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Hematopoietic Stem Cell Transplantation

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

Phase 1 & 2

Waitlist Available

Research Sponsored by Bioverativ, a Sanofi company

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Severe SCD with specific manifestations such as clinically significant neurologic events, history of acute chest syndrome, frequent pain crises, priapism, regular RBC transfusion therapy, and echocardiographic finding of tricuspid valve regurgitant jet (TRJ) velocity

Ages 18 to 40

Must not have

History of active malignancy within past 5 years, hematologic malignancy, or family history of cancer predisposition syndrome

Contraindication to plerixafor, apheresis, or busulfan

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at weeks 13 and 52

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new treatment for sickle cell disease which involves transplanting stem cells. The goal is to evaluate the safety and effectiveness of the treatment.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects may include reactions related to the immune system's response to treatment, issues from stem cell mobilization like bone pain or nausea, and typical chemotherapy-related effects such as fatigue, digestive upset or blood count changes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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I am between 18 and 40 years old.

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I have been diagnosed with sickle cell disease.

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I understand the study's risks and goals and can sign the consent form.

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I am healthy enough for stem cell collection and a stem cell transplant.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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I cannot use plerixafor, undergo apheresis, or take busulfan due to health reasons.

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I have seizures that are not controlled by medication.

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I have undergone gene therapy before.

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I currently have a serious infection.

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I have a genetic variant affecting my blood's oxygen use.

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My blood has a high level of fetal hemoglobin.

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I have a history of serious bleeding problems.

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I have had a stem cell or organ transplant before.

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I do not have major issues with my brain, kidneys, liver, lungs, or heart.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at weeks 13 and 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at weeks 13 and 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and at weeks 13 and 52 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 Participants With Adverse Events (AEs)

Number of Participants With Serious Adverse Events (SAEs)

Percentage of Participants With Successful Engraftment

+3 more

Secondary study objectives

Change From Baseline in Haptoglobin Levels

Change From Baseline in Lactate Dehydrogenase (LDH) Levels

Change From Baseline in Patient-Reported Outcomes Measurement Information System 57 (PROMIS-57) Scale Score

+14 more

Awards & Highlights

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: 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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Plerixafor

2011

Completed Phase 3

~710

Busulfan

2008

Completed Phase 4

~1710