient for the first 5 minutes of the transfusion. If two transduction products are administered, the second transduced product will be administered without delay after the first.
- Conditions
- Sickle Cell Disease
- Being tested
- single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a
- Keywords
- Gene therapy, lentivirus vector, BCL11A, Fetal Hemoglobin
- Medical subject headings
- Anemia, Sickle Cell, beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11aBiological
single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a
| Study group | Role | What they receive |
|---|---|---|
| Treatment arm | Experimental | open-label, non-randomized, single center, pilot and feasibility, single arm cohort study of a single infusion of autologous bone marrow derived CD34+ HSC cells transduced with lentiviral vector containing a short-hairpin RNA targeting BC11A. Receives: Biological: single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rescue of hematopoiesis after conditioning
Rescue of hematopoiesis after conditioning (defined by absolute neutrophil count (ANC) greater than or equal to 0.5 x 109 /L for three consecutive days), achieved within 7 weeks following infusion (i.e., "primary engraftment").
Measured: within 7 weeks following infusion of gene transfer product
Secondary outcomes (1)
Expression of transgene
- Presence of the transgene (vector copy number) in the following samples: i) Peripheral blood cells [measured in whole blood, mononuclear cells, and the following sorted populations: CD3+, CD15+, CD19+, and CD56+] at 6 weeks, and then every 6 months until 2 years after gene transfer.
Measured: at 6 weeks post gene transfer then every 6 months until 2 years after gene transfer
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2017
- Study start13 Feb 2018
- Primary completion13 May 2026 (estimated)
- Study completion13 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label, non-randomized, single center, pilot and feasibility, single arm cohort study
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- David Williams
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, David Williams, Boston Children's Hospital
- Investigators
- Erica Esrick, MD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03282656
- Sponsor's protocol ID
- P00026188
- Status last verified
- Jan 2026
- First submitted
- 22 Aug 2017
- Last updated
- 9 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Esrick EB, Lehmann L, Federico A, Vincon H, Liu B, Daley H, Dansereau C, De Oliveira S, Everett JK, Kao PC, Moore TB, Morris E, Trebeden-Negre H, Shaw KL, Roach GD, Ritz J, Roche AM, Silva O, Grant PE, Bushman FD, Kohn DB, Jain A, London WB, Justus DG, Armant M, Manis JP, Williams DA. Long-term stability of posttranscriptional genetic silencing of BCL11A using a shmiR vector in Sickle Cell Disease. Blood. 2026 Jun 2:blood.2026033871. doi: 10.1182/blood.2026033871. Online ahead of print.
PubMed 42233421Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, Daley H, MacKinnon B, Morris E, Federico A, Abriss D, Boardman K, Khelladi R, Shaw K, Negre H, Negre O, Nikiforow S, Ritz J, Pai SY, London WB, Dansereau C, Heeney MM, Armant M, Manis JP, Williams DA. Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. N Engl J Med. 2021 Jan 21;384(3):205-215. doi: 10.1056/NEJMoa2029392. Epub 2020 Dec 5.
PubMed 33283990
Study at a glance
From the registry record, last updated by the sponsor on 9 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT03282656
- Acronym / sponsor ID
- P00026188
- Conditions
- Sickle Cell Disease
- Keywords
- Gene therapy, lentivirus vector, BCL11A, Fetal Hemoglobin
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 10 participants (actual)
- Lead sponsor
- David WilliamsUniversity, hospital or other
- Study start
- 13 Feb 2018 (actual)
- Primary completion
- 13 May 2026 (estimated)
- Study completion
- 13 Nov 2026 (estimated)
- First posted
- 14 Sep 2017 (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.
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace the patient's disease gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of GVHD, reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to introduce the gene into the patient's own blood stem cells is to engineer and use a modified version of a virus (called a 'vector') that efficiently inserts the "correcting" genetic material into the cells. The vector is a specialized biological medicine that has been formulated for use in human beings.
The investigators have recently discovered a gene that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition. In summary, the advantages of a gene therapy approach include: 1) it can be used even if the patient does not have a matched donor available; 2) it may allow a reduction in the amount of chemotherapy required to prepare the patient for the transplant; and 3) it will avoid the strong medicines often required to prevent and treat GVHD and rejection. The goal is to test whether this approach is safe, and whether using gene therapy to change the expression of this particular gene will lead to increased fetal hemoglobin production in people with sickle cell disease.
Detailed description
This is an open-label, non-randomized, single center, pilot and feasibility, single arm cohort study involving a single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a. Accrual will be a maximum of 7 evaluable patients with SCD. The study will have three strata:
- Stratum 1: ages ≥18-40
- Stratum 2: ages ≥12-<18
- Stratum 3: ages ≥3-<12
To determine the feasibility and safety of administering a lentiviral gene transfer vector encoding a small hairpin (sh) RNA targeting the γ-globin gene repressor, BCL11A, in patients with severe SCD.
Patients will undergo standard work-up for autologous bone marrow transplantation according to institutional guidelines and then undergo two bone marrow harvests at a minimum of 4 weeks apart that will be used for a back-up marrow (minimum of 2 x 106 CD34+ cells/kg) and for a harvest of autologous bone marrow for gene transfer.
Patients will receive blood transfusions for a period of 3 months prior to the planned date of product infusion, with a goal of achieving a HbS level ≤ 30% by the time of gene transfer. If the subject is already on a chronic transfusion regimen as part of baseline disease management, the regimen may be continued. The timing of transfusions will be coordinated to occur within 7 days prior to any procedures requiring anesthesia such as bone marrow harvest.
Hematopoietic cells will be collected from the patient in advance of the treatment, to serve as a salvage procedure ("back-up graft"), should there be no hematopoietic recovery observed following the injection of genetically-manipulated cells, or should manipulated cells fail to meet release criteria. Bone marrow (up to 20 mL/kg) will be harvested from the patient under general anesthesia from the posterior iliac crests on both sides by multiple punctures at a minimum of 4 weeks prior to gene therapy. A portion of the bone marrow containing at least 2 x 106 CD34+ cells/kg will be frozen and stored unmanipulated according to standard clinical procedures for autologous bone marrow collection to constitute the back-up graft. If the number of CD34+ cells that exceeds 2 x 106 cells/kg is greater than or equal to 1 x 106 cells/kg, these excess cells would be processed for transduction, transduced, and then frozen. Transduction will be carried out on the selected CD34+ cells and transduced cells will be cryopreserved. If cells from the first harvest were transduced and frozen, these gene-modified cells will be thawed and prepared for infusion in parallel with the product from the subsequent harvest(s). In this case two separate products would be infused. All cell manipulation procedures and release testing will be performed in the transduction facility in accordance with Good Manufacturing Practice (GMP) following process specific standard operating procedures. Final Drug Product will be accompanied by a Certificate of Analysis, documenting that all release testing is complete and within specification. Subjects will receive myeloablative conditioning with Busulfan administered on days -5 to -2, prior to infusion of transduced cells. Cells will be infused intravenously over 30-45 minutes after standard prehydration and premedication according to Boston Children's Hospital Hematopoietic Stem Cell Transplantation Unit standard guidelines. This standard requires that the patient be on continuous cardiac, respiratory and oxygen saturation monitor throughout the infusion and for 30 minutes afterwards. Vital signs will be measured and recorded pre-transfusion, 15 minutes into transfusion, every hour for duration of infusion, and end of transfusion. The RN will stay with the patient for the first 5 minutes of the transfusion. If two transduction products are administered, the second transduced product will be administered without delay after the first.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label, non-randomized, single center, pilot and feasibility, single arm cohort study
| Arm | Type | Receives |
|---|---|---|
| Treatment arm | Experimental | Biological: single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11aopen-label, non-randomized, single center, pilot and feasibility, single arm cohort study of a single infusion of autologous bone marrow derived CD34+ HSC cells transduced with lentiviral vector containing a short-hairpin RNA targeting BC11A. |
Who may qualify
- Ages
- 3 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (19 listed)
- Diagnosis of SCD with genotype HbSS, HbS/0 thalassemia, HbSD, or HbSO
- Severe symptomatic SCD, defined by the presence of one or more of the following clinical complications:
- Age 3 years to 40 years .
- Failure of hydroxyurea therapy due to lack of clinical improvement or inability to tolerate due to side effects (e.g., myelosuppression, gastrointestinal symptoms, or hepatic enzyme elevations). Clinical criteria must be met despite taking hydroxyurea for greater than or equal to 6 months, unless contraindicated or not tolerated. Patients taking hydroxyurea who still meet all inclusion criteria are eligible for the trial.
- No HLA-genotypically identical related bone marrow donor available
Key exclusion criteria (10 listed)
- Contraindication to bone marrow harvest, or to administration of conditioning medication (busulfan).
- Subjects who have undergone allogeneic transplant previously.
- Known positive HIV serology or HIV nucleic acid testing, or positive serology for HCV, HBV, or HTLV.
- Uncontrolled infection.
- Active malignancy.
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 |
|---|---|---|
| single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11asingle infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a | Biological | Treatment arm |
Primary outcome
Rescue of hematopoiesis after conditioning
Time frame: within 7 weeks following infusion of gene transfer product
Rescue of hematopoiesis after conditioning (defined by absolute neutrophil count (ANC) greater than or equal to 0.5 x 109 /L for three consecutive days), achieved within 7 weeks following infusion (i.e., "primary engraftment").
1 secondary outcome
Expression of transgene
Time frame: at 6 weeks post gene transfer then every 6 months until 2 years after gene transfer
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jan 2026.
- Derived from this study: PubMed 42233421Esrick EB, Lehmann L, Federico A, Vincon H, Liu B, Daley H, Dansereau C, De Oliveira S, Everett JK, Kao PC, Moore TB, Morris E, Trebeden-Negre H, Shaw KL, Roach GD, Ritz J, Roche AM, Silva O, Grant PE, Bushman FD, Kohn DB, Jain A, London WB, Justus DG, Armant M, Manis JP, Williams DA. Long-term stability of posttranscriptional genetic silencing of BCL11A using a shmiR vector in Sickle Cell Disease. Blood. 2026 Jun 2:blood.2026033871. doi: 10.1182/blood.2026033871. Online ahead of print.
- Derived from this study: PubMed 33283990Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, Daley H, MacKinnon B, Morris E, Federico A, Abriss D, Boardman K, Khelladi R, Shaw K, Negre H, Negre O, Nikiforow S, Ritz J, Pai SY, London WB, Dansereau C, Heeney MM, Armant M, Manis JP, Williams DA. Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. N Engl J Med. 2021 Jan 21;384(3):205-215. doi: 10.1056/NEJMoa2029392. Epub 2020 Dec 5.
- 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.
Erica Esrick, MD principal investigator
Boston Children's Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a in people with Sickle Cell Disease, run by David Williams at 2 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 19 inclusion and 10 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: under way, not looking for participants, as of the sponsor's last update on 9 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Rescue of hematopoiesis after conditioning (time frame: within 7 weeks following infusion of gene transfer product). 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 NCT03282656, which also lists 2 linked publications.
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 9 Jan 2026.