CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: CSL Behring

Disqualifiers: HPRT deficiency, TPMT deficiency, Alpha thalassemia, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 48 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop all current medications, but if you are on hydroxyurea, you will need to stop it before certain procedures in the trial.

Who Is on the Research Team?

Study Director

Principal Investigator

CSL Behring

Are You a Good Fit for This Trial?

Inclusion Criteria

Diagnosis of sickle cell disease with the homozygous HbS homozygous genotype (HbSS) or an HbSβ thalassemia variant (ie, HbSβ0 thalassemia or HbSβ+ thalassemia) genotype, confirmed by hemoglobin studies.

Fetal hemoglobin (HbF) ≤ 15%.

≥ 2 episodes of acute chest syndrome in the last 2 years.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Mobilization and Apheresis

Collection of CD34+ hematopoietic stem/progenitor cells by apheresis after mobilization with plerixafor

Up to 2 days

Conditioning

Reduced intensity conditioning with melphalan

2 days

Treatment

Administration of CSL200 and monitoring for adverse events

Up to 48 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CSL200

How Is the Trial Designed?

Group I: CSL200 Experimental Treatment 1 Intervention

Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734

Who Is Running the Clinical Trial?

CSL Behring

Lead Sponsor

Trials

204

Recruited

1,207,000+

Dr. Paul McKenzie

CSL Behring

Chief Executive Officer since 2023

PhD in Chemical Engineering from Carnegie Mellon University, B.S. in Chemical Engineering from the University of Pennsylvania

Dr. Bill Mezzanotte

CSL Behring

Chief Medical Officer since 2021

MD from Duke University