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Gene Therapy
CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease
Phase 1
Waitlist Available
Research Sponsored by CSL Behring
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 48 weeks
Awards & highlights
All Individual Drugs Already Approved
No Placebo-Only Group
Summary
This trial is testing CSL200, a treatment for adults with severe sickle cell disease. It involves collecting special blood cells from the patient, using a drug to help move these cells into the bloodstream, preparing the body with mild chemotherapy, and then giving back the treated cells. The goal is to see if this approach is safe and effective.
Eligible Conditions
- Sickle Cell Disease
- Sickle Cell Anemia
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 48 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 48 weeks
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Number of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs) associated with the administration of CSL200
Secondary study objectives
Number of subjects receiving plerixafor and number of plerixafor doses administered by subject
Number of subjects undergoing apheresis and number of apheresis sessions by subject
The number of subjects undergoing reduced intensity conditioning with melphalan and able to receive CSL200
+2 moreAwards & Highlights
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: CSL200Experimental Treatment1 Intervention
Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
Find a Location
Who is running the clinical trial?
CSL BehringLead Sponsor
199 Previous Clinical Trials
1,205,080 Total Patients Enrolled
Study DirectorStudy DirectorCSL Behring
1,281 Previous Clinical Trials
500,501 Total Patients Enrolled