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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at weeks 13 and 52
Awards & highlights

PRECIZN-1 Trial 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.Check my eligibility
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.See study design
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.

PRECIZN-1 Trial 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.

PRECIZN-1 Trial Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Participants With Adverse Events (AEs)
Number of Participants With Serious Adverse Events (SAEs)
Percentage of Participants With Successful Engraftment
+3 more
Secondary outcome measures
CD34 + HSPC Yield from Plerixafor Stem Cell Mobilization
Change From Baseline in Haptoglobin Levels
Change From Baseline in Lactate Dehydrogenase (LDH) Levels
+19 more

PRECIZN-1 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
~720
Busulfan
2008
Completed Phase 3
~1120

Find a Location

Who is running the clinical trial?

Bioverativ, a Sanofi companyLead Sponsor
17 Previous Clinical Trials
1,028 Total Patients Enrolled
Sangamo TherapeuticsLead Sponsor
28 Previous Clinical Trials
941 Total Patients Enrolled
Medical MonitorStudy DirectorSangamo Therapeutics
1,649 Previous Clinical Trials
979,850 Total Patients Enrolled

Media Library

BIVV003 (Hematopoietic Stem Cell Transplantation) Clinical Trial Eligibility Overview. Trial Name: NCT03653247 — Phase 1 & 2
Sickle Cell Disease Research Study Groups: BIVV003
Sickle Cell Disease Clinical Trial 2023: BIVV003 Highlights & Side Effects. Trial Name: NCT03653247 — Phase 1 & 2
BIVV003 (Hematopoietic Stem Cell Transplantation) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03653247 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What criteria must potential participants meet to be eligible for this investigation?

"Prospective participants must have been diagnosed with anemia and sickle cell, as well as be between 18 and 40 years old to be considered for this medical trial. The total number of individuals accepted is limited to 8 persons."

Answered by AI

Are geriatric individuals excluded from participating in this experiment?

"Within the bounds of 18 and 40 years old, this study is allowing individuals to join."

Answered by AI

How pervasive is the clinical trial in this city?

"Karmanos Cancer Institute in Detroit, Michigan (Investigational Site Number 101), Children's Hospital of Philadelphia_Investigational site number 108 in Philadelphia, Pennsylvania and 6 other clinical trial sites are participating in this study."

Answered by AI

What diseases is BIVV003 known to ameliorate?

"BIVV003 is a popular medication for treating leukemia, as well as allogeneic hematopoietic stem cell transplant, chronic myelogenous leukemia, conditioning regimens for allogenic stem cell transplantation therapy and thrombocythemia-essential."

Answered by AI

Can you tell me what prior research has been done concerning BIVV003?

"Currently, there are 114 trials in progress researching the effects of BIVV003. Out of these experiments, 13 have reached Phase 3 and they all originate from Philadelphia, Pennsylvania - however, there are 1113 different sites running studies related to this drug."

Answered by AI

Are there any enrollment opportunities for this clinical trial at the present time?

"Indeed, the clinicaltrials.gov portal states that this medical trial is enrolling patients. First advertised on March 6th 2019 and most recently updated on September 13th 2022, 8 participants are sought from six distinct sites."

Answered by AI

What is the sample size for this trial?

"To properly execute this trial, 8 individuals that meet the criteria must be recruited. The primary sponsor and manager of the study is Bioverativ (a Sanofi company). Karmanos Cancer Institute in Detroit, Michigan and Investigational Site Number 101 located in Bethesda, Maryland are two sites where recruiting will take place."

Answered by AI
~2 spots leftby Jul 2025