Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age2-90
SponsorEssen Biotech
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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).
Disqualifiers
Subjects 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 available.
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 subjects ≤ 16 years of age (e.g. TCD velocity >200 cm/sec) requiring ongoing chronic transfusions, a Screening TCD or TCDI velocity > 200 cm/sec (central read), a Screening MRA showing > 50% stenosis or occlusion in the circle of Willis (central read), or a Screening MRA showing the presence of Moyamoya (central read).
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
Trial design
Design model
Single group
Treatments tested in this trial
Drug Product is administered by IV infusion following myeloablative conditioning with busulfan
GeneticAn autologous CD34+ cell-enriched population from patients with sickle cell disease (SCD), which includes hematopoietic stem cells (HSCs) that have been transduced with the BAH243 lentiviral vector (LVV) carrying the βA-T87Q-globin gene, is preserved in a cryopreservation solution.
Treatment groups
85 Participants
are divided into 1 treatment groupGroup A: Experimental: Treatment (CD19/BCMA-CAR T cells, chemotherapy)Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
VOE-CR
Proportion of subjects achieving complete resolution of VOEs between 6 months and 18 months after drug product infusion
Time frame
6-18 months post-transplantSecondary outcomes
1
sVOE-CR
Proportion of subjects achieving complete resolution of severe vaso-occlusive events, between 6 months and 18 months after drug product infusion
Time frame
6-18 months post-transplant2
The proportion of subjects achieving Globin Response
Globin Response, defined as meeting the following criteria for a continuous period of at least 6 months after drug product infusion: * Weighted average HbAT87Q percentage of non-transfused total Hb\* ≥30% AND * Weighted average non-transfused total Hb\* increase of ≥3 g/dL compared to baseline total Hb\* OR weighted average non-transfused total Hb\* ≥10 g/dL * non-transfused total Hb is the total g/dL of HbS + HbF + HbA2 + HbAT87Q
Time frame
6-24 months post-transplantSponsors and contacts
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Source ClinicalTrials.gov