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New treatment option for Sickle Cell Disease
Official title Gene Editing For Sickle Cell Disease
ClinicalTrials.gov ID: NCT06506461
What this study is testing
What is Plerixafor?
Plerixafor is an investigational medicine, given as an once-daily injection under the skin, being studied as a potential treatment for sickle cell disease.
Also referred to as Mozobil®.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 24
You may be able to join if
- Age ≥18 years and ≤24.9 years.
- Patients with SCD (Hb SS, Hb SB0 and Hb SB+ genotype) who have experienced EITHER (a) 2 or more SCD-related vaso-occlusive events (acute pain events...
- Failure, intolerance, or refusal of hydroxyurea therapy.
- Patients must be eligible for autologous stem cell transplant as per investigator's judgment.
- Females of childbearing potential (i.e., those who are post-menarchal with an intact uterus and at least 1 ovary, and those who are less than 1 year...
You likely can't join if
- Availability of an human leukocyte antigen (HLA)-matched sibling who is willing and able to donate an appropriate graft for hematopoietic cell...
- Karnofsky or Lansky performance score \< 80.
- Pregnant, as confirmed by positive serum or urine pregnancy test within 14 days before enrollment (if female).
- Breastfeeding.
- Uncontrolled (undergoing appropriate treatment and with progression of clinical symptoms) or clinically significant bacterial, viral, or fungal...
- Patients with confirmed Hepatitis B or Hepatitis C infections.
See the full eligibility criteria
- Age ≥18 years and ≤24.9 years.
- Patients with SCD (Hb SS, Hb SB0 and Hb SB+ genotype) who have experienced EITHER (a) 2 or more SCD-related vaso-occlusive events (acute pain events, acute chest syndrome, priapism and splenic sequestration) per year in...
- Failure, intolerance, or refusal of hydroxyurea therapy.
- Patients must be eligible for autologous stem cell transplant as per investigator's judgment.
- Females of childbearing potential (i.e., those who are post-menarchal with an intact uterus and at least 1 ovary, and those who are less than 1 year postmenopausal) must agree to use acceptable method(s) of...
- Males must agree to use effective contraception from start of mobilization through at least 6 months post-infusion.
- Patients should be willing to participate in an additional long-term follow-up study after completion of this trial.
- Availability of an human leukocyte antigen (HLA)-matched sibling who is willing and able to donate an appropriate graft for hematopoietic cell transplantation (HCT).
- Karnofsky or Lansky performance score \< 80.
- Pregnant, as confirmed by positive serum or urine pregnancy test within 14 days before enrollment (if female).
- Breastfeeding.
- Uncontrolled (undergoing appropriate treatment and with progression of clinical symptoms) or clinically significant bacterial, viral, or fungal infections within 1 month before enrollment.
- Patients with confirmed Hepatitis B or Hepatitis C infections.
- Patients with confirmed seropositivity or positive nucleic acid amplification test (NAAT) for human immunodeficiency virus (HIV) or human T-cell lymphotropic virus (HTLV).
- Patients with a history of stroke.
- Serum conjugated (direct) bilirubin \> 2× the upper limit of normal for age, or serum alanine transaminase (ALT) \> 3× the upper limit of normal for age as per the local laboratory. Participants with hyperbilirubinemia...
- Left ventricular shortening fraction \< 25% or ejection fraction \< 45% by echocardiogram.
- Estimated creatinine clearance less than 60 mL/min/1.73m\^2.
- Diffusion capacity of carbon monoxide (DLCO) \< 50% (adjusted for hemoglobin) OR baseline oxygen saturation \< 85% in patients unable to perform pulmonary function tests.
- Prior HCT or gene therapy.
- Known hepatic cirrhosis, bridging hepatic fibrosis, or active hepatitis. Appropriate ultrasound or magnetic resonance (MR) imaging may be used to define the presence and degree of cirrhosis. Liver biopsy may be...
- Active known malignancy, myelodysplasia, abnormal cytogenetics, or immunodeficiency.
- Patients with history of a significant bleeding disorder.
- Cerebrovascular procedure within 6 months, including pial synangiosis for moyamoya.
- Patients with history of untreated moyamoya disease or presence of moyamoya disease at screening that in the opinion of the investigator puts the people at the risk of bleeding.
- Evidence of a pathogenic clonal variant in any candidate gene detected by a standard, licensed next-generation sequencing clinical assay for gene mutations associated hematological malignancies.
- Patients with history of intolerance, contraindication, or known sensitivity to plerixafor or motixafortide or busulfan. Prior anaphylactic reaction with excipients of the proposed product.
- Patients with participation in another clinical study with an investigational drug/product within 30 days of screening or fewer than 5 half-lives of the investigational agent whichever is longer from screening.
- Patients with history of alloimmunization to RBC antigens and for whom the investigator anticipates that there will be insufficient RBC units available for the duration of the study.
The study team makes the final eligibility decision.
Where it's taking place
- Memphis, Tennessee, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 24 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Memphis, Tennessee, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.