Stem Cell Transplant for Sickle Cell Disease
(PRECIZN-1 Trial)

Recruiting in Palo Alto (17 mi)

+4 other locations

Age: 18 - 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Bioverativ, a Sanofi company

No Placebo Group

Trial Summary

What is the purpose of this trial?This is an open label, multicenter, Phase 1/2 study in approximately eight adults with severe Sickle Cell Disease (SCD). The study will evaluate the safety, tolerability, and efficacy of autologous hematopoietic stem cell transplantation using BIVV003.

Eligibility Criteria

Adults aged 18-40 with severe Sickle Cell Disease (SCD) who are clinically stable for stem cell mobilization and transplantation. They must have a history of significant SCD complications, adequate organ function, and agree to contraception use. Excluded are those with contraindications to the drugs used, previous transplants, certain heart conditions, or active infections.

Inclusion Criteria

I have severe sickle cell disease with major symptoms or complications.

I have completed all my cancer screenings recommended for my age.

I am between 18 and 40 years old.

+5 more

Exclusion Criteria

Current enrollment in an interventional study or recent receipt of an investigational drug

Known allergy or hypersensitivity to specific medications or investigational product excipients

I have had cancer or a blood cancer in the last 5 years, or my family has a history of cancer.

+18 more

Participant Groups

The trial is testing BIVV003 in combination with Plerixafor and Busulfan for autologous hematopoietic stem cell transplantation in patients with severe SCD. It aims to assess safety, tolerability, and effectiveness of this treatment approach.

1Treatment groups

Experimental Treatment

Group I: BIVV003Experimental Treatment3 Interventions

Participants will receive plerixafor as subcutaneous (SQ) administration followed by myeloablative conditioning therapy with intravenous (IV) busulfan. BIVV003 will then be administered as a 1-time IV infusion of autologous Cluster of Differentiation 34 + Hematopoietic Stem/Progenitor Cell (CD34+HSPC) transfected ex vivo with zinc finger nuclease (ZFN) messenger ribonucleic acid (mRNAs) targeting the B-cell lymphoma/leukemia 11A (BCL11A) locus.