Actively Recruiting
Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease
Led by Essen Biotech · Updated on 2024-11-05
85
Participants Needed
1
Research Sites
73 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Have a diagnosis of sickle cell disease with b2S/b2S, b2S/b20, or b2S/b2+ genotype
- Be between 2 and 50 years old at time of consent
- Weigh at least 6 kg
- Have a Karnofsky performance status of 60 or higher if 16 years or older, or a Lansky performance status of 60 or higher if younger than 16 years
- Have been treated and followed for at least 24 months prior to consent at medical centers with detailed sickle cell disease records
- Have experienced at least 4 protocol-defined vaso-occlusive events in the 24 months prior to consent, with appropriate supportive care
- Have either failed hydroxyurea treatment or be intolerant to it as judged by the investigator
- Agree to use one method of highly effective contraception from screening to at least 6 months after infusion if of childbearing potential
- Provide written informed consent by the participant or legal guardian
You will not qualify if you...
- Medically appropriate for allogeneic hematopoietic stem cell transplantation with an available HLA-matched related donor
- Severe cerebral vasculopathy including history of stroke, abnormal transcranial Doppler requiring chronic transfusions, or imaging showing >50% stenosis or Moyamoya
- Positive for HIV-1, HIV-2, hepatitis B or C, HTLV-1, or active syphilis
- Active bacterial, viral, fungal, or parasitic infection
- Advanced liver disease including cirrhosis, active hepatitis, significant fibrosis, or liver iron concentration 615 mg/g unless biopsy shows no active disease
- Inadequate bone marrow function with low neutrophil or platelet counts
- Contraindications to plerixafor, busulfan, or other conditioning medications
- Need for therapeutic anticoagulation during conditioning through platelet engraftment
- Unable to receive packed red blood cell transfusion
- Prior allogeneic transplant
- Prior gene therapy
- Current or prior malignancy or immunodeficiency except certain treated skin cancers
- Immediate family history of familial cancer syndrome
- Female who is breastfeeding, pregnant, or planning pregnancy from screening to 6 months post-infusion
- Any condition making the participant ineligible for HSCT
- Participation in another investigational drug study within 30 days
- Chromosomal abnormalities or genetic mutations increasing risk of myelodysplastic syndrome or acute myeloid leukemia
- Genetic mutations causing inactivation of two or more b1-globin genes
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
District One Hospital
Beijing, Beijing Municipality, China, 086-373
Actively Recruiting
Research Team
R
Rhoda M Smith, Phd
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here