CSL200 for 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

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 48 weeks for reporting.

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 more

Awards & 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

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