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New treatment option for Sickle Cell Disease
Official title Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease
ClinicalTrials.gov ID: NCT06399107
What this study is testing
What is Drug Product is administered by IV infusion following myeloablative conditioning with busulfan?
Drug Product is administered by IV infusion following myeloablative conditioning with busulfan is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for sickle cell disease.
Also referred to as BAH243.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is an open-label, non-randomized, single-dose Phase 1/2 trial involving around 85 adult and pediatric participants aged between 2 and 50 years with sickle cell disease (SCD). It aims to assess the effectiveness of hematopoietic stem cell transplantation (HSCT) using BAH243 for SCD.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 90
You may be able to join if
- Have a diagnosis of SCD, with either βS/βS, βS/β0, or βS/β+ genotype.
- Be ≥2 and ≤50 years of age at time of consent.
- Weigh a minimum of 6 kg.
- Have a Karnofsky performance status of ≥60 (≥16 years of age) or a Lansky performance status of ≥60 (\<16 years of age).
- Be treated and followed for at least the past 24 months prior to Informed Consent in medical center(s) that maintained detailed records on sickle...
You likely can't join if
- people for whom allogeneic hematopoietic stem cell transplantation (allo-HSCT) is medically appropriate per PI judgment and a willing, human...
- Severe cerebral vasculopathy, defined by any history of overt ischemic or hemorrhagic stroke, a history of abnormal transcranial Doppler (TCD) or TCD...
- Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 or HIV-2), hepatitis B, hepatitis C, human T-lymphotropic virus-1 (HTLV-1)...
- Clinically significant, active bacterial, viral, fungal, or parasitic infection
- Advanced liver disease, such as
- clear evidence of liver cirrhosis, active hepatitis or significant fibrosis (based on MRI or liver biopsy)
See the full eligibility criteria
- Have a diagnosis of SCD, with either βS/βS, βS/β0, or βS/β+ genotype.
- Be ≥2 and ≤50 years of age at time of consent.
- Weigh a minimum of 6 kg.
- Have a Karnofsky performance status of ≥60 (≥16 years of age) or a Lansky performance status of ≥60 (\<16 years of age).
- Be treated and followed for at least the past 24 months prior to Informed Consent in medical center(s) that maintained detailed records on sickle cell disease history.
- In the setting of appropriate supportive care measures (e.g., pain management plan), have experienced at least 4 protocol-defined VOEs in the 24 months prior to informed consent.
- Have either experienced HU failure at any point in the past or must have intolerance to HU (intolerance is defined as the patient being unable to continue to take HU per PI judgment).
- Female and male people of childbearing potential agree to use 1 method of highly effective contraception from Screening to at least 6 months after drug product infusion.
- Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s).
- people for whom allogeneic hematopoietic stem cell transplantation (allo-HSCT) is medically appropriate per PI judgment and a willing, human leukocyte antigen (HLA)-matched related hematopoietic stem cell donor is...
- Severe cerebral vasculopathy, defined by any history of overt ischemic or hemorrhagic stroke, a history of abnormal transcranial Doppler (TCD) or TCD imaging (TCDI) for people ≤ 16 years of age (e.g. TCD velocity \>200...
- Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 or HIV-2), hepatitis B, hepatitis C, human T-lymphotropic virus-1 (HTLV-1), active syphilis.
- Clinically significant, active bacterial, viral, fungal, or parasitic infection
- Advanced liver disease, such as
- clear evidence of liver cirrhosis, active hepatitis or significant fibrosis (based on MRI or liver biopsy)
- liver iron concentration ≥15 mg/g unless liver biopsy shows no evidence of cirrhosis, active hepatitis or significant fibrosis
- Inadequate bone marrow function, as defined by an absolute neutrophil count of \<1×10\^9/L (\<0.5×10\^9/L for people on hydroxyurea treatment) or a platelet count \<100×10\^9/L.
- Any contraindications to the use of plerixafor during the mobilization of hematopoietic stem cells and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative...
- Patients needing therapeutic anticoagulation treatment during the period of conditioning through platelet engraftment
- Unable to receive pRBC transfusion.
- Prior receipt of an allogeneic transplant.
- Prior receipt of gene therapy.
- Any prior or current malignancy or immunodeficiency disorder, except previously treated, non-life threatening, cured tumors such as squamous cell carcinoma of the skin.
- Immediate family member with a known or suspected Familial Cancer Syndrome.
- Female subject is breastfeeding, pregnant or will attempt to become pregnant from Screening to at least 6 months after drug product infusion.
- Any other condition that would render the subject ineligible for HSCT.
- Participation in another clinical study with an investigational drug within 30 days of screening.
- Presence of a chromosomal abnormality or genetic mutation that may put the subject at an increased risk of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) per Investigator's judgment.
- Presence of genetic mutations that result in the inactivation of 2 or more α-globin genes
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, Beijing Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 90 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, Beijing Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.